Zevra Therapeutics' Drug Becomes First FDA-Approved Treatment For Ultra-Rare Neurodegenerative Disease

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

Miplyffa, in combination with Johnson & Johnson's 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 drug approved by the FDA to treat NPC. 

NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction.

It is caused by changes in either the NPC1 or NPC2 gene, affecting the necessary transport of cholesterol and other lipids within a cell. As a result, these cells do not function as they should, ultimately causing organ damage.

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

The committee voted favorably (11 yes, 5 no) that the data supports that arimoclomol is effective in treating patients with NPC.

GeMDAC was established in December 2023 to advise the agency on products used for the diagnosis, prevention or treatment of genetic metabolic diseases. 

The safety and effectiveness of Miplyffa were evaluated in a randomized, double-blind, placebo-controlled 12-month trial in patients.

Miplyffa's efficacy was demonstrated by the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS) score in the patients who used miglustat as their background treatment.

The R4DNPCCSS is a measure of NPC disease progression. Higher scores signify a greater severity of the disease.

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

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

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

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

Price Action: ZVRA stock is up 1.01% at $8.01 at the last check on Friday.

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