AQNEURSA ® (levacetylleucine) Recommended for EU Approval by the CHMP to Treat Niemann-Pick Disease Type C
"This positive CHMP opinion represents another important milestone in expanding access to AQNEURSA to the global NPC community," said Dr. Marc Patterson, Chief Medical Officer of IntraBio. "The recommendation reflects the strength of our clinical data and the potential for AQNEURSA to be a foundational therapy for NPC, delivering meaningful benefits for patients. We are proud to work alongside the NPC community to bring this long-awaited treatment option to even more families.'
NPC is a rare, inherited lysosomal disorder characterized by progressive neurological deterioration, leading to loss of motor function, difficulties with speech and swallowing, and cognitive decline. 3 NPC affects both children and adults, significantly impacting quality of life and daily functioning. 3,4
"For NPC families like mine, this positive opinion brings long-awaited hope for a treatment that can actually offer improvements," said Carmelo Fernández, President of Fundación Niemann-Pick de España. "We have waited years for a therapy that can make a meaningful difference in the lives of people with NPC, and today's announcement brings us one step closer."
The positive CHMP's opinion is based on results from IntraBio's pivotal Phase III randomized, placebo-controlled, clinical trial (IB1001-301; NCT05163288), which evaluated the impact of AQNEURSA on neurological symptoms and functioning in pediatric and adult patients (n=60) with a confirmed diagnosis of NPC. AQNEURSA significantly improved neurological symptoms and functional abilities across its primary and all secondary endpoints within 12 weeks of treatment versus placebo, and was well tolerated throughout the development program. 1 The CHMP opinion was further supported by long-term extension phase data showing that treatment with AQNEURSA had disease-modifying and neuroprotective effects, helping to reverse disease progression over time. 2 Detailed results from the IB1001-301 trial were published in The New England Journal of Medicine in February 2024. 1
About AQNEURSA
AQNEURSA was approved by the U.S. Food and Drug Administration (FDA) on 24 September 2024 for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. 5
Since approval in the U.S., AQNEURSA has experienced rapid adoption as a frontline therapy for NPC with continued growth in demand.
IntraBio remains on track to proceed with additional global regulatory submissions for AQNEURSA in 2025 and beyond.
IntraBio's Phase III Pivotal trial investigating N-Acetyl-L-Leucine (levacetylleucine) for Ataxia-Telangiectasia has completed recruitment in under two months, ultimately over-enrolling the trial by over 167%. Data readout is expected in Q1 of 2026.
U.S. IMPORTANT SAFETY INFORMATION
Embryo-Fetal Toxicity
Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. The decision to continue or discontinue AQNEURSA treatment during pregnancy should consider the female's need for AQNEURSA, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease.
Pregnancy and Lactation
For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with AQNEURSA. Advise females of reproductive potential to use effective contraception during treatment with AQNEURSA and for 7 days after the last dose if AQNEURSA is discontinued.
There are no data on the presence of levacetylleucine or its metabolites in either human or animal milk, the effects on the breastfed infant or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for AQNEURSA and any potential adverse effects on the breastfed infant from levacetylleucine or from the underlying maternal condition.
Adverse Reactions
The most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
Drug Interactions
Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine or N-acetyl-D-leucine. The D-enantiomer, N-acetyl-D-leucine, competes with levacetylleucine for monocarboxylate transporter uptake, which may reduce the levacetylleucine efficacy.
Monitor more frequently for P-gp substrate related adverse reactions when used concomitantly with AQNEURSA; AQNEURSA inhibits P-gp; however, the clinical significance of this finding has not been fully characterized.
To report SUSPECTED ADVERSE REACTIONS, contact IntraBio Inc. at 1-833-306-9677 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please click here for Full Prescribing Information for AQNEURSA: https://www.aqneursahcp.com/wp-content/prescribing-information.pdf
About Niemann-Pick Disease Type C
Niemann-Pick