1. Understanding FDA’s Priority Review Voucher System
  2. FDA’s Expedited Development and Approval Programs
  3. Panel on Financial Incentives FDA Orphan Drugs Program and Priority Review Vouchers – June 10, 2019
  4. FDA Approval in HER2+ Breast Cancer, Priority Review in NSCLC, and More
  5. FDA Approval in NSCLC, Priority Review Designation in SCLC, CRL to Biosimilar, and More
  6. FDA Approval in AML, Priority Review Designation in NSCLC, and More
  7. Fuqua Research into Action: The Priority Review Voucher

Understanding FDA’s Priority Review Voucher System

hello everyone and welcome to,understanding FDAs Priority Review,voucher system presented by ro Im Erica,sagon ASCII editor with fierce pharma,and Ill be moderating this webinar our,speakers today are Kelly Ronnie senior,research at scientists with row Sheila,della Irizarry integrated product,development associate with Roe and Devon,Rosenthal research scientists with Roe,you can read their full BIOS on the left,side of your window just a few technical,notes before we begin if youd like to,download the slide deck please click the,files button at the lower left corner of,your screen this webinar is being,recorded and will be available on demand,within 24 hours after the event we will,follow the presentations with a Q&A,session please submit your questions,during or after the presentations by,clicking the Q&A button on the lower,left corner of your screen now lets,begin Devin please go ahead thanks to,the introduction and good afternoon,everybody,next slide please so to start talking,about the Priority Review voucher system,what were going to do is set a,framework for this discussion and talk,about what the standard pharma model is,for incentivizing drug development so,for a traditional drug within Pharma you,look for a disease that has a large,population of patients that need some,sort of therapy next slide please,you then look for this population of,patients to have the ability to pay for,the therapy next slide and next slide,and then,goes ahead and develops a drug to target,this disease affecting this large,population of patients sorry we can go,back its live Pharma develops therapy,to target the disease affecting this,large population of patients with the,ultimate result of profit for the,pharmaceutical company and so this is,just a high-level overview of what the,traditional incentive model looks like,and what some of the variables impacting,that are we go to the next slide we can,take a look at non-standard cases and so,in particular and relevant to this,conversation will be rare diseases and,tropical diseases we go to the next,slide you can see if we use the same,variables in these equations for rare,disease first off what shifts is a,patient population size youre dealing,with a much smaller number of potential,patients who still have the ability to,pay and so a therapy could be generated,for them if we go to the next slide and,take a look at tropical diseases you see,the balance here ships where you have a,much larger population of patients these,are typically more global diseases,however theres not the same potential,for reimbursement or payment from these,patients for therapies that are,developed next slide consequently,basically shifts in these variables,theres a much smaller potential profit,at the end of the line for,pharmaceutical companies next slide,which just from a purely business,standpoint often its not a strong,incentive for companies to develop,therapies in these spaces despite what,need there might actually be you know,for these patient populations next slide,and so the question then is how can this,process of drug development actually be,incentivized in a way that makes sense,from a business standpoint for these,pharmaceutical companies to move ahead,next slide and this is where we get into,discussion of the Priority Review,voucher system next slide so here Im,going to give a high-level overview of,how Priority Review voucher incentivize,the drug development process and well,go into some more detail about all the,various aspects and regulations around,all of this and subsequent slides but,from a high-level view if you have a,biotech company,or a pharma company and youre,developing a therapy for either a,qualifying tropical disease or rare,pediatric disease and you carry that,drug through to approval and the ARB LA,approval you are eligible to receive one,of these Priority Review voucher x next,slide these review vouchers incentivize,this process and essentially two,different ways the first way is that,your company can actually use this,Priority Review voucher for other,therapies youre developing not just,tropical disease or rare pediatric,disease targeting therapies like you get,the next slide please,but any therapy in your pipeline and,this provides Priority Review so it,shortens the NBA or BL a review time,giving you a competitive advantage in,terms of getting to market quicker next,slide the other mechanism is something,thats unique to the voucher system and,a more common topic of conversation when,you hear about this the Priority Review,voucher system and this is that the,vouchers can actually be sold to another,company so your company develops a,therapy for one of these rare and,neglected diseases you can then sell,this for some level of profit to another,company and the proceeds from that sale,return to your company to help fuel,additional development of other,therapies and so a quick anecdote about,this this is it obviously provides a,nice windfall at the end of the path you,know for your company thats developing,this therapy this also has upstream,benefits those companies are looking to,fundraise early on in development and so,I can tell a specific example of being,at a conference a number of months ago,and seeing a CEO pitch to a group of,investors and the CEO was CEO of a,company that was developing a disease,era P targeting a very rare pediatric,disease I want to say total patient,population of about 10 patients globally,and I was very interested to see how,this would go because you have this room,of investors theyre used to seeing,these presentations about much larger,populations much larger potential,payments and reimbursement potential for,a drug and theyre all not even long and,when somebody asks how are you going to,make money off of this,and the CEO says well were going to be,eligible for one of these Priority,Review voucher I certainly looked at the,room and saw all these investors nod,their heads along presentation went,along and this generated a lot of,interest and so its not just the,windfall at the end of the road its the,incentive that it provides upstream as,well in order to help develop these,therapies for diseases that may,otherwise not have companies focusing on,them next slide please,and so if we take a look you can see,that the sale process actually works in,the real world theres been four,vouchers sold to date that theres,publicly available sell information,about and you can see this has been an,increasing sale price process so the,initial voucher sold for somewhere,around sixty to seventy million dollars,and the most recently reported sale was,up around three hundred fifty million,dollars and remember this is money,thats going back into the pockets of,these companies to help fuel this drug,development process next slide Ill turn,this over to Kelly whos going to talk,into more detail about the Priority,Review voucher themselves next slide,please,Priority Review voucher czar vouchers,have the potential to reduce approval,time for a drug these vouchers reduce,review time of a marketing application,by FDA from ten months which is the,standard review time to potentially six,months which is the priority review time,you will often hear this voucher in the,literature and out-and-about referred to,as a golden ticket or the prize it is,granted at marketing approval and is an,incentive for companies to produce drugs,for neglected tropical diseases and rare,pediatric diseases the voucher can then,be used for priority review of any,subsequent application together this may,mean faster access to treatment for,patients as well as the potential for,more profit for a drug company next,slide,the Priority Review voucher idea was,first proposed from david ridley,Henninger belsky and Jeffrey Moe at Duke,University and their 2006 paper,developing drugs for developing,countries and the idea in this paper is,that each is linked to drugs for,diseases that disproportionately affect,developing countries are underprivileged,c

