The Company’s lead Duchenne Muscular Dystrophy (DMD) product candidate, Eteplirsen, and is an antisense phosphorodiamidate morpholino oligomer (PMO) therapeutic in Phase III clinical development for the treatment of individuals with DMD having an error in the gene coding for dystrophin that is amenable to skipping exon 51.
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Exondys 51 is approved for people with a specific mutation of the dystrophin gene, affecting some 13 percent of people with the disorder.
Woodcock acknowledged the concerns in a statement, emphasizing that the drug would still have to undergo more trials to prove its efficacy. The drug acts on a protein called dystrophin, which plays a role in the growth of muscle fibers.
Research on a controversial new drug to treat a rare form of muscular dystrophy was in-part conducted in Iowa City. DMD patients are typically wheelchair-bound by their early teens and often die in their 20s or 30s. As the disease progresses, life-threatening heart and respiratory conditions can occur. Executives said they anticipated health insurers would reimburse most patients for the cost, which they described as being in the middle of the typical price range for rare genetic disease treatments.
“I think this is a collaborative effort that shows the FDA, companies and the patient community can work together toward a single goal, and that is improving the lives of patients”, said Furlong, founder and president of Parent Project Muscular Dystrophy, a nonprofit that helped fund travel and other expenses related to the study.
Approval was based on the surrogate endpoint of increased dystrophin in skeletal muscle in patients who received the drug, according to the FDA.
Monday’s decision requires Sarepta to conduct a two-year study to confirm that its treatment can improve motor function in Duchenne patients losing the ability to stand and walk. Eteplirsen is created to enable RNA to skip over the part of the DNA with a problematic mutation, enabling a functional (though shorter) dystrophin protein to be produced. If the trial fails to verify clinical benefit, the FDA can begin the process to revoke its approval.
The most common side effects reported by participants taking Exondys 51 in the clinical trials were balance disorder and vomiting.
The FDA granted eteplirsen fast track designation, which facilitates the development and review of drugs meant to treat serious conditions and have the potential to address an unmet medical need.
The analyst upgraded the stock to buy with a fair value of $65 and noted that Sarepta had also received a pediatric review voucher, limiting potential near-term dilution. Advocacy groups – composed largely of parents of the children – have been major supporters of DMD developers and clinical trials seeking to test disease-modifying drugs. It was also granted priority review and orphan drug designation. In remarks created to appease the crowd, Janet Woodcock, who heads the agency division that approves drugs, said that “It’s possible to reach different conclusions based on the data presented today …”
Sarepta Therapeutics, Inc.is a biopharmaceutical company.
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The agency gave itself extra time to decide.
