By Sneha S K
Feb 13 (Reuters) – Disc Medicine said on Friday the U.S. Food and Drug Administration declined to approve its drug to treat a rare genetic disorder.
The drug, 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.
Reuters had exclusively reported last month that FDA reviewers pushed back bitopertin’s review by two weeks on concerns about trial data and its risk for abuse.
The delay was due to the agency’s concerns over whether pain-free time in the sun – a secondary trial goal – was a statistically solid measure of efficacy, or if other biomarker data could justify approval, Reuters had reported.
The company was testing bitopertin as a treatment for erythropoietic protoporphyria, a blood disorder which makes patients extremely sensitive to sunlight.
The condition leads to the buildup of protoporphyrin in the blood, leading to extreme sensitivity to sunlight, which can cause painful skin reactions.
Disc said the agency concluded the two mid-stage trials did not show a clear link between the reduction in patients’ protoporphyrin levels and their tolerance to sunlight.
“The decision is still surprising even with the advance signaling from the media and adds to evidence of internal agency turbulence and a meaningful shift in approval standards for rare-disease therapies,” said Truist analyst Danielle Brill.
The company on Friday said the issue raised by the regulator is “readily addressable” as it expects results from its ongoing late-stage study in the fourth quarter.
Disc plans to respond to the FDA’s rejection letter once the late-stage study is completed and expects an updated FDA decision by mid-2027.
The rejection is not the end of the world for Disc, Brill said, adding the late-stage trial if positive should support full approval.
Shares of the company were flat in after-market trading after closing down 21%.
(Reporting by Sneha S K in Bengaluru; Editing by Vijay Kishore)
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