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FDA grants ezutromid rare pediatric disease status for Duchenne muscular dystrophy
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The FDA has granted a rare pediatric disease designation to Summit Therapeutics for ezutromid in the treatment of Duchenne muscular dystrophy, according to a company press release.
The Rare Pediatric Disease Voucher Program, established by the FDA to foster development of new drugs for the prevention and treatment of rare pediatric diseases, will allow Summit — based in Oxford, United Kingdom — to request a priority review of a subsequent marketing application for a different product. The announcement follows the FDA granting fast track and orphan drug designations for ezutromid.
“Rare Pediatric Disease designation builds upon the Fast Track and Orphan Drug designations which the FDA has already awarded to ezutromid, recognizing a significant unmet medical need in the treatment of [Duchenne muscular dystrophy (DMD)],” Summit CEO Glyn Edwards said in the press release. “We plan to leverage these regulatory advantages in the continued clinical development of ezutromid, which is currently in a Phase 2 clinical trial called PhaseOut DMD, to bring ezutromid to patients in need as quickly as possible.”
Duchenne muscular dystrophy, a genetic disorder characterized by muscle deterioration and weakness, is the most common type of muscular dystrophy. It is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptoms typically emerge between the ages of 3 and 5 years, worsening over time, with patients often succumbing to the disease in their 20s or 30s. Although Duchenne muscular dystrophy primarily affects boys, it can in rare cases also affect girls. It occurs in about one out of every 3,600 male infants worldwide.
According to Summit, ezutromid, an orally administered utrophin modulator, has “potential as a disease-modifying treatment for all patients” with DMD, “regardless of their underlying dystrophin gene mutation. The company, noting that utrophin is functionally and structurally similar to dystrophin, pointed to preclinical studies suggesting that a continued expression of utrophin has a positive effect on muscle performance.
Earlier this month, the FDA granted accelerated approval to Exondys 51 (eteplirsen, Sarepta Therapeutics) for treatment of DMD. It is the first drug approved in the United States to treat patients with the rare disease. The eteplirsen injection is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects 13% of patients with Duchenne muscular dystrophy.
Under the provisions of the accelerated approval process, the FDA is requiring Sarepta Therapeutics to conduct a clinical trial to confirm the drug’s clinical benefit, specifically determining whether eteplirsen improves motor function in patients with the disease who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. If the trial fails to verify clinical benefit, the FDA can begin the process to revoke its approval.
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