Disc Medicine eyes standard US approval for drug after FDA denies fast-track nod

By Siddhi Mahatole

Feb 17 (Reuters) – 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.

Reuters had exclusively reported last month that FDA reviewers pushed back review of bitopertin by two weeks on concerns about trial data and the drug’s risk for abuse.

Shares of the company were up nearly 8% in ‌early ​trading.

Disc was testing bitopertin as a ⁠treatment for erythropoietic protoporphyria, a ⁠blood disorder that makes patients extremely sensitive to sunlight.

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.

Most companies then 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,” CEO John Quisel said on a call with analysts.

Quisel said the company plans ⁠to refile its application following the FDA’s complete response letter, noting that the agency typically has a six-month review goal for such applications. ⁠He expects a potential decision ‌on traditional approval around mid-2027.

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.

Quisel 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.

(Reporting by Siddhi Mahatole in Bengaluru; Editing by Leroy Leo ​and Shailesh Kuber)