Zevra to face FDA adcomm in August over previously rejected rare disease drug

The FDA’s recently created Genetic Metabolic Diseases Advisory Committee will meet on Aug. 2 to review Zevra Therapeutics’ arimoclomol as a treatment for the rare and fatal genetic disorder known as Niemann-Pick disease type C.

The drug was rejected once before, in 2021, and the FDA’s decision to convene an advisory committee was greeted as good news by investors, who sent Zevra shares $ZVRA up more than 15% on Tuesday. The new review of the drug has already been delayed once, when in March Zevra said the FDA postponed its decision by three months and had the Florida-based company submit additional data. The FDA didn’t disclose what questions the committee would address.

Arimoclomol is a small molecule drug delivered three times a day, with the intent of slowing the rare disease’s progression. It has been…
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