FDA’s Expedited Development and Approval Programs

hello everyone and welcome to FDAs,expedited development and approval,programs presented by Rose and Morgan,Edwards McGee operations manager of life,sciences at fierce markets and Ill be,moderating this webinar our speaker,today is David shoemaker senior vice,president of R&D at row you can read,this full bio on the right side of your,window just a few technical notes before,we begin if you have trouble reading a,slide please hold and drag the right,corner of the slide window to enlarge it,please also disable your pop-up blocker,to participate in the interactive parts,of the presentations if youd like to,download the slide deck please click the,purple globe at the bottom of your,screen this webinar is being recorded,and will be available on demand within,24 hours after the event we will follow,the presentation with a Q&A session,please submit your questions during or,after the presentation using the Q&A box,at the right of your window okay were,ready to begin David please go ahead,thanks very much okay what Id like to,do for you today is to outline the FDA,is the u.s. FDAs expedited development,and approval programs thank you have the,next slide theyre listed here,accelerated approval breakthrough,therapy designation Priority Review and,fast-track and as I note here you may,qualify for more than one program Im,going to go through these in order in,the relative value that we at row,assigned to these programs and I do want,to point out that there is a 2014,guidance that the FDA has put out on,these programs it summarizes it very,well and so Im not going to repeat that,guidance but Im going to add some color,around these programs based on my,experience and Rose experience in,general with the different programs the,next slide sort of deals with the first,of a couple definitions Im going to,give I think its important for everyone,to read and understand this serious,life-threatening disease or condition,youll find this populated all over the,guidance and,it still is somewhat you know unclear to,some people what this means and so the,only way to really have this agreed upon,with FDA is to talk with FDA about your,particular program and whether or not,you are treating a serious and,life-threatening disease some such as,you know oncology indications or,life-threatening rare diseases its,pretty self-evident others it can be,more ambiguous so consequently I want to,encourage you to engage with FDA in,discussing your program if youre going,to apply for one of these programs the,next is available therapy this is,described pretty well in the guidance,and obviously theyre talking about and,approved or licensed u.s. product that,is for the same indication that you are,seeking its also you know the,consideration needs to be given for the,standard of care for a particular,disease or condition that youre,examining so these are things that you,need to be aware of for your particular,development program so unmet medical,need this is fairly straightforward,where there is no available therapy for,the disease or the condition that youre,pursuing however when there is an,available therapy and the product that,youre developing needs to distinguish,itself in some way shape or form such as,some improved efficacy or some improved,safety profile it really needs to be,clear to both you and to FDA dont just,assume that FDA is going to agree with,you on the novel property of your your,therapy so that needs to be discussed,and also you may have been instances,where theres another accelerated,approval product that has already been,granted market approval and that may be,waiting for validation in a post,marketing trial but you may,to consider compounds such as that when,youre developing your program so lets,jump right in to the accelerated,approval program and what is this and,what does it mean this program has been,around since the early 90s,for drugs and it was conferred on,biologics as well and its been recently,updated and amended in the fideya Act in,2012 and basically the products that are,eligible are those with benefit over,existing treatments and again standard,of care needs to be considered in this,as well,and if a product is in a horse race with,your product and it makes it to market,ahead of your by a few months FDA still,must consider that product in its,evaluation of your program so were all,in a horse race I think we realize that,we have to stay apprised of competitors,as we develop our products the benefits,of this program are significant you know,basically the major advantage is you,essentially do away with phase 3 youre,able to do 1 versus 2 adequate and well,controlled studies that are required for,the approval of most standard therapies,consequently it shortens the development,time and again in in our opinion this is,without doubt the most valuable,expedited program for a company to,engage in but what is the catch the,catch is that a surrogate endpoint is,required and a lot of people assume that,theyre going to be able to take,advantage of this program without really,defining or agreeing with FDA what the,surrogate endpoint is so lets spend a,little bit of time on discussing,surrogate endpoints and Ill show you in,the next slides at FDA,has actually issued a publication,recently in July on on surrogate,endpoints but its not necessarily a,final approval criteria it is an,indication to FDA that your product has,a very good chance of warranting,approval at some point so it can be an,assessment of some aspect of the disease,for instance in cancer the ultimate,yardstick that FDA uses to evaluate the,efficacy of a cancer drug or biologic is,is survival however there are,measurements such as objective response,rate or you know extension of survival,that that FDA will accept in the interim,and its generally allows your clinical,studies to be conducted in smaller,numbers over shortage of periods of time,it takes an enormous amount of time in,order to evaluate survival so if youre,able to demonstrate the FDA a reduction,in tumor burden or overall response rate,that will be an indication to them that,it potentially could result in overall,survival but that survival study will,still be asked for by the FDA they will,want validation that whatever effect,youre measuring is a surrogate endpoint,is validated eventually after they,approve your product via the accelerated,approval pathway so this is the diagram,that I think probably would have been,useful 20 years ago because I dont,think it was as well understood as its,becoming today even within FDA so if you,look at the right-hand side clinical,endpoints thats what FDA is concerned,with for approval and everything short,of that is makes FDA nervous,degree so validated surrogate endpoints,are surrogate endpoints that have been,demonstrated time and again to result in,a clinical endpoint that FDA considers,sufficient for approval surrogate,endpoints are our surrogate endpoints,that are still being evaluated by FDA,they havent been convinced that they in,all cases lead to the clinical endpoint,that they desire before they make an,approval and biomarkers you know weve,been working on biomarkers and FDA has,been supporting the development of,biomarkers for 10 or 15 years I still,dont think that we have a biomarker,that is adequate to convince FDA that it,will result in a defined clinical,endpoint sufficient for approval I think,another thing that I learned I didnt,realize this it was in this document,that FDA published in July was that,between 2010 and 2012 the FDA approved,45% of their drugs on the basis of a,validated surrogate endpoint so FDA is,is going this way theyre encouraging,everybody to develop biomarkers to,develop surrogate endpoints but unless,your surrogate endpoint is a validated,surrogate endpoint youre going to have,some additional work to do post,marketing potentially to give you some,examples I just thought it would be,useful to give you some examples of some,validated surrogate endpoint these are,the ones that FD

