FDA Approves Levacetylleucine for Treatment of Niemann-Pick Disease Type C
The approval comes shortly after the FDA approved arimoclomol from Zevra Therapeutics to treat neurological manifestations of the disease in combination with miglustat.
The FDA approved levacetylleucine (Aqneursa) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients who weigh 15 kg or more, IntraBio said in a release.1 The decision makes levacetylleucine the first stand-alone therapy for the disease approved in the United States.
FDA Approves Levacetylleucine for Treatment of Niemann-Pick Disease Type C / Tada Images - stock.adobe.com
The approval of levacetylleucine was based on data from the IB1001-301 trial (NCT05163288), in which the therapy met its primary endpoints of Scale for the Assessment and Rating of Ataxia (SARA) and functional SARA (fSARA). The therapy also met all of its key secondary endpoints, including Spinocerebellar Ataxia Functional Index (SCAFI), Modified Disability Rating Scale, and EuroQuol-5 Dimension (EQ-5D) Quality of Life Scale. Data from the study was published in The New England Journal of Medicine.2
READ MORE: Opill Marks a New Era for Birth Control Access
“The FDA approval of Aqneursa marks a significant breakthrough for those living with NPC,” Laurie Turner, Family Services Manager at the National Niemann-Pick Disease Foundation, said in a release.1 “For too long, our community has been without an approved therapy for the treatment of NPC. Today we celebrate this tremendous milestone for individuals and families living with NPC. We are immensely thankful for the dedication to innovative research that has led to this approval, and we are ready to help families embark on this new chapter of treatment.”
IB1001-301 was a pivotal, randomized, double-blind, placebo controlled, multi-center phase 3 study evaluating the safety, tolerability and efficacy of levacetylleucine in patients aged 4 and older with a confirmed diagnosis of NPC. The study included 3 periods: a 2 week baseline period, a 12 week period where patients randomly received either levacetylleucine or placebo, and another 12 week period where patients received the opposite treatment they initially received. The study cohort included 60 patients with a confirmed genetic diagnosis of NPC.
The study found that patients who received levacetylleucine showed a greater improvement in fSARA score compared to those who received placebo, with a mean treatment difference of -0.4. At baseline, the mean SARA score was 15.68. After 12 weeks of treatment, the mean change in total SARA score was −1.97±2.43 points, compared to −0.60±2.39 points in the placebo group.
Levacetylleucine was seen to be well tolerated, with a similar incidence of adverse events between the treatment and placebo groups. The most common adverse events were abdominal pain, dysphagia, upper respiratory tract infections, and vomiting. No treatment-related serious adverse events occurred during the study period.
The approval of levacetylleucine comes shortly after the FDA approved another treatment for NPC. Arimoclomol (Miplyffa) from Zevra Therapeutics was approved for the treatment of neurological manifestations of NPC in combination with miglustat in adult and pediatric patients aged 2 years and older. The therapy is an orally delivered, first-in-class investigational medication that increases the activation of transcription factors EB (TFEB) and E3 (TFE3), which results in the upregulation of coordinated lysosomal expression and regulation genes.
“Today’s action further underscores the agency’s commitment to support development of new treatments for rare diseases,” 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 release.3 “This approval again demonstrates the FDA’s commitment to work with the scientific community to overcome the unique challenges that may arise with rare disease drug development.”
READ MORE: FDA Updates Resource Center
Pharmacy practice is always changing. Stay ahead of the curve: Sign up for our free Drug Topics newsletter and get the latest drug information, industry trends, and patient care tips, straight to your inbox.
