FDA Approved AQNEURSA Ushers In New Hope For Niemann-Pick Disease Type C Patients
In a landmark decision, the U.S. Food and Drug Administration (FDA) has approved AQNEURSA™ (levacetylleucine), the first stand-alone therapy for treating neurological symptoms of Niemann-Pick disease type C (NPC), a rare and fatal genetic disorder.
Developed by IntraBio Inc., this breakthrough offers hope to patients and families who have long awaited an effective treatment for this devastating condition.
A Long-Awaited Therapeutic Breakthrough
NPC is a lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes, leading to impaired cholesterol and lipid transport within cells.
NPC is a lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes, leading to impaired cholesterol and lipid transport within cells.
This results in progressive neurological decline, organ damage, and a drastically shortened lifespan, with most patients surviving only 13 years on average.
Until now, treatment options were limited, with miglustat (an enzyme inhibitor) being one of the few available therapies, though it only slows disease progression rather than addressing the root cause.
AQNEURSA, an acetylated derivative of the amino acid leucine, works by enhancing mitochondrial function, correcting metabolic dysfunction, and improving lysosomal activity.
While its exact mechanism remains under investigation, preclinical studies suggest it helps restore neuronal membrane potentials and reduces neuroinflammation, key factors in NPC-related neurodegeneration.
Clinical Trial Success: Rapid And Significant Improvements
The FDA approval was based on Phase III trial data (IB1001-301, NCT05163288), a randomized, double-blind, placebo-controlled crossover study involving 60 NPC patients aged 4 and older.
The primary efficacy endpoint was measured using the functional Scale for the Assessment and Rating of Ataxia (fSARA), a modified version of the SARA scale focusing on gait, sitting, stance, and speech disturbances.
Key findings from the trial include:
Significant neurological improvement within 12 weeks of treatment.
A mean treatment difference of -0.4 points in fSARA scores compared to placebo, indicating reduced symptom severity (p < 0.001).
Patients switching from placebo to AQNEURSA saw further improvements, reinforcing the drug’s efficacy.
Well-tolerated safety profile, with the most common side effects being abdominal pain, dysphagia, upper respiratory infections, and vomiting.
A New Era In NPC Treatment
The approval of AQNEURSA marks a major milestone in NPC therapy, offering not just symptomatic relief but potential neuroprotective and disease-modifying effects.
Laurie Turner of the National Niemann-Pick Disease Foundation hailed the decision, stating, “For too long, our community has been without an approved therapy. Today, we celebrate this tremendous milestone for individuals and families living with NPC.”
Notably, just days before AQNEURSA’s approval, the FDA also greenlit Miplyffa (arimoclomol), another NPC treatment, underscoring the agency’s commitment to rare disease therapies.
Future Prospects For Aqneursa
AQNEURSA is now available for adults and pediatric patients weighing ≥15 kg1. IntraBio has also launched AQNEURSA Cares™, a patient support program offering financial assistance, insurance navigation, and educational resources.
Beyond NPC, AQNEURSA is being investigated for other neurodegenerative disorders, including GM1/GM2 gangliosidosis, ataxia-telangiectasia, and Sandhoff disease, potentially expanding its therapeutic reach.
New Hope For Patients
The FDA’s approval of AQNEURSA represents a transformative advancement in NPC treatment, bringing hope to patients who previously had no disease-specific therapy.
As research continues, this drug may pave the way for broader applications in neurological and lysosomal storage disorders, reaffirming the power of scientific innovation in tackling rare diseases.
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