This may be the wrong conclusion, but whether it is or isnt is knowable only by management, fda and god at this point, the analyst adds. The fda s latest briefing on eteplirsen doesnt bode well for the drugs chances of approval this year. Sareptas stock soars premarket as fda news fuels hope for. Vice president, regulatory affairs hurley consulting associates ltd.
Fda lashes back at sareptas arguments 20160427 fdanews. Senators and members of the house signed letters supporting an accelerated approval. Rbc capital markets analyst matthew eckler says consistent highquality trial results should make. Fda urged to approve sarepta dmd drug by dozens of medical. Sarepta therapeutics srpt gains following posting of fda may 18 meeting briefing docs article stock quotes 1 comments 0 free breaking news alerts from. Treatmentrelated andor moderate or severe teaes potentially indicative. Sarepta included additional six minute walk test and loss of ambulation data, the company said. Please refer to your new drug application nda submitted under section 505b of the federal food, drug, and cosmetic act for golodirsen.
Srpts eteplirsen drug for the treatment of duchenne muscular dystrophy dmd is a. No storm for sarepta, but has the fda created its own tempest. The fda gave accelerated approval to the first drug for the crippling disease duchenne muscular dystrophy, from sarepta, after sharp disagreements within the agency. The fda attacked the dystrophin data, noting that after 3. Sarepta drug future hinges on fda panel the boston globe. Sarepta and a group of advocates for duchenne therapies will use the delay to hone their presentations to the advisory panel, including the companys rebuttal of a briefing document issued by the. Sarepta slammed by fda, stock is now intriguing barrons. Food and drug administration fda has accepted its new drug application nda for eteplirsen to treat duchenne muscular dystrophy dmd. The fda s decisons about exondys 51 and vyondys 53, both developed by sarepta therapeutics, offer a rare glimpse into the world of drug regulation. Mar 22, 2016 fda urged to approve sarepta dmd drug by dozens of medical experts.
Opening the lid on sareptas drug approvals in the pipeline. Please refer to your new drug application nda submitted under section 505b of the. Brieffda staff maintains negative outlook on sareptas. Eteplirsen nda 206488 pcnsd advisory committee meeting briefing document figure 19. How to become a member of an advisory committee, common questions, and the laws. Behind the sarepta drug approval was intense fda bickering. Both drugs are designed to skip over a genetic mutation in the dna sequence for dmd. Patrick omalley executive director, regulatory affairs 215 first street, suite 415 cambridge, ma 02142 dear mr. Fdaeditscomplete round6 03aug16eteplirsen pi to sarepta. Biotech sarepta therapeutics stock price was cut in half friday after the fda released highly critical briefing documents for an advisory committee evaluating sarepta s muscular dystrophy drug.
Duchenne is a rare genetic disease that causes progressive muscle degeneration because of an absence or. The patient communities and medical experts in the field are starting to put the. Apr 21, 2016 fda staff says it does not agree with sareptas characterization of inaccuracies in the initial fda briefing document tone of fda staff briefing documents on sareptas dmd drug seem. Sep 24, 2016 drugmaker sarepta plans to use the same approach now to target other mutations related to the disease, and to use those study results to comply with the fda s requirement for more and better studies.
Race for the first drug approval by fda for treating. Patient advocates descend on fda panel as sarepta squares off. But in a briefing document released in advance of a key gathering monday of an fda advisory committee, agency staffers wrote that the clinical data submitted by sarepta. Yesterday, the food and drug administration made history, approving a drug to treat duchenne muscular dystrophy that works by targeting the. And fda approval, because you cant bring up that company without immediately starting a regulatory affairs argument. Cumulative loss of ambulation over 3 years in eteplirsentreated patients. Eteplirsen is an exonskipping drug that targets a section of dna called exon 51, and may help up to percent of duchenne muscular dystrophy dmd.
