Leerink’s Joseph Schwartz and Dae Gon Ha aren’t buying the bullish argument that a delayed FDA decision on Sarepta Therapeutics’ (SRPT) Duchenne muscular dystrophy drug is good news.
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Undeniably, there’s a significant need for new DMD treatments; but the FDA officially rejected another DMD drug that works similarly earlier this year, and that decision was based on a trial that involved more patients.
Sarepta Therapeutics now sits at a #3 (Hold) on the Zacks Rank. Sarepta is also developing therapeutics for the treatment of rare, infectious and other diseases. It affects roughly one in every 3,500 boys world-wide.
Currently, Sarepta is developing multiple, steric-blocking, antisense oligonucleotides, created to restore protein production in a rare and fatal genetic disorder, DMD. Eteplirsen uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. The DMD patient population is small, and eteplirsen only works in about 13% of them. “For a disease like Duchenne muscular dystrophy and other diseases that are highly debilitating and nearly certainly fatal, we hope you will employ these flexibilities and considerations for the maximum benefit of patients who have no other alternative”, the senators wrote in their letter. Promoting the synthesis of a shorter dystrophin protein is meant to slow the decline of ambulation and mobility seen in DMD patients. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure.
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Federal health regulators will take more time to review a highly-contested drug for muscular dystrophy that has become a flashpoint in the debate over patient access to experimental medicine. We encourage investors and potential investors to consult our website regularly for important information about us.
