WASHINGTON D.C. — The U.S. Food and Drug Administration (FDA) has made a landmark decision to approve Aqneursa (levacetylleucine) for the treatment of neurological symptoms linked to Niemann-Pick disease type C (NPC), providing new hope for those afflicted by this rare genetic disorder. This approval, the second in a week for NPC, signifies the FDA’s resolute pledge to advance the development of treatments for rare diseases.
Niemann-Pick disease type C is a debilitating condition caused by mutations in the NPC1 or NPC2 gene, disrupting lipid transport within cells and leading to severe neurological and organ dysfunction. The prognosis for NPC is grim, with many patients succumbing to the disease in their teenage years.
“This is the second treatment the FDA has approved for NPC within the span of a week. Today’s action further demonstrates the agency’s commitment to support development of new treatments for rare diseases,” stated Janet Maynard, M.D., M.H.S., director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, within the FDA’s Center for Drug Evaluation and Research. Her statement underscores the FDA’s dedication to partnering with the scientific community to tackle the challenges presented by rare disease drug development.
The approval followed a comprehensive 24-week clinical study with 60 participants aged four and older, all confirmed to have NPC with neurological symptoms. The study employed a modified assessment tool, the functional SARA (fSARA), to evaluate the drug’s effectiveness. Patients receiving Aqneursa showed significant improvement over those given a placebo.
Despite its promise, Aqneursa is accompanied by a warning about the potential risks of embryo-fetal harm if used during pregnancy. Reported side effects include abdominal pain, difficulty swallowing, respiratory infections, and vomiting. The drug is administered orally, with dosage adjusted according to body weight.
The FDA granted Aqneursa Priority Review, Fast Track, Orphan Drug, and Rare Pediatric Disease designations, highlighting its potential to swiftly meet critical medical needs. The approval is a result of the collaboration between the FDA and IntraBio Inc., the drug’s developer.
For patients and families affected by NPC, Aqneursa offers a beacon of hope, promising improved management of symptoms and enhancing quality of life. This approval not only marks a significant milestone for those battling NPC but also underscores the FDA’s commitment to facilitating advancements in the treatment of rare diseases, paving the way for continued research and innovation in this critical area.
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