More: them review

Panel on Financial Incentives FDA Orphan Drugs Program and Priority Review Vouchers – June 10, 2019

like to go over something you are,joining your conference room,you are the host back that is immediate,for a menu in vaccines deferred how does,all the rules apply the same way for,vaccines so epi doesnt have a,preference for whether you are seeking a,voucher for drug application versus a,vaccine application if you meet all the,requirements of the different PRV,programs then youre eligible for,issuance of a PRV,the only distinction is that for,vaccines its generally regulated by,Center for biologics Evaluation and,Research so if you need to connect with,FDA then there there are different,offices and divisions within Sieber so,that they can walk you through some of,the requirements for for POV for,vaccines and of course drugs are mainly,regulated under cedar so if you have,interests in any of the drugs of for PRV,then you will contact one of those,appropriate review divisions responsible,for the review of that drug product in,cedar question – can you get a rare,pediatric disease status if you do not,pursue pediatric inpatient as your first,marketing indication so if youre,talking about adding a rare pediatric,disease indication to your currently,approved application then the answer is,no because one of the common requirement,is that the drug does not or the,application does not have an active,ingredient thats already approved so,you will not meet that requirement if,you were to add a pediatric indication,thats not your first martini,communication question three well the,decision to renew the rare pediatric,disease PRV program be made before the,sunset dates are there any indications,that suggest renewal or non-renewal so,unfortunately at this point we dont,have any information on whether the are,PDP RV program will be renewed but the,are PDP RV statute does instruct the,Government Accountability Office or,accounting office to provide a study on,how effective the RP deeper,is and I believe that study should be,undergoing at this point or once way,under at this point on so down the road,we may have more information but at this,point unfortunately is too early in the,process and we do not have that,information,number four kindly give your thoughts on,any difference between the PRV for drugs,or biologics and if for biologics where,do we reach out for the application for,the PRV so again you know as what,question one FDA does not have a,preference for drugs versus biologics if,youre interested in pursuing an,application that we eligible for PRP on,and again you know we do have Center for,biologics Evaluation research for,certain products that are regulated by,Sieber and then some biologics are also,regulated by cedar so you will need to,find out the appropriate review division,and reach out to that review division to,determine your qualification for such,drug products number five when is the,right time to submit a PRV application,after opening of a 90 or so the probe,with them is if you are interested in,submitting an application that will,qualify for particular PRV contact the,review division or the appropriate,review divisions as soon as possible,that way they can walk you through the,requirements for demonstrating whether,your product will be eligible for PRP,and the sooner you start that process,the better off you will be because then,you will understand like you know what,you would need to prove or show the FDA,in order to be eligible for PRP question,six is there a specific FDA for more,specific format for PRV application the,current guidance which was discussed say,today as well does not specify the,method for application also at what,stage of the drug development cycle,should the application be done so for,the first part of the question FDA does,not have a specific form to fill out for,PRV application basically what happens,is that when you request the FDA to,evaluate whether you will be eligible,for,you will need to demonstrate in the,application how you will meet all the,conditions required for FDA to issue a,prv,that said for a rare pediatric disease,PRB you can submit a request to the,office orphan product development to,receive a rare pediatric disease,designation and that will be beneficial,in terms of the time insensitive on for,rare pediatric disease pRb so thats the,only exception see the other PRV,programs at Chapel disease and medical,countermeasures we do not have a,specific foreign require and again you,know the parallelism is always,communicate with the dozen or the,appropriate review division or office,thats responsible for your application,well in advance so you know what those,requirements are when we submit your,application so that last remark dresses,the second part of this question,question 7-0 flexibility on FDA side on,which age range satisfies the pediatric,voucher based on the sponsors actual,phase 3 data for example maybe it was a,Greek on proxy of phase 3 that ages 0 to,18 was satisfied PRD but then after a,phase 3 it is found that only ages 5 to,18 show clinical effectiveness so the,requirement for rare pediatric disease,application is that the disease must,affect individuals less up to actually 0,to 18 years of age and FDA interpret,that to be anyone below 19 years of age,so we show that the the disease affects,individuals ages 5 to 18 years of age,then that will satisfy portia portion of,the rare pediatric disease requirement,and also the second requirement for rare,pediatric disease is that it must affect,the pediatric population of less than 19,years of age over 50% in prevalence so,even if there are you know a lot of,adults who are affected on this disease,as long as we show that the majority of,the patient population effect there are,pediatrics then you should,okay okay question eights for rare,pediatric disease should the population,go up to the to the 18th birthday or up,to the 19th birthday and I believe I,just addressed that um it will be anyone,who is less than 19 years of age so it,could be anywhere up to that 19th,birthday so one day before the 19th,birthday,second part of this question if RPD,sunsets on September 30 2020 what,happens that the application that the,RPD is based on is not approved so if,the application is not approved when,that sunset date on the sunset provision,provides an additional date September 30,2022 where the application must must be,approved by it so you still have,additional two years after that after,September 30 2020 on so if your,application is not approved by September,30 2022 and theres no reauthorization,for the Arkadiy PRV program then you,will no longer be eligible for pediatric,disease PRP what happens if the doctor,is not redeemed,so once the voucher has been issue,theres no expiration on the use of the,voucher at this point that Im aware of,of course you know Congress can always,enact some ball down the road but at,this point theres no expiration once a,doctors issue on you can use it,indefinitely in the future question 12,does the original word rare disease and,DA need to be approved before PRV is,issued and the answer is yes because the,PRV is not effective until the,application is approved and also certain,conditions make change such as if,another application containing the same,active ingredient were approved then you,will no longer meet one of those,conditions for PRV issuance again I just,talked about the sunset provision if the,application is not approved prior to,when the program sunset then if they may,not be able to issue you PRD a question,Im gonna skip question 13 for now,question 14 is the PRD fee of 2.4,million in addition to the NDA,application fee of 2.4 million so the,responses yes,PRV fees that are due are in addition to,any fees that are required under the,Prescription Drug User Fee Act so in,this case if you were to submit an,application that requires a full,participate then youre looking at about,five millions in total fees and then,number 16 can a sponsor apply for both,and be granted both f6 savings under a,written request and our PD PRP so Im,not

