Zevra Therapeutics drug is the first FDA-approved treatment for extremely rare neurodegenerative disease

On Friday, the FDA approved Zevra Therapeutics, Inc. (NASDAQ:ZVRA) Miplyffa (arimoclomol), an oral medication for Neimann-Pick disease type C (NPC).

Miplyffa, in combination with Johnson & Johnson (NYSE:JNJ) enzyme inhibitor Zavesca (miglustat) is approved to treat neurological symptoms associated with NPC in adults and children two years of age and older.

Miplyffa is the first FDA-approved drug to treat NPC.

NPC is a rare genetic disorder that causes progressive neurological symptoms and organ dysfunction.

The cause is changes in the NPC1 or NPC2 gene that affect the necessary transport of cholesterol and other lipids within a cell. As a result, these cells do not function as intended, ultimately leading to organ damage.

Miplyffa was the first product application discussed at the inaugural meeting of the Genetic Metabolic Diseases Advisory Committee (GeMDAC) in August.

The committee voted positively (11 votes in favour, 5 against) because the data demonstrate the efficacy of arimoclomol in the treatment of patients with NPC.

GeMDAC was established in December 2023 to advise the agency on products used to diagnose, prevent or treat genetic metabolic diseases.

The safety and efficacy of Miplyffa were studied in a randomized, double-blind, placebo-controlled 12-month study in patients.

The efficacy of Miplyffa was demonstrated by the reassessed 4-domain NPC Clinical Severity Scale (R4DNPCCSS) score in patients using miglustat as background treatment.

The R4DNPCCSS is a measure of the disease progression in NPC. Higher values ​​indicate a more severe course of the disease.

Compared to placebo, Miplyffa resulted in a slower progression of the disease.

In 2021, the FDA issued a full response letter on arimoclomol as a treatment option for Niemann-Pick disease type C.

The CRL requested additional qualitative and quantitative evidence to further support the validity and interpretation of the 5-domain NPC Clinical Severity Scale, particularly the swallowing domain.

Zevra Therapeutics resubmitted its New Drug Application (NDA) for arimoclomol to the FDA in December 2023.

Price promotion: ZVRA shares were up 1.01% to $8.01 at last check on Friday.

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This article: Zevra Therapeutics' drug is the first FDA-approved treatment for an extremely rare neurodegenerative disease.

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