BioMarin: The DMD Drug and the FDA Committee Options

Today, the FDA advisory panel will discuss BioMarin (BMRN) product drisapersen for Duchenne muscular dystrophy based on documents released to the committee by the FDA. The FDA said, “The benefit of drisapersen in exon 51 skip amenable patients is inconclusive at this time. Therefore, the benefit-risk assessments were not made.”

Analysts interpreted the FDA statement as a condemnation of the drug, which does not really tell what the FDA meant to say and why it said it. Some analysts stated that the FDA has signaled that there is no reason to approve BioMarin’s Duchenne muscular dystrophy drug drisapersen and others said, that the FDA statement was clear that drisapersen should not be approved”.

Is that right?

We have reasons to believe that such interpretations are not accurate. The FDA did not say anything that we didn’t know since the phase 3 trial results have demonstrated drisapersen did not meet the endpoints of the trials. The FDA did not say more than what the co-developers of drisapersen Prosensa Holding N.V. and GlaxoSmithKline said after the announcement of Phase 3 trial results.  As a matter of fact, what the FDA said is what led GlaxoSmithKline to abandon ship after the trial results were revealed.

Prohost Comments

Our interpretation is totally different than the above interpretations. We believe the FDA was telling the special Committee, “Knowing in fact that the product has failed the traditionaltests, are you ready to approve it 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 specific treatment on the market, which could provide any kind of dystrophin, which is what these patients really need?”

What made us believe in our scenario is that it was the FDA itself that had called on the developers of   drisapersen and eteplirsen to come forward and file NDA for approval. At the time, the invited firms were Prosensa, which was acquired later on by BioMarin, and Sarepta, which is the producer of eteplirsen.  Drisapersen and eteplirsen are the only products that yield dystrophin, regardless of its kind.

In other words, the FDA, in our opinion said it does not know what the drugs can do when used for  long. The agency said it cannot assess the risk and it leaves it up to the committee to decide whether the side effects of eteplirsen might be better or worse than leaving the patients face the well-recognized horrible outcome of the untreated disease without any specific treatment.

We believe that what the committee would decide will impact both BioMarin and Sarepta drugs. The most damage of course will be in SRPT.

We don’t really know what the committee will decide upon, or how it would voteIt all depends on whether the committee would abide by the usual criteria used in approving drugs, or will listen with compassion to the patients and their families’ observations and hopes, considering them as important as the automatic way of approving drugs regardless of the degree of disease severity or the products’ actions and side effects.

We are anxiously waiting for the verdict.

Leave a Reply