The US Food and Drug Administration (FDA) has approved Zevra Therapeutics’ Miplyffa (arimoclomol) to treat Niemann-Pick disease type C (NPC), an ultra-rare lysosomal storage disorder affecting one in every 100,000 to 120,000 births.
Miplyffa is now the first drug to be approved by the regulator for NPC and has been specifically authorised for use in combination with the enzyme inhibitor miglustat to treat neurological symptoms associated with the disease in adults and children aged two years and older.
It is estimated that 900 people in the US are currently living with NPC, which is caused by mutations in the NPC1 or NPC2 genes and characterised by the inability to properly metabolise lipids.
Patients progressively lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation and fine motor skills.
Administered orally, arimoclomol is designed to increase the production and activation of molecular chaperone proteins, which could increase the breakdown of accumulating lipids.
The FDA’s decision on the drug follows a recent recommendation from its Genetic Metabolic Diseases Advisory Committee and was supported by a totality of data, including results from a one-year trial in NPC patients aged two to 19 years.
The Miplyffa/miglustat combination halted disease progression through the 12 months of treatment, as demonstrated by a decrease of 0.2 points from baseline on the 4-domain NPC Clinical Severity Scale, compared to 1.9 points of progression for patients treated with miglustat alone.
Janet Maynard, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, said: “NPC is a serious disease that leads to enormous adverse impacts on patients and families. Despite extensive research efforts, there have not been approved treatments to meet the significant needs of patients.
“The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering.”
Neil McFarlane, Zevra’s president and chief executive officer, described the approval as a “monumental milestone for NPC patients and their family members in the US”.
The company, which has also been granted a priority review voucher in conjunction with the authorisation, said it will immediately initiate its launch activities for Miplyffa and expects the drug to be commercially available in eight to 12 weeks.