Zevra Therapeutics Inc. (ZVRA), a rare disease therapeutics company, Thursday, announced the commercial availability of Miplyffa for the treatment of Niemann-Pick disease type C along with AmplifyAssist to ensure the accessibility of the drug.
Miplyffa, in combination with the enzyme inhibitor miglustat, was approved by the FDA in September of this year to treat neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and children 2 years of age and older.
NPC is a neurodegenerative lysosomal storage disorder that causes cholesterol and lipid accumulation within the cells due to mutations in the NPC1 or NPC2 genes. Patients with NPC experience restrictions in both physical and cognitive abilities, characterized by significant neurological issues affecting speech, thinking, swallowing, walking, and precise movements.
Seeking to address access barriers to Miplyffa, the company has also launched AmplifyAssist, a comprehensive patient support program, facilitating timely prescription refills.
ZVRA closed Thursday's trading at $9.13, up 1.78%.
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