Amryt Pharma is filing a formal dispute resolution request (FDRR) with the FDA after receiving a CRL that delays the production of its drug for a rare skin disorder.
Amryt Pharma is filing a formal dispute resolution request (FDRR) with the U.S. Food and Drug Administration after receiving a complete response letter that effectively delays production plans for its proposed drug for a rare skin disorder.
The FDA granted Priority Review status to Oleogel-S10 (Filsuvez) in early June for the treatment of epidermolysis bullosa, a rare, genetic skin disease characterized by having extremely sensitive skin that tears and blisters even from minor friction. If the NDA is approved, Amryt said it would apply for a priority review voucher. There is no approved treatment for the disorder as of this writing.
However, in February, the regulator issued a CRL saying that it cannot allow the drug to move forward in its present form. Amryt was asked to submit supporting evidence of its drug candidate’s efficacy and capabilities in addressing EB. The company had committed to providing everything the FDA needed to put the concern to rest.
Oleogel-S10 showed much promise following positive outcomes from its Phase III EASE study, which involved 223 participants, including 156 pediatric patients, across 58 sites in 28 countries. The three-month double-blind trial was the largest ever global study on patients living with EB.
Its latest decision to pursue an FDRR for its new drug application for Oleogel-S10 follows a Type A meeting with the FDA to discuss concerns raised in the February CRL. The FDR will be filed with the Center for Drug Evaluation and Research, an evaluating body within the FDA, by which NDA applicants seek to resolve issues not fixed at the division level.
“Filsuvez was, this week, approved in Europe by the European Commission, and we continue to believe that the strong body of data from the EASE trial, which was the largest ever Phase 3 trial conducted in EB, supports FDA approval of this vital therapy for EB patients with a high unmet medical need. The Formal Dispute Resolution process provides an opportunity to raise our scientific disagreement within CDER,” Dr. Joe Wiley, CEO of Amryt Pharma, commented in a statement.
The European Commission has approved Filsuvez for the treatment of partial-thickness wounds associated with dystrophic and junctional EB in patients six months and older. In April, the European Medicine Agency’s Committee for Medicinal Products for Human Use recommended the drug’s approval in the European Union.
“The CHMP recommendation for approval of Filsuvez® in Europe is the most significant milestone in Amryt’s history and represents a major positive development for European patients that suffer from this debilitating condition. This is the culmination of years of hard work from all the Amryt team. I’d like to formally thank both them and all the patients, caregivers and physicians for their commitment and efforts in getting us to this point,” Dr. Wiley noted in a separate statement.