More: accubow 2 0 review

FDA Approval in HER2+ Breast Cancer, Priority Review in NSCLC, and More

today an FDA approval and her2 positive,breast cancer a prior to review,designation and lung cancer a,breakthrough therapy designation in head,and neck cancer applications accepted in,ovarian cancer and breast cancer and an,O doc decision in lung cancer,welcome to Enclave news network Im,Jeana Columbus the FDA has approved a,supplemental new drug application for,narrative in combination with capes Ida,beam for the treatment of adult patients,with advanced or metastatic her2,positive breast cancer who had received,at least two prior anti her2 based,regimens in the metastatic setting the,approval is based on findings from the,phase three Nala trial which show that,the combination of neurontin M and keeps,Ida beam reduced the risk of disease,progression or death by 24 percent,compared with lapatin M plus Capeside,abeam moreover the objective response,rate was thirty two point eight percent,for the narrative arm and twenty six,point seven percent for the lapatinib,arm respectively the median duration of,response was eight point five months,versus five point six months,respectively,neurontin was initially approved for the,extended adjuvant treatment of adult,patients with early-stage her2,overexpressed or amplified breast cancer,following adjuvant trust whos mad based,therapy and non small cell lung cancer,the FDA granted a priority review,destination to a supplemental new drug,application for bra got nib for the,first line treatment of patients with al,causative metastatic disease as attacted,by an fda-approved,test the application is based on the,phase three Aalto one L trial in which,treatment with forgotten M demonstrated,a 57 percent reduction in the risk of,disease progression or death versus,Crisanta nib in patients with advanced,out positive NS ALC who had not received,a prior alkene hibbott ur the two-year,investigator assessed results also,showed that forgotten M blade to a 76,percent reduction in the risk of disease,progression or death in newly diagnosed,patients who had brain metastases at the,time of enrollment moreover with the,two-year follow-up data the trials,results were evaluated and reported by,both study investigators and blind an,independent review committee at the data,cutoff for the second interim analysis,results showed that by independent,review assessment there was a 51%,reduction in the risk,of disease progression or death with,forgotten him over chrisandem the FDA is,expected to make a decision on the s NDA,by June 23rd 2020 the FDA has granted a,breakthrough therapy designation to,Debbie oh one one four three for the,treatment of patients with previously,untreated unresectable locally advanced,squamous cell carcinoma of the head and,neck in combination with standard,cisplatin based concomitant,fractionation chemo radiation therapy,the designation is based on results from,a phase one to trial and which Debbie L,one 143 plus CRT demonstrated a twenty,point eight percent improvement an,investigator assessed 18-month local,regional control rate compared with CRT,plus placebo after two-year follow-up,period progression-free survival also,favored the Debbie oh one one four three,arm Debbie oh one one forty three was,also found to have a predictable and,manageable safety profile and did not,compromise the full delivery of standard,CRT w11 43 is a potential first in-class,oral antagonist of inhibitor of,apoptosis protein that sensitizes tumor,cells to chemo radiation by promoting,programmed cell death and fostering,anti-tumor immunity an ovarian cancer,the fda accepted a supplemental new drug,application for nur opera for use as a,frontline maintenance treatment for,women with advanced ovarian cancer who,responded to platinum-based chemotherapy,regardless of biomarker status the,application is based on findings from,the phase three prima study in which,frontline maintenance therapy with nur,opera improved medium progression-free,survival by five point six months,compared with placebo for patients with,newly