pharmaphorum August 5, 2024
An FDA advisory committee has recommended approval of Zevra Therapeutics’ arimoclomol for the ultra-rare disorder Niemann-Pick disease, three years after the regulator rejected the drug.
The Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted 11 to five that the data support the approval of the drug for Niemann-Pick disease type C (NPC), a progressive genetic disorder that leads to the accumulation of fatty substances in tissues and can cause a range of symptoms, including liver and lung disease and neurological impairment.
In June 2021, when arimoclomol was owned by Danish biotech Orphazyme, the FDA issued a complete response letter for the drug and asked for an additional clinical trial, setting the programme back.
After the EMA’s human medicines committee also said...
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