FDA greenlights first treatment for Niemann-Pick disease type C
NPC is a progressive disease where the patient has a mutation in NPC1 or NPC2 – genes that are involved in the processing of cholesterol and other lipids inside cells. Cells with this mutation have a faulty management of lipids, leading to organ damage and neurological symptoms including difficulty with speech and cognition.
Delivered as an oral treatment three times per day, Miplyffa boosts the activity of two proteins in the cell called transcription factors EB (TFEB) and E3 (TFE3). These proteins upregulate genes encoding proteins that help lipids within cells to return to healthy levels. Miplyffa is specifically approved for use in combination with miglustat, a drug that is used off-label to treat NPC in around 80% of patients in the U.S.
Miplyffa was approved based on results from a phase 2 trial where the drug slowed the progression of NPC compared to patients given a placebo. The company plans to launch the drug within 12 weeks of its approval and expects to set an average price of $85,000 per month for each patient, said president and CEO of Zevra Therapeutics Neil McFarlane in a public conference call.
At the same time, Zevra launched a patient support program called AmplifyAssist to help patients and carers with personalized insurance coverage education and assistance with finding funding for their needs.
“The FDA approval of Miplyffa marks a significant moment for those living with NPC and the global NPC community,” stated Elizabeth Berry-Kravis, professor at Rush University Medical Center, who worked with Zevra to research Miplyffa use in a real-world setting. She added that effective NPC treatment “requires multiple treatment options due to the complexity of the disease” and that “patients will now have more access to treatments to tackle this devastating disease.”
Arimoclomol was previously developed by the Danish company Orphazyme, which rose to fame in 2021 as a so-called ‘meme’ stock whose price was influenced by social media. In the same year, Orphazyme was slapped with a rejection of arimoclomol from the U.S. FDA in 2021, with the regulator requesting more data. Grappling with bankruptcy, Orphazyme sold its assets to Zevra (then known as KemPharm) in 2022 for $12.8 million.
Another candidate in development is IntraBio’s small molecule therapy which is being reviewed by the U.S. FDA with a decision expected in the coming days. And the Dutch company Azafaros is taking its candidate nizubaglustat into phase 3 testing in patients with a genetic diagnosis of either GM2 gangliosidosis or NPC next year.
