STAT+: FDA rejects rare disease therapy from Disc Medicine, early recipient of commissioner’s voucher 

WASHINGTON — The Food and Drug Administration on Friday rejected bitopertin, a therapy developed by Disc Medicine to treat the rare blood disorder porphyria, which makes patients extremely sensitive to sunlight. 

It’s the first experimental drug to go through FDA Commissioner Marty Makary’s new program to fast-track drug reviews. The agency previously approved a generic antibiotic through the new drug review program in December.

The FDA, in its letter rejecting bitopertin, cited “uncertainties” about the correlation between the blood-based biomarker used as the efficacy goal in Disc’s clinical trials and clinical benefit for patients. The agency posted the rejection letter on its website Friday afternoon, sending Disc shares down 31% to $49 in afternoon trading. 

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