Zevra Therapeutics plans to immediately start its launch activities for arimoclomol, marketed as Miplyffa, which is anticipated to be commercially available in 8 to 12 weeks in the United States.
Janet Maynard, MD, MHS
(Credit: LinkedIn)
According to a recent announcement, the FDA has granted approval of Zevra Therapeutics’ arimoclomol (Miplyffa), an oral medication, for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and children 2 years of age and older. The treatment, which is combined with the enzyme inhibitor miglustat, becomes the first drug approved by the agency to treat patients with this condition.1,2
The approval was based on data from a randomized, double-blind, placebo-controlled 12-month trial assessing arimoclomol among patients 2 to 19 years of age who had a molecularly confirmed diagnosis of NPC. In the trial, investigators randomized 50 patients 2:1 to treatment with weight-adjusted arimoclomol (31-mg to 124-mg) or placebo orally 3 times per day.
Among all the participants in the trial, 39 of them received miglustat as background treatment (78%). Findings showed that patients treated with arimoclomol, in combination with miglustat, had a slower disease progression, as shown by a 0.2 reduction from baseline on the rescored 4-domain NPC Clinical Severity Scale compared with 1.9 points of progression in the placebo group.
“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,” Janet Maynard, MD, MHS, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in the FDA’s Center for Drug Evaluation and Research, said in a statement.1 “The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering.”
The agency noted that arimoclomol contains a warning in the prescribing information for hypersensitivity reactions such as hives and angioedema, swelling under the skin. The FDA also recommends that patients who experience these adverse reactions should stop using the treatment. Additionally, the agency suggests that pregnant women or women who plan to become pregnant should not take arimoclomol. The most common adverse effects reported by patients treated with arimoclomol include upper respiratory tract infection, diarrhea and weight reduction. The treatment, in combination with miglustat, should be taken orally with or without food based on the recommended dose for the patient’s body weight, as recommended by the agency.
“Until now, those living with NPC have had no FDA-approved treatment to combat this devastating disease,” Laurie Turner, Family Services Manager, National Niemann-Pick Disease Foundation, said in a statement.2 “For more than 30 years, NNPDF and the community have been working to find treatments for NPC, and we are grateful for the diligence and commitment of the researchers, clinicians, families and Zevra for making this approval possible.”
Previously, the FDA granted arimoclomol priority review, orphan drug, rare pediatric disease, fast track, and breakthrough therapy designations for the treatment of patients with NPC. In August 2024, the therapy was the first product application discussed at the Genetic Metabolic Diseases Advisory Committee, a committee established to advise the agency for products that are used for prevention or treatment of genetic metabolic diseases.3
“NPC is an ultra-rare, relentlessly progressive, degenerative, and fatal disease for which there were no FDA-approved treatment options until today,” Neil F. McFarlane, president and chief executive officer at Zevra Therapeutics, said in a statement.2 “The approval of Miplyffa is a monumental milestone for NPC patients and their family members in the U.S. 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.”
“The FDA approval of Miplyffa marks a significant moment for those living with NPC and the global NPC community,” Elizabeth Berry-Kravis, MD, PhD, professor in the departments of pediatrics, neurological sciences, anatomy and cell biology and the director of the RUSH Pediatric Neurosciences F.A.S.T. Center for Translational Research at Rush University Medical Center, said in a statement.2 “Effective management of NPC requires multiple treatment options due to the complexity of the disease. Until today, there were no approved therapies in the U.S. for NPC. With this labeled indication, patients will now have more access to treatments to tackle this devastating disease.”
