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.
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.
Those trials largely failed or yielded mixed results. Niemann Pick, or NPC, is slow-moving and can have a different constellation of symptoms in different patients, including motor deficits, cognitive deficits, seizures and difficulty hearing and swallowing — making it hard to show that a drug is having an impact after a year, particularly if that impact is modest.
This article is exclusive to STAT+ subscribers
Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+.
Already have an account? Log in
Already have an account? Log in
