FDA approves Zevras Miplyffa as first drug for Niemann-Pick disease type C

The U.S. Food and Drug Administration (FDA) has approved Zevra Therapeutics' Miplyffa (arimoclomol) to treat Niemann-Pick disease type C (NPC), an extremely rare lysosomal storage disease that affects one in 100,000 to 120,000 births.

Miplyffa is now the first drug approved by the regulator for NPC and has been specifically approved for use in combination with the enzyme inhibitor miglustat to treat the neurological symptoms associated with the disease in adults and children aged two years and older.

An estimated 900 people in the United States currently suffer from NPC. The disease is caused by mutations in the NPC1 or NPC2 genes and is characterized by the inability to properly metabolize lipids.

Due to physical and cognitive limitations, patients increasingly lose their independence. Significant neurological impairments are evident in speaking, perception, swallowing, walking and fine motor skills.

When administered orally, arimoclomol is thought to increase the production and activation of molecular chaperone proteins, which may accelerate the degradation of accumulated lipids.

The FDA's decision on the drug follows a recent recommendation from its Advisory Committee on Genetic Metabolic Disorders and was supported by a variety of data, including results from a one-year study of NPC patients between the ages of two and 19.

The Miplyffa/Miglustat combination stopped disease progression during 12 months of treatment, as demonstrated by a 0.2 point decrease from baseline on the four-point NPC Clinical Severity Scale, compared with 1.9 points of progression in patients treated with miglustat alone.

Janet Maynard, director of the Office of Rare Diseases, Pediatrics, Urology and Reproductive Medicine in the FDA's Center for Drug Evaluation and Research, said, “NPC is a serious disease that has a tremendous negative impact on patients and families. Despite extensive research efforts, there are no approved treatments that address the significant needs of patients.

“The first approval of a safe and effective drug option for NPC will undoubtedly meet the essential medical needs of those affected.”

Neil McFarlane, President and CEO of Zevra, called the approval a “monumental milestone for NPC patients and their families in the United States.”

The company, which was also granted a Priority Review Voucher in connection with the approval, said it will begin launch activities for Miplyffa immediately and expects the drug to be commercially available in eight to twelve weeks.