A drug developer with no products on the market, rose to its highest value in five months after saying it plans to submit its Duchenne muscular dystrophy treatment for approval this year.
On Nov. 12, Sarepta shares fell the most in 16 years after the Food and Drug Administration had called an application to sell the experimental eteplirsen compound “premature.” The FDA has provided new guidance that will enable the submission of the drug by the end of 2014, the Cambridge, Massachusetts-based company said today in a statement.
Sarepta will rely on data from an existing phase II study out of three phases typically required for approval. Sarepta is working with the FDA to help the agency gain better understanding of a measurement of a protein responsible for muscle movement the study relies on to provide a reasonable assumption of the drug’s benefit, Sarepta said.
In a New England Journal of Medicine paper last year, Sarepta’s competitor Prosensa (with partner GSK) reported in a similar-sized study that in 10 out of 12 patients, the percent of fibers expressing dystrophin following treatment with their exon skipper PRO051 was between 60 and 100%. Yes, 60-100% and nost just 34-52% as in Sarepta's case.
What is more, the immunofluorescence intensity and Western blot data in the Prosensa study showed that those fibers expressing dystrophin did so at much reduced levels compared to healthy muscle, on the order of 5-20% (overall bulk levels). In other words, it is quite likely that the ~50% of fibers that express dystrophin in the Sarepta study together express at most 3-10% of normal dystrophin, well short of what is expected to be therapeutic. It is also possible, however, that Sarepta’s assay is very sensitive such that even fibers with just 1% or so of wild-type signals were counted in.
Given that stating the percent fibers expressing any dystrophin is almost meaningless, you have to wonder why Sarepta has not presented the data.
FRAUDSTER - SRPT CEO not Highlighting the fact that the FDA is asking for now new data, and that is a risk. 168 Weeks biopsy cannot be performed as only 2 boys’ increases levels of dystrophin muscles, now that is missing and what it causes muscular decline quickly and lost the ability to walk - SRPT is not disclosing. .
Probably you did not understand - RESULTS FROM eteplirsen following same path of drisapersen phase 2 study. Management of SRPT realized it and they Hide data from public as easy to conclude even for layman that both drug acts in same way. Since Prosensa /GSK’s Phase III clinical study of drisapersen FAILED, and now GSK put this to Garbage, SRPT is trying to find way out somehow to put pressure on FDA to get the ball rolling.
At the same time PTC Therapeutics is back with ROCHE support and probably their Ataluren is in late stage clinical development Phase 3 approval would be before SRPT so SRPT is now playing LAST GAME under the name of FDA approval. They know very well after spending so much of public fund to run more trials the results would be the same.