Muscular dystrophy drug advocates plan to pack crucial. Those are just a few of the issues the fda had with sarepta therapeuticss recent drug application, according to new documents released. Drugmaker sarepta plans to use the same approach now to target other mutations related to the disease, and to use those study results to comply with the fda s requirement for more and better studies. Golodirsen works similarly to exondys 51, the first drug approved by the fda for the treatment of dmd. Sarepta therapeutics said the fda wouldnt issue a decision on the companys muscular dystrophy drug by thursdays deadline, raising expectations that the agency may approve a treatment that. Sarepta says fda asks for more data for its musclewasting. Page 1 of 2 100 corporate court south plainfield, nj 07080 908.
Fda urged to approve sarepta dmd drug by dozens of medical experts. Sarepta wins controversial fda approval for first dmd drug raps. The fda has delayed its decision on sarepta therapeutics duchenne muscular dystrophy candidate eteplirsen, citing the companys submission of fouryear efficacy data as a major amendment to the nda. Jan 15, 2016 earlier, the fda released briefing documents for sareptas drug candidate, called eteplirsen, ahead of a meeting of its advisory committee next week. Fda documents show safety concerns over a sarepta drug despite receiving approval, new documents show that the fda wasnt entirely convinced about the. Sarepta therapeutics receives notification of pdufa. But in a briefing document released in advance of a key gathering monday of an fda advisory committee, agency staffers wrote that the clinical data submitted by sarepta overall did not provide. Sarepta therapeutics srpt shares plunged 55% after the fda raised concerns related to the duchenne muscular dystrophy candidate, eteplirsen, in its briefing documents. Eteplirsen nda 206488 pcnsd advisory committee meeting briefing document table 24. Pcnsd advisory committee meeting briefing document. Food and drug administration 10903 new hampshire avenue silver spring, md 20993 1888info fda 18884636332 contact fda.
Sep 15, 2016 sarepta therapeutics srpt got a big bounce yesterday on reports that one of its biggest critics inside the fda had left the regulatory agency. In briefing documents posted ahead of the january meeting, the fda asked for more evidence to solidify. April 21 reuters fda staff says it does not agree with sarepta s characterization of inaccuracies in the initial fda briefing document. A clinical investigation is essential to the approval if the agency could not have approved the application. Sarepta is seeking accelerated approval for eteplirsen for patients with dmd who have a confirmed mutation of the dystrophin gene amenable. In the first study, intended to satisfy victoza pmr. Sarepta says that it has received final meeting minutes from the fda, and that its statements today are based on those written minutes. Patient deaths, testing concerns and weak arguments. Its approval came with a condition requiring sarepta to carry out a postapproval clinical trial to confirm the drugs effectiveness. Earlier, the fda released briefing documents for sarepta s drug candidate, called eteplirsen, ahead of a meeting of its advisory committee next week. Fda documents show safety concerns over a sarepta drug. Fda delays sareptas pdufa date of dmd candidate february 12, 2016 the fda has delayed its decision on sarepta therapeutics duchenne muscular dystrophy candidate eteplirsen, citing the companys submission of fouryear efficacy data as a major amendment to the nda. Sep 19, 2016 bowing to pressure from patient advocates, the u. Toward the end of trading thursday, the dow traded down 0.
Duchenne moms say fda documents widen the divide between. Fda delays sareptas pdufa date of dmd candidate 20160212. The fdas decisons about exondys 51 and vyondys 53, both developed by sarepta therapeutics, offer a rare glimpse into the world of drug regulation. Apr 22, 2016 sarepta, though, has some major league political supporters. Sarepta therapeutics shares tank as hopes for new drug. Ataluren briefing document food and drug administration. New fda documents shed light on sarepta drug rejection boston. Sarepta management thinks that their detailed discussions with the fda went well. Sep 14, 2016 suntrust said the logical decision for the fda would be to deny approval for sarepta therapeutics inc nasdaq. Sarepta stock plunges as fda questions muscular dystrophy.
Food and drug administration on monday approved a treatment for duchenne muscular dystrophy even though an outside panel of experts and the. However, the fda s briefing documents seem to indicate otherwise, meaning that its looking like sarepta will ultimately have to complete a latestage study to. Exondys 51 eteplirsen is an antisense oligonucleotide indicated for the treatment of duchenne muscular dystrophy dmd in patients who have a confirmed mutation of the dmd gene that is amenable to exon 51 skipping. Sarepta tanks, eteplirsen briefing document raises concerns. The fda notes in its briefing documents that the data sarepta collected to show that dystrophin levels were increasing in the trial patients compared just four patients who received the drug to. Code of federal regulations, title 21, food and drugs, ces, pt.