diagnosed advanced ovarian cancer,who responded to platinum-based,chemotherapy in the overall study,population the median PF s and the nur,opera arm was thirteen point eight,months versus eight point two months in,the placebo group translating to a,thirty eight percent reduction in the,risk of progression or death with the,addition of the PARP inhibitor in,patients with tumors that tests positive,for homologous recombination deficiency,the meeting PFS was twenty one point,nine months with nur opera verses ten,point four months for placebo shortly,after the FDAs acceptance the European,Medicines Agency validated a type two,variation foreigner opera as a,first-time maintenance treatment for,pain,with advanced ovarian cancer who have,responded to platinum-based chemotherapy,regardless of biomarker status in breast,cancer the fda accepted a biologics,license application for a fixed dose,combination of per to slab and trusters,map with hyaluronidase administered by,cutaneous injection in combination with,intravenous chemotherapy for the,treatment of eligible patients with her2,positive disease the application is,based on findings from the phase three,fredericka study and which the,subcutaneous fixed dose combination of,Purdues mAb and trastuzumab,demonstrated non-inferiority to,intravenous formulations of the two,drugs with respect to pharmacokinetics,clinical activity and safety the total,pathologic complete response in the,breast and Xillia and safety also were,similar between the two treatment,regimens the safety analysis showed that,incidences of the most common adverse,events were similar between both arms,including alopecia nausea diarrhea,anemia and asthenia the FDAs oncologic,drugs Advisory Committee voted six to,five in favor of intravenous Ram a serum,AB injection for use in combination with,erlotinib as a frontline treatment for,patients with metastatic non-small cell,lung cancer whose tumors Harbor EGFR X,and 19 deletions or exon 21 substitution,mutations the FDA schedule the oncologic,drugs Advisory Committee hearing to,discuss data supporting a supplemental,biologics license application for the,combination in this setting which was,based on findings from the Phase three,relay trial and the study results show,that the addition of REM serum AB to,erlotinib that led to a 41 percent,reduction in the risk of disease,progression or death compared with or,latinum alone in the first line,treatment of patients with EGFR mutated,NSEL see the committee saw to discuss,whether the risk benefit profile of irma,serum AB plus or latinum could be,adequately assessed without overall,survival data and whether the,improvements seen in PFS is clinically,meaningful in the context of additive,toxicity and the combination arm in,relay the rate of grade 3 or higher,adverse events was 72% compared with 54%,for a lot in abalone and the rate of,serious adverse events was 29% and 21,percent respectively this week resides,Allen dr. Richard S Fenn,the University of California Los Angeles,to discuss the use of immunotherapy and,patients with unresectable,hepatocellular carcinoma in our,management of unresectable liver cancer,weve really seen some dramatic changes,in the past few years for the for the,for a decade or longer all we had was,Sarandon which had shown its ability to,improve survival in the first line,setting and then for many many years we,had numerous phase three failures both,in the frontline setting as well as in,the second line setting and in the past,few years weve seen the results of,several phase three studies which have,led to approvals of new drugs in the,first-line settings such as Levante nib,and in the second line setting Cabazon,nib reg RAF nib and Remy serum at,theres been a big interest in immuno,Oncology agents in all of cancer,medicine and this is eventually filtered,down into the liver cancer space both,Nivola mAb and Pember lism ab have,received accelerated approval in the,second line setting based on phase one,two studi

FDA Approval in NSCLC, Priority Review Designation in SCLC, CRL to Biosimilar, and More

