Disc Medicine to Pursue Standard FDA Approval After Fast-Track Rejection for Bitopertin

Disc Medicine said on Tuesday it will pursue a traditional U.S. approval pathway for its rare disease drug after the Food and Drug Administration declined to approve the treatment under a new fast-track review program. Shares of the company were up nearly 15%.

Bitopertin was being reviewed under the FDA's "national priority voucher" program, which fast-tracks the process to one to two months from the typical 10-12 months. The drug was being reviewed under the accelerated approval pathway, in which the FDA evaluates whether changes in a biomarker is reasonably likely to predict clinical benefit.

FDA reviewers pushed back review of bitopertin by two weeks on concerns about trial data and the drug's risk for abuse. Disc was testing bitopertin as a treatment for erythropoietic protoporphyria, a blood disorder that makes patients extremely sensitive to sunlight.

The CEO said he expects a potential decision on traditional approval around mid-2027. Most companies seek traditional approval from the agency, which typically leads to higher use and better insurance coverage.

While the policy debate around accelerated approval has been going on for years, "recent decisions have indicated an increasingly stringent approach to this question of when an accelerated approval is appropriate," the CEO said.

The company expects late-stage data on the drug in the fourth quarter this year, but modifying the trial "is not on the menu right now," he added.

The CEO said that "there was no obvious skepticism apparent in information requests as we went along" and added that the first indication of potential challenges came from media leaks, which he said were unexpected.