Drugs

Disc Medicine Says FDA Open to Bitopertin Approval Path

Disc Medicine says it has reached agreement with FDA on a potential regulatory path forward for its investigational therapy bitopertin, with the agency indicating that a successful Phase 3 (APOLLO) trial could support a response to the drug's recent complete response letter and potentially lead to traditional approval for treating erythropoietic protoporphyria (EPP), a rare genetic disorder characterized by severe, painful reactions to sunlight.

The company says it remains on track to submit its complete response letter package by the end of the year following release of Phase 3 data later this year.

The APOLLO study is a global, randomized, double-blind, placebo-controlled Phase 3 trial evaluating bitopertin in patients aged 12 years and older with EPP and the related disorder X-linked protoporphyria. The trial’s co-primary endpoints measure both clinical benefit and disease biology. Investigators are evaluating patients' average monthly time spent in sunlight without pain during daytime hours over the final month of a six-month treatment period, as well as changes in whole-blood metal-free protoporphyrin IX (PPIX), the toxic metabolite that accumulates in patients with EPP.

The update follows a setback earlier this year when FDA issued a Complete Response Letter that rejected the company’s submission seeking accelerated approval and as part of the Commissioner’s National Priority Voucher pilot program. The letter acknowledged that there was sufficient evidence that bitopertin significantly lowers whole blood metal-free PPIX, but FDA concluded that the trials did not show evidence of association between percent change in PPIX and sunlight exposure-based endpoints, as measured in the trials, despite the strong mechanistic and biological plausibility supporting the use of the PPIX biomarker in protoporphyria, according to the company.

Bitopertin is described as an oral inhibitor of glycine transporter 1, a protein involved in heme biosynthesis within developing red blood cells. By modulating heme production, the therapy is designed to reduce accumulation of protoporphyrin IX, the molecule responsible for the painful phototoxic reactions experienced by patients with EPP.

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