IntraBio Inc is very excited to confirm the U.S. Food and Drug Administration (FDA) has approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.
Quick Facts:
AQNEURSA was approved by the FDA on September 24, 2024, marking it as the second treatment for NPC approved within a week, following the approval of Miplyffa.
AQNEURSA is indicated for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in both adults and pediatric patients who weigh at least 15 kgs.
AQNEURSA targets and corrects the genetic mutations responsible for Niemann-Pick disease type C (NPC), helping to reduce lipid accumulation in cells and slow disease progression.
On September 24, 2024, the FDA approved AQNEURSA, marking the second treatment for Niemann-Pick disease type C (NPC) approved within a week. This rapid succession of approvals, including Miplyffa earlier in the week, underscores the FDA’s commitment to addressing rare diseases and providing new therapeutic options for patients. This breakthrough is particularly impactful for Australian families, who have long awaited advancements in treatment options.
In their announcement, the FDA stated, “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, M.D., M.H.S., director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in the FDA’s Center for Drug Evaluation and Research. “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.”
What is AQNEURSA?
AQNEURSA (levacetylleucine, IB1001) is a novel therapy designed to address the underlying causes of NPC. It works by targeting and correcting the genetic mutations responsible for the disease, potentially slowing its progression and improving the quality of life for patients.
How Does AQNEURSA Work?
AQNEURSA is designed to address the underlying causes of NPC by targeting the genetic mutations responsible for the disease. These mutations affect the body's ability to metabolize lipids, leading to their accumulation in cells. AQNEURSA works by correcting these faulty cellular processes, thereby reducing lipid buildup. This action helps slow the progression of the disease, potentially allowing patients to maintain their abilities and independence for a longer period. By improving cellular function, AQNEURSA aims to enhance the overall quality of life for patients and their families, offering hope and a better future for those affected by NPC.
What is the prescribing information for AQNEURSA?
AQNEURSA (levacetylleucine) is an oral medication approved for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and paediatric patients weighing at least 15 kilograms. Here is a summary of the prescribing information:
Dosage and Administration
Safety Information
Clinical Study
For more detailed information, including specific dosing guidelines and comprehensive safety information, please refer to the official prescribing information provided by the FDA¹².
What Does This Mean for NP-C Families in Australia?
The FDA approval of AQNEURSA is a significant milestone, but there are still a few steps before it becomes widely available in Australia. As with Miplyffa, the ANPDF will actively advocate for the swift approval and accessibility of AQNEURSA in our country. Advocacy will be crucial as we take the first steps in joining efforts to urge the Therapeutic Goods Administration (TGA) to expedite the approval process for these treatments. Together, we can push for progress and support those affected by Niemann-Pick disease.
Stay connected with ANPDF for updates and to participate in advocacy efforts to bring these treatments to Australia.
Looking Ahead
The FDA approval of AQNEURSA is a monumental step forward in the fight against Niemann-Pick disease. For Australian families, it brings hope, potential access to advanced treatment, and a renewed sense of purpose in advocating for a brighter future. Together, we can continue to push for progress and support those affected by this challenging condition.
Together, we can make a difference.
To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."
Ways to support NP-C in Australia:
Check out upcoming events
Shop at our online NPC store
Fundraise your way! Check our free resources and inspo online.
Sources:
(1) FDA Approves New Drug to Treat Niemann-Pick Disease, Type C. https://www.fda.gov/news-events/press-announcements/fda-approves-new-drug-treat-niemann-pick-disease-type-c
(2) FDA Approves New Drug to Treat Niemann-Pick Disease, Type C - PR Newswire. https://www.prnewswire.com/news-releases/fda-approves-new-drug-to-treat-niemann-pick-disease-type-c-302257740.html
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