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Sarepta Therapeutics had to deal with a market share drop after the Food and Drug Administration rejected Raxone, the rival Duchenne muscular atrophy drug.
Experts say that Sarepta’s chances could be dimmed by the recent rejection.
Duchenne muscular atrophy is a rare genetic disorder that causes a gradual deterioration of muscles and later impacts all body functions. It only affects little boys, and it results in death at the age of 20 or 30.
One in 3,500 boys gets the disease, and most of them are losing all motor functions until they reach adulthood. The condition has no cure.
FDA Suite of Rejections
The families and patient advocacy groups put a lot of pressure on FDA to approve the new drugs. However, the agency did not find hard evidence of clinical benefits in the medicines that were submitted for approval so far.
FDA recently rejected Kyndrisa, developed by BioMarin Pharmaceuticals, Ataluren, created by PTC Therapeutics, and Raxone from Santhera. All the three drugs presented a low rate of positive results.
Raxone, the latest drug to be rejected, had forwarded a Phase 3 study to FDA. The agency requested a new trial, which will take another three years. Santhera will start as soon as possible. However, the results will come only in 2019.
Eteplisen, the experimental drug developed by Sarepta, had the same difficult road of approval. Earlier this year, FDA rejected a small trial testing based on 12 DMD patients. The results were a 0.9% increase in dystrophin production. FDA requested muscle biopsy data for patients that are on the 48th week of treatment.
Sarepta Still Has A Chance
Raxone’s rejection in Phase 3 of the trial could be a sign that the FDA will not readily accept Eteplisen either.
Raxone was created to stimulate mitochondria, which would improve lung function for Duchenne muscular atrophy patients.
Eteplisen uses another strategy. It tries to act directly on the dystrophin production. Over the course of four years, the drug showed improvements in patients’ history data.
As a consequence, analysts recommend Sarepta shares as the Raxone’s rejection will probably have no impact on Eteplisen as the drug works entirely different.
Eteplisen will probably work only on 1,300-1,900 patients in the state. Roxanne will have a larger spectrum of applications, including Leber’s hereditary optic neuropathy, Freidrich’s ataxia, or Alzheimer.
The political pressure, patient advocacy and the positive results on the disease may all act in the favor of Sarepta. Other market analysts think that, in fact, FDA will continue to reject all DMD medicines until they have their high standards attained.
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