Intercept’s Liver Drug Wins FDA Approval To Treat Rare Disease

June 13, 2016

The FDA ended a busy week by giving a thumbs-up late Friday to obeticholic acid as a treatment for primary biliary cirrhosis, a rare disease in which a patient’s own immune system attacks the liver.

Obeticholic acid, to be marketed under the brand Ocaliva, is being developed by Intercept Pharmaceuticals (NASDAQ: ICPT) of New York. Approval was all but guaranteed by a unanimous vote of support from a group of outside advisors to the FDA in April.

The approval arrived late Friday night, in sharp contrast to the FDA’s announcement Wednesday that it would delay judgment on eteplirsen, an experimental treatment for Duchenne muscular dystrophy from Cambridge, MA-based Sarepta Therapeutics (NASDAQ: SRPT).

The Duchenne situation has taken on political baggage. Two senators, spurred by vocal patient groups that hope to sway the agency’s final decision, have asked the FDA to overrule its scientists and advisors who have recommended the drug’s rejection.

For Intercept, approval for obeticholic acid provides fresh momentum. The company must now convince to doctors who specialize in liver disease that the drug, despite a few lingering questions from the advisors last month, is worth prescribing for PBC. The data from the clinical trials have not shown that the drug actually helps people live longer or makes their lives better.

Read more at the source.

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