today an FDA approval and non-small-cell,lung cancer a priority review,designation in lung cancer a complete,response letter for a trust whose am a,biosimilar halted enrollment on an easy,h2 inhibitor trial and disappointing,findings in trials of kidney cancer and,your Thiele carcinoma,welcome to Enclave news network Im Gina,Columbus the FDA has approved,omerta nib as a first-line treatment for,patients with non small cell lung cancer,whose tumors Harbor EGFR mutations,specifically exon 19 deletions or exon,21 l85 8 are substitution mutations the,approval of the third-generation,irreversible EGFR tyrosine kinase,inhibitor is based on the pivotal,results of the phase 3 flora study in,which frontline o smert Neb reduced the,risk of progression or death by 54,percent versus standard therapy with her,lot nib or japhet nem in the,double-blind study the median,progression-free survival was ten point,two months with standard therapy and,eighteen point nine months with OSA,Martin M the PFS benefit with OSA mert,nib extended across all pre-specified,subgroups in patients with brain,metastasis the median PFS with,aasta.martin M was fifteen point two,months compared with nine point six,months with standard therapy moreover,the objective response rate with OSA,Martin M was 77 percent versus 69,percent with erlotinib and Chaffin M the,median duration of response with OSA,Burnham was seventeen point six months,versus nine point six months in the,comparator arm also a non small cell,lung cancer the FDA has granted a,Priority Review designation to a,supplemental biologics license,application for an Ebola map for the,treatment of patients with small cell,lung cancer with disease progression,following two or more lines of therapy,the application is based on data from,the phase one – checkmate,o three to trial in which single agent a,bola mob led to a median overall,survival of four point four months and a,one-year OS rate of 33% in patients with,progressive SCLC following more than one,prior line of therapy under the priority,review the FDA is scheduled to make its,decision by August 16th,2018 the open-label phase one -,checkmate,oh three to trial evaluated Nivola matt,mono therapy or the combination of,nivolumab and apple uma mad at different,dosing schedules and patients with,advanced or metastatic solid tumors,including SCLC the median OS in the,three milligram Ebola mom one milligram,epi luma matte group was 6.0 months and,the one year OS rate was 35% and the one,milligram Nivola mom 3 milligram voluma,mab arm the median OS was seven point,seven months and one year OS was 43% the,overall response rate in the single,agent nivolumab arm was 10% and the ORR,s were 23 percent and 19 percent in the,one milligram nivolumab,3 milligram epi luma map and 3 milligram,nivolumab one milligram of aluminum arms,respectively the median duration of,response was not yet reached with single,agent of ala mom the FDA has issued a,complete response letter to Pfizer,regarding a biologics license,application for the trust chisme,biosimilar PF zero five two eight zero,zero one for citing the need for,additional technical information Weiser,had previously reported data from the,reflections be three two seven zero to,study at the 2017 as no Congress would,show that the biosimilar had achieved,equivalence an objective response rate,versus trustees mab when the agents were,combined with paclitaxel for the,frontline treatment of patients with,her2 positive metastatic breast cancer,survival rates at one year were 56,percent with the biosimilar and 52,percent with trust whose mob im,progression-free survival rates for,eighty eight point eight four percent,versus eighty seven point nine six,percent respectively a separate trial,reflections be three two seven zero four,was presented at 2017 as no congress and,also demonstrated clinical equivalence,regarding the efficacy and safety of the,Pfizer biosimilar and trustees mob,trustees mom has approved FDA,indications for the treatment of,patients with her2 positive breast,cancer as well as her2 positive,metastatic gastric or gastroesophageal,Junction adenocarcinoma the only,fda-approved biosimilar for chest whos,mad is my l1 4:01,Oh also non known under the trade name,of very the FDA has halted enrollment on,clinical trials of the easy h2 inhibitor,Tazz met Assad and patients with various,solid tumors and hematologic,malignancies the agency placed the,partial clinical hold on the Tees,medicine program after Epping the,manufacturer of the drug provided a,safety update detailing a pediatric,patient with advanced poorly,differentiated chordoma enrolled in a,phase one trial who developed a,secondary t-cell lymphoma the patient,had been enrolled in the study for,approximately 15 months at the time of,the safety update and had reached a,confirmed partial response following the,diagnosis of the secondary malignancy,the patient stopped his medicine and is,now receiving therapy for t-cell,lymphoma,according to Epis im this is the only,incident of secondary lymphoma reported,among over 750 patients who have,received the agent in their clinical,program previously enrolled patients,without disease progression can continue,on study the tazed medicine program is,exploring the drug as a single agent,across several tumor types including,epithelioid sarcoma follicular lymphoma,diffuse large b-cell lymphoma and,mesothelioma combination trials are,exploring the agent in DLBCL and,non-small-cell lung cancer in renal cell,carcinoma interim analysis findings of,the Phase three adapt trial revealed,that the immunotherapy ro cobble density,is unlikely to meet any of the studies,primary endpoints in patients with,metastatic disease based on the findings,our ghost therapeutics the manufacturer,of the agent has ended the trial one of,the four Co primary endpoints median,overall survival and the intent to treat,population was twenty eight point two,months for the combination of,republicansll t with standard therapy,versus thirty-one point two months with,standard therapy alone two other primary,endpoints that were missed at the most,recent analysis were OS for patients who,are alive at the previous interim,analysis,in February 2017 and OS among all,patients with at least 12 months of,follow-up data there was insufficient,data to assess the fourth Co primary,endpoint of five-year survival Argos had,recently submitted a protocol amendment,for the trial to the FDA detailing his,for Co primary endpoints the median,survival in the control group is the,longest reported to date from any study,of the intermediate porvis patients with,metastatic RCC results of the Phase,three,range trial showed that the combination,of REM serum AB and docetaxel in,patients with locally advanced or,unresectable metastatic Yura field,carcinoma who progressed on,platinum-based chemotherapy led to a,positive trend but not a statistically,significant improvement in overall,survival the findings come from an,analysis of the secondary endpoint of OS,previously reported data showed that the,study met its primary endpoint with an,improvement in progression-free survival,of 1.3 1 months the combination also led,to a near doubling in the objective,response rate vs. docetaxel alone at a,median follow-up of five point zero,months in the intent to treat population,the investigator assessed median PFS was,four point zero seven months with the,combination therapy versus two point,seven six months for patients who,received docetaxel alone by independent,blinded assessment median PFS was four,point zero four months versus two point,four six months in favor of her in a,serum AB and docetaxel this week we sat,down with dr. Reuter a Love Rush,University Medical Center to discuss,factors influencing adjuvant therapy use,for her2 positive breast cancer theres,a number of factors that we take into,account when we decide which adjuvant,regimen to use and these would include,the nodal status the tumor size the,hormone receptor

