Zevra Therapeutics has receieved approval from the FDA for Miplyffa to treat Type C Niemann-Pick disease (NPC).
The threapeutic can now be used in combination with miglustat for the treatment of NPC in adults and paediatric patients older than 2 years of age.
Because NPC is a rare condition, the company has also receieved a rare disease priority review voucher from the regulator.
The oral treatment is designed to reduce the neurological symptoms of NPC in patients, and has been shown to halt disease progression compared to patients given miglustat only.
Zevra estimates that Miplyffa will be commercially available in the US with eight to twelve weeks.
CEO and President of Zevra Theapeutics, Neil McFarlane, commented: “NPC is an ultra-rare, relentlessly progressive, degenerative, and fatal disease for which there were no FDA-approved treatment options until today,”
“The approval of Miplyffa is a monumental milestone for NPC patients and their family members in the US. We are immensely grateful for the unwavering support we have received over the years from the families and individuals impacted by NPC as well as the collaborative efforts of advocacy groups, researchers, and clinicians.”
It is estimated that 900 people in the US current live with NPC, which is currently fatal for those who have it.
It can affect everybody differently, but patients will characteristically experience progressive physical and cognitive limitations, with neurological impairments ranging from speech to cognition, swalling and fine motor skills.
“The FDA approval of MIPLYFFA marks a significant moment for those living with NPC and the global NPC community,” stated Dr. Elizabeth Berry-Kravis, Professor, Departments of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Director, RUSH Pediatric Neurosciences F.A.S.T. Center for Translational Research at Rush University Medical Center.
“Effective management of NPC requires multiple treatment options due to the complexity of the disease. Until today, there were no approved therapies in the US for NPC. With this labelled indication, patients will now have more access to treatments to tackle this devastating disease.”