The Food and Drug Administration on Friday approved the first treatment for Niemann-Pick disease, type C, the culmination of years of advocacy and stutter-start drug development efforts in the rare neurological condition.
Zevra Therapeutics, the manufacturer, said on Monday it will charge on average around $1.02 million a year for the treatment. Pricing is weight-based, so will vary between $480,000 and $1.26 million, Zevra said.
The drug, known chemically as arimoclomol and now marketed as Miplyffa, was one of three that companies put in clinical trials around a decade ago, after parents of children with the disease pushed scientists and companies to focus on the condition and helped steer their efforts.
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