FDA Approval in AML, Priority Review Designation in NSCLC, and More

today an essay approval in acute myeloid,leukemia a priority review designation,and non-small-cell lung cancer an,exclusive sit-down interviews with,doctors Tanya spoke eyes Bob and Jeffrey,Oxnard,welcome to enclave news network Im Gina,Columbus the FDA has approved CPX 351,for adult patients with newly diagnosed,therapy related acute myeloid leukemia,or AML with mild dysplasia related,changes the approval for CPX 35:1 which,is a fixed combination of donna rubus n,n cetera beam comes several months ahead,of the fda deadline it was based on an,improvement in overall survival in a,phase three study comparing CPX 351 with,traditional site era beam and on Aruba,sin known as 7+3 for patients with newly,diagnosed T AML or a ml MRC results,showed that the median OS was nine point,five six months with CP x three five one,versus five point nine five months with,seven plus three representing a 31%,reduction in the risk of death,additionally the complete response or CR,with incomplete platelet or neutrophil,recovery rate was forty seven point,seven percent versus 33.3% for CP x 3 5,1 and 7 plus 3 respectively for CR alone,the rates were thirty seven point three,percent for CP x three five one and,twenty five point six percent for seven,plus three at twelve months the OS rate,was 41.5% in the CP x three five one arm,versus twenty seven point six percent in,the seven plus three group at 24 months,thirty one point one percent of patients,enrolled in the CP x three five one arm,of the study remained alive compared,with twelve point three percent with,seven plus three,the median event-free survival was two,point five three months with CP x 35 one,versus one point three one months with,seven plus three CP x three five one was,approved with a boxed warning advising,against interchanging the medication,with other donna rubus n and or cetera,beam containing products the FDA also,against using CPX 351 in patients with a,history of serious hypersensitivity -,Don Aruba son cetera beam or any,component of the formulation and,non-small-cell lung cancer,the FDA has granted a priority review to,a supplemental new drug application for,elective for the frontline treatment of,patients without positive locally,advanced or metastatic disease the,agency is scheduled to issue a final,ruling by November 30th 2017 election,aid was previously approved for patients,with metastatic alka positive NS plc who,have progressed on or are intolerant to,creatinine the frontline application is,based on results from the phase 3 Alex,and Jay Alex studies when compared with,kasatonov a lexington improved,progression-free survival by 53% in the,Alex study and by 62% in the je Alex,trial a Lexington was also found to,reduce the risk for progression in the,central nervous system by 84% versus,crizotinib in the Alex trial the,12-month cumulative array of CNS,progression for patients with or without,existing CNS metastases at baseline was,nine point four percent in the election,of arms and 41.4% for crizotinib this,week we sat down with dr. tanios Bach I,fob of Mayo Clinic to share his insight,on the recent FDA accelerated approval,of nivolumab,for the treatment of adult and pediatric,patients with microsatellite instability,high or mismatch repair deficient,metastatic colorectal cancer that has,progressed following treatment with a,florid pyrimidine oxaliplatin and arena,chicken we have now accumulated large,amounts of data about MSI high,colorectal cancer which occurs in about,four percent of patients with metastatic,colorectal cancer its a higher rate in,the earlier stages but for this,discussion of course the relevance is,metastatic colon cancer colorectal,cancer so four percent is not a small,group so it says meaningful a number of,patients given the number of patients,that get diagnosed with metastatic,colorectal cancer,every year in the United States and so,that has significant implications we,knew there was a prior approval with,member lizallen and MSI high solid,tumors where the the the main study,included colorectal cancer that were MMR,proficient versus MMR deficient that,suggested that MMR deficient or MSI high,tumors have close to 60 Plus percent,response rate and these responses can be,durable and with follow-up action a lot,of them end up being complete responders,and then evolve in lab essentially has,its own data in colorectal cancer and,MSI high showing very similar trends,large response rate many complete,responders durable response very similar,profile and then led to the second you,know approval in the space for new,volume at the MSI high approval was for,colorectal cancer only with fabulous,amendments for all solid tumors but,nonetheless I think this adds another,option for our patients with metastatic,colorectal cancer in the MSI high space,of course the question becomes how do,you choose between the two we have data,with federalism and we have data with,known evil in lab theyre both in MSI,high they both look very similar so how,do you choose between the two there,again theres no data that compares the,two head-to-head historically they seem,to be similar so its patient and,physician preference practice preference,some physicians are more familiar have,worked more with no volume and that made,a demand of being there their default,for others in maybe temporal ISM and I,think either way you will benefit the,patients with Amazon high tumor with the,application of one or the other agent so,this is a welcome addition its great to,see that happening and in a space where,we think we can make a big difference,for our patients and responses are just,literally amazing youll never use these,agents in those patients and the very,response these responses can can last,for years and,and again I think like I said the,conundrum is for the practitioner and,how do you choose between the two agents,that are available to you and then the,absence have had to have data theres,really no way to choose between one or,the other other than patient preference,physician preference and other factors,that may be related to the practice we,also met up with dr. Jeffrey Oxnard of,Dana Farber Cancer Institute who,discussed his views on the utility of,liquid biopsies in the field of lung,cancer when Im thinking about who to,send a liquid biopsy on the question I,am thinking for an individual patient is,does their cancer shed DNA we are,looking for DNA that is effectively the,needle in the haystack the haystack,being all the patients germline DNA the,needle being the tumor DNA floating,around there and there are certain,characteristics that are associated with,DNA shed what we found in our studies so,far is the more metastatic sites of,disease they have the more DNA shed if,they have a liver or bone Mets more shed,of DNA versus if this is a lung cancer,patient with small lung nodules,asymptomatic the chance of that being a,shedding cancer with a with a productive,liquid biopsy is smaller and so this is,something we each need to really develop,a sense about as clinicians which is in,which patients is this tending to be a,useful assay and in which patients is,this negative and falsely negative,because there was simply no DNA in the,specimen for us to assess if you think,about a biopsy there is a pathologist,looking at that specimen telling you yep,its got tumor in it this is a good one,for testing but when you do a blood test,there is no one telling you weve got,good tumor DNA this is adequate and so,you have to sort of trust is this a,cancer thats shedding DNA because if,its negative youre falling back on,tissue testing the standard as our,backup test because sensitivity is not,perfect with these assays thats all for,today thank you for watching on class,news network,Im Jena Columbus

