In our yesterday’s article asking about how the FDA advisory committee has to vote regarding Biomarin product Kyndrisa (drisapersen) for Duchenne muscular dystrophy. We got the answer yesterday during the meeting that took a much longer time than committees’ meetings usually take. At the end of the long session that included several patients’ and patients’ relatives’ testimonies, the FDA Advisory Committee was incapable of voting for or against the approval of the drug. All the members did was restating the well-recognized inconsistencies in the late phase clinical trial and all they added to what we already knew was that the inconsistencies in the Phase 3 trial have weakened Biomarin’s argument that DMD patients would do better on drisapersen than on the conventional non-specific treatments given to DMD patients to date.
Everybody knew about the inconsistencies, but we really did not know how much it would weaken Biomarin’s argument. As a matter of fact, we still don’t know and we believe that the members of the Committee do not not know, that’s why they preferred not to vote in favor of approval or rejection, leaving that decision for the FDA to take care of.
Yesterday we wrote: “We believe the FDA was telling the special Committee, “Knowing in fact that the product has failed the traditional tests, are you ready to approve the drug based on the fact that the disease is torturing children and killing them? Are you ready to approve it considering the fact that there is no other specific treatment on the market, which could provide any kind of dystrophin that these patients really need?”
We believe that the Committee came to the same conclusion about what the agency wanted them to vote for, but couldn’t.
We still do not know what the FDA decision might be. All we know is that a rejection of drisapersen based on Tuesday’s meeting will make it very difficult for the FDA to approve Sarepta drug eteplirsen. First and foremost, all the inconsistencies in the Biomarin trial results came in the phase 3 trial. Needless to remind that Sarepta has yet to finish its phase 3 trial, which means that the results of the larger trial are non-existent. In addition, Sarepta’s eteplirsen is similar to Biomarin’s product drisapersen and skips the same exon 51, as does drisapersen.
On the other side of the coin, the approval of Biomarin drug, might facilitate the approval of Sarepta drug eteplirsen, which could also beat biomarin drug’s sales in case the advisory Committee and the FDA confirm the Sarepta’s claims that its drug is safer than Biomarin’s drug.
Read about gene-editing in Prohost Letter #387. Also, in the upcoming issue #388, which will be posted next Sunday, you might be able to better assess the value of Sarepta and the value of BioMarin Program that specializes in gene-editing.