The FDA has recently approved two new treatments for Niemann-Pick Type C (NPC), a rare and progressive genetic disorder, offering new hope for patients and families affected by this devastating condition. These approvals mark a significant advancement in the treatment landscape for NPC, which previously had no approved therapies.
Miplyffa: A New Option in Combination Therapy
On September 20, 2024, the FDA approved Miplyffa (arimoclomol) by Zevra Therapeutics, in combination with the enzyme inhibitor miglustat, for the treatment of neurological symptoms associated with NPC in adults and children aged 2 years and older. NPC is characterized by the body's inability to transport cholesterol and other lipids within cells, leading to organ damage and neurological impairments. The average lifespan for individuals with NPC is approximately 13 years.
The approval of Miplyffa was based on data from a randomized, double-blind, placebo-controlled 12-month trial involving patients aged 2 to 19 years with a molecularly confirmed diagnosis of NPC. The results demonstrated that Miplyffa, in combination with miglustat, halted disease progression through 12 months of treatment, as demonstrated by a decrease of 0.2 points from baseline on the R4DNPCCSS compared to 1.9 points of progression for patients treated with miglustat alone.
"The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering," said Janet Maynard, M.D., director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA Center for Drug Evaluation and Research.
Aqneursa: A Stand-Alone Treatment Option
Just days later, on September 25, 2024, the FDA approved Aqneursa (levacetylleucine) by IntraBio Inc. as a stand-alone therapy for the treatment of neurological manifestations of NPC in adults and pediatric patients weighing at least 15 kg. This approval provides another much-needed treatment option for individuals with NPC.
The approval of Aqneursa was based on data from a randomized, double-blind, placebo-controlled, two-period, 24-week crossover study. The study included 60 patients aged 4 years or older with NPC and at least mild neurological symptoms associated with the disease. The patients received 12 weeks of placebo and 12 weeks of levacetylleucine. They were also able to receive miglustat as background therapy.
Compared with placebo, the FDA said levacetylleucine was associated with improved function, as measured by a modified version of the Scale for the Assessment and Rating of Ataxia.
Implications for the NPC Community
These approvals represent a significant milestone for the NPC community, which has long awaited effective treatment options. The availability of both Miplyffa and Aqneursa offers hope for improved management of symptoms and enhanced quality of life for individuals with NPC.
"This is the second treatment the FDA has approved for NPC within the span of a week. Today’s action further underscores the agency’s commitment to support development of new treatments for rare diseases," said 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.
While both drugs offer potential benefits, they also come with certain risks and side effects. Miplyffa carries a warning for hypersensitivity reactions, while Aqneursa includes a warning about potential risks of embryo-fetal harm if used during pregnancy. Healthcare professionals should carefully consider these factors when prescribing these medications.
With these new treatment options now available, the NPC community can look forward to a future with improved care and management of this challenging condition.