Fuqua Research into Action: The Priority Review Voucher

this is Duke University,hi im david ridley i want to tell you,about a paper i wrote with my colleagues,at duke university that paper became law,the law could save a lot of lives its,called the Priority Review voucher,the voucher is intended to encourage,development of treatments for neglected,diseases these diseases are neglected,because drug makers cant make much,money from developing treatments so drug,companies are reluctant to invest,millions of dollars if most of the,people who need the treatments are poor,examples of neglected diseases include,malaria leishmaniasis and dengue,consider malaria there are 200 million,episodes of malaria each year resulting,in a half million deaths most victims,are kids consider leishmaniasis its,transmitted by sand flies,it kills 25,000 people each year it,disfigures even more its victims are,ostracized because it causes severe,scarring and eats away at mouth and nose,tissue considered angee dengue is,transmitted by mosquitoes,it is known as breakbone fever because,of the bone-crushing intensity of the,flu-like symptoms it brings dengue is,found around the world including in,neighboring countries in the Caribbean,recently I was in st. Maarten where,there were warning signs about dengue,there are no treatments for dang,how can we encourage drug companies to,develop treatments for neglected,diseases my colleagues Jeff Moe Henry,Grabowski and I proposed a solution its,a prize the Priority Review voucher if a,drug company develops a treatment for a,neglected disease such as malaria,leishmaniasis or dengue they win a prize,they win a voucher for faster review of,a different drug for example a,cholesterol-lowering drug that could be,a blockbuster and that voucher can be,sold to another company,we think that faster review at the Food,and Drug Administration could be worth,hundreds of millions of dollars,in 2006 the proposal from Jeff Mahendra,Garbowsky and I was published in the,journal Health Affairs under the title,developing drugs for developing,countries the journal Health Affairs,held a press conference at the National,Press Club I presented our proposal,there in this Health Affairs article we,proposed a novel incentive mechanism as,Phil said so the pharmaceutical,companies will have an incentive to,conduct research and development for,drugs for neglected diseases after the,presentation a reporter named Laura,blink horn from congressional quarterly,approached me she told me that Senator,Brownback would like our idea Senator,Brownback a Republican from Kansas,brought in senator sherrod Brown a,Democrat from Ohio they and other,senators like Senator Lieberman made the,Priority Review voucher law in 2007 bill,gates spoke about the Priority Review,voucher at the World Economic Forum in,Davos in 2008 he said under a law signed,by President Bush last year any drug,company that develops a new treatment,for a neglected disease like malaria or,TB can get priority review from the Food,and Drug Administration for another,product theyve made if you develop a,new drug for malaria youre profitable,cholesterol-lowering drug could go on,the market a year earlier this priority,review could be worth hundreds of,millions of dollars,whats the value of voucher we estimate,that a voucher could be worth a couple,hundred million dollars if its applied,to a blockbuster drug there are three,sources of value from the voucher the,first reaching the market earlier and,theres time value of money the second,being on the market for longer because,you launched earlier and have the same,effect of patent expiration date in many,cases and third their competitive,benefits of launching closer to a,competitor or even before a competitor,recently I received a number of calls,about the value of a voucher theres a,company with a drug for a leishmaniasis,they hope that that drug will be,approved this spring thats really all,that company has so the value of the,company is the value of the,leishmaniasis drug which is the value of,the voucher so these bankers wanted my,help,estimating the value of a voucher,because thats the value of the company,and conditional an approval we think the,voucher is worth a couple hundred,million dollars,recently I hosted in my classroom Jean,Seymour Jean is the CEO of nanovirus,ides nano virus side Nano they used,nanotechnology to go after viruses in,side kill them nanovirus side was,pursuing diseases like HIV and flu,because you can make money developing,treatments for HIV and flu now because,the Priority Review voucher theyre,developing a drug called Danny side it,could be the first treatment for dengue,Ive been focused on diseases like,malaria leishmaniasis and dengue but in,fact theres a longer list of diseases,that are eligible for a voucher there 16,from blinding trachoma to yas and in,2012 the list of voucher eligible,diseases was expanded on a trial basis,to rare pediatric diseases another,impact of the voucher is to inspire,similar mechanisms the US Patent and,Trademark Office started a program,called patents for Humanity if you use,your patent for humanitarian ends you,can win a voucher which you can sell for,faster patent review so thanks to a,paper a reporter a senator a CEO we may,soon have new treatments for neglected,diseases like dengue so far it looks,quite promising if youd like to learn,more about the Priority Review voucher,please check out my web page just Google,Priority Review voucher you should see,my webpage and also feel free to contact,me I love talking about this

Categorized in:

Tagged in:

, ,