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Novel Drug & Emergent Therapeutics | Pharma | Rare Diseases | Neurology
Aqneursa (levacetylleucine) for treating neurological symptoms of Niemann-Pick disease type C (NPC) in adults and pediatric patients
Published on MedED: 7 October 2024
Originally published: 24 September 2024
Source: FDA Press Release
Type of article: Novel Drug & Emergent Therapy News
MedED Catalogue Reference: MNDR001
Category: Rare Diseases
Crossreference: Neurology
Category tags: pacemaker leads, Boston Scientific,
| Product Category | Product Name | Company | Status |
|---|---|---|---|
| FDA Approved |
24 September 2024, FDA Press Release
The U.S. FDA has approved Aqneursa (levacetylleucine) for the treatment of the neurological symptoms of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kilograms.
This marks the second FDA approval within a week for NPC, highlighting its commitment to rare disease treatments.
Miplyffa (arimoclomol), was the first drug approved by the FDA to treat NPC. Taken as an oral preparation in combination with the enzyme inhibitor treatment Miplyffa, was approved to treat the neurological symptoms associated with NPC in adults and children two years of age and older. The drug was approved on the 20th of September.
Niemann-Pick disease type C (NPC) is a rare, inherited lysosomal storage disorder affecting approximately 1 in 100,000 live births. Patients with NPC experience a combination of systemic, neurological, and psychiatric symptoms, leading to significant impairments in daily functioning. Despite existing treatments, none have effectively targeted the full range of debilitating symptoms that severely impact patients’ quality of life.
Aqneursa, developed by IntraBio Inc., is the first stand-alone therapy for NPC, a genetic disorder caused by mutations in the NPC1 or NPC2 genes. These mutations disrupt lipid transport within cells, leading to progressive neurological decline and organ damage.
The approval was based on a pivotal Phase 3 trial (NCT05163288), which showed that Aqneursa significantly improved neurological symptoms, as measured by the functional Scale for the Assessment and Rating of Ataxia (fSARA). The study enrolled 60 participants aged 4 years and older with mild neurological symptoms, and it showed that Aqneursa outperformed placebo in terms of symptom management, specifically with a significant reduction in ataxia scores.
While Aqneursa showed a strong efficacy profile, common side effects included abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting. Aqneursa also carries a warning regarding embryo-fetal harm, emphasizing the need for caution in pregnant patients.
Aqneursa’s approval was granted under multiple FDA designations, including Fast Track, Priority Review, Orphan Drug, and Rare Pediatric Disease, reflecting the urgency of addressing rare disorders like NPC.
Clinical Trials & Supporting Information
| Clinical Study State | ClinicalTrial.gov Registration | Study Name |
|---|---|---|
|
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
|
Access Original Press Release
14 September 2024 | FDA Approves New Drug to Treat Niemann-Pick Disease, Type C
Access Company Press Release
IntraBio Announces U.S. FDA Approval of AQNEURSA for the Treatment of Niemann-Pick Disease Type C
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