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 granted FDA orphan, fast track, breakthrough and rare pediatric designations.
Zevra Therapeutics, formerly called KemPharm, rebranded in early 2023 following its 2022 acquisition of arimoclomol for $12.8 million from Orphazyme.
In 2021, Orphazyme received a complete response letter for arimoclomol after the FDA identified three issues, according to Zevra investor slides: It wanted more evidence about the use of a clinical severity measurement, more analysis of some missing data, and more support and data related to efficacy evidence.
Zevra said it believes it has addressed the FDA issues in the CRL by providing additional evidence to support the use of what’s known as the Niemann-Pick type C Clinical Severity Scale and by conducting additional studies used to support the potential mechanism of action.
In April, the company said it would present data at the Society for Inherited Metabolic Disorders annual meeting in North Carolina from its expanded access program, which is an FDA program where patients can access experimental drugs outside of clinical trials. The company said in a press release that the data support the use of arimoclomol in adults with the disease, with a clinically meaningful slowing of disease progression.
The company estimates there are only about 1,800 people with Niemann-Pick disease type C in the US and Europe. It typically causes the abnormal processing of lipids like cholesterol in tissues, which can build up, including in the brain.
The FDA has until Sept. 21 to decide on the approval of arimoclomol.