Miplyffa commercially available for treatment of Niemann-Pick disease type C

Zevra Therapeutics has announced that Miplyffa, the first Nieman-Pick disease type C treatment approved by the FDA, is commercially available.

Miplyffa (arimoclomol) is recommended for usage in tandem with miglustat to address symptoms related to the neurodegenerative lysosomal storage disorder for patients aged 2 years and older, the company said in a press release.

The oral, three-times-per-day therapeutic, which can be administered by either the patient or a caregiver, was approved by the FDA in late September.

Days later, the FDA also approved Aqneursa (levacetylleucine, IntraBio Inc.) for the same purpose.

Commercialization of Miplyffa was bolstered by results from a phase 3 clinical trial, in which the therapeutic was found to arrest disease progression more significantly compared with placebo at 1 year as measured by change in the Niemann-Pick type C Clinical Severity Scale score.

“Early adoption of Miplyffa has exceeded our expectations, and we are pleased to report that product is now available for shipment to NPC patients,” Josh Schafer, Zevra’s chief commercial officer and executive vice president of business development, stated in the release. “We continue to receive prescription enrollments through our patient services program, AmplifyAssist, and are supporting patients as they navigate the benefits verification process to obtain Miplyffa.”