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FDA Rejects Promising Drug Despite Priority Voucher
14 Feb
Summary
- FDA rejected Disc Medicine's rare disease drug bitopertin.
- Drug had received a voucher for accelerated review.
- Rejection raises concerns about FDA's regulatory consistency.
Disc Medicine Inc. has experienced the rejection of its experimental compound, bitopertin, by the US Food and Drug Administration (FDA). This drug was intended for erythropoietic protoporphyria, a rare disorder causing severe light sensitivity.
Just four months prior, bitopertin was selected as one of the first nine medicines to receive a Commissioner's National Priority Voucher. This initiative aims to expedite reviews for treatments addressing national health priorities, typically within one to two months.
However, the FDA did not issue a decision within that timeframe. Instead, the agency rejected the application outright, citing a need for more trial data and questioning the study design and clinical significance. This situation, alongside Moderna Inc.'s mRNA flu vaccine facing similar scrutiny, has intensified concerns about the FDA's growing unpredictability.
Analysts suggest the voucher program's value is now under scrutiny, and the overall process for new medical product development faces questions. Disc Medicine's shares fell 22% following the FDA's decision, with the company stating the issues are "readily addressable" and a new study is underway.
