STAT+: FDA rejects rare disease therapy from Disc Medicine, early recipient of commissioner\u2019s voucher\u00a0
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WASHINGTON \u2014 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.\u00a0<\/p>\n
It\u2019s the first experimental drug to go through FDA Commissioner Marty Makary\u2019s new program to fast-track drug reviews. The agency previously approved<\/a> a generic antibiotic through the new drug review program in December.<\/p>\n The FDA, in its letter rejecting bitopertin<\/a>, cited \u201cuncertainties\u201d about the correlation between the blood-based biomarker used as the efficacy goal in Disc\u2019s 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.\u00a0<\/p>\n Continue to STAT+ to read the full story\u2026<\/a><\/p>\n<\/div>\n