Fda briefing document peripheral and central nervous system drugs advisory committee meeting. Sep 19, 2016 there are several striking things about sareptas fda approval this week for its controversial and longdelayed dmd drug, but just how it gained approval from the u. Sarepta hit with call to drop duchenne drug costs thestreet. Fda snubs sarepta s exon 53targeting dystrophy drug. Jan 21, 2016 the fda notes in its briefing documents that the data sarepta collected to show that dystrophin levels were increasing in the trial patients compared just four patients who received the drug to. Heres why sarepta therapeutics is getting crushed today. Bowing to pressure, fda approves sareptas duchenne drug. Eteplirsen nda 206488 pcnsd advisory committee meeting briefing document 3 6. Fda snow delay adds to woes for sareptas muscular dystrophy med. Clinical studies contributing to pharmacodynamic endpoints and clinical. Sarepta plummets as fda slams muscular dystrophy drug. Fda s rebuff of sarepta s new antisense drug leaves the duchenne community perplexed. Breakfast technical briefing on biotech stocks sarepta. This highly visible public meeting with the agency is a critically important activity to the companya positive fda advisory committee meeting sets the stage for a successful launch of the companys product.
Why sarepta therapeutics shares are skyrocketing 25%. The fda uses committees and panels to obtain independent expert advice on scientific, technical, and policy matters. Those are just a few of the issues the fda had with sarepta. Sarepta to seek fda approval for second dmd treatment biospace.
Brieffda staff maintains negative outlook on sareptas dmd. Sarepta wins fda nod for embattled dmd drug but with a. Preparing for an fda advisory committee meeting mddi online. Sarepta therapeutics srpt gains following posting of fda. Sarepta therapeutics receives notification of pdufa extension.
Sarepta announces posting of briefing information for fda. Approval of victoza included 2 postmarketing requirements pmrs to evaluate the effects of liraglutide on proliferative ccell lesions in mice. Why sarepta therapeutics shares are skyrocketing 25% today the departure of a critic of its duchenne muscular dystrophy drug from the fda is sparking optimism that. Now that fda has rejected ptcs translarna and biomarins drisapersen, sarepta s eteplirsen becomes the only choice to be approved by fda as the first drug for treating dmd.
Oct 09, 2017 today, some analysts are offering their take on what might be in store for sarepta and at the fda. Fda delays sareptas pdufa date of dmd candidate 201602. Doubts that sarepta would ever reach this milestone emerged in late january, when the fda released briefing documents ahead of a. The us food and drug administration fda on monday approved sarepta therapeutics first drug to treat patients with duchenne muscular. The accelerated approval of exondys 51 was based on the surrogate endpoint of increase in dystrophin in skeletal muscle observed. Sarepta and a group of advocates for duchenne therapies will use the delay to hone their presentations to the advisory panel, including the companys rebuttal of a briefing. Sarepta announces posting of briefing information for fda panel. Sep 19, 2016 in the approval of sarepta s drug, the fda s commissioner, dr. Cambridge, massachusetts headquartered sarepta therapeutics inc. Mar 12, 2018 sarepta anticipates filing for approval of golodirsen by the end of the year. The agency made it clear that it doesnt agree with sarepta s characterization of inaccuracies in briefing documents the fda released prior to a canceled jan. Food and drug administration has requested for additional data from an ongoing study for its musclewasting treatment as the agency decides whether to approve.
These talks, though, went into greater detail than the briefing documents that had been supplied earlier. The candidates pdufa date has been pushed to may 26. Strategies for preparing for meetings with fda susan m. New fda documents shed light on sarepta drug rejection. Exondys 51 skips exon 51 and golodirsen is designed to skip exon 53 of the dmd gene. Sareptas eteplirsen approved after contentious internal fda debate. Fda s assessment as delineated in their briefing book do not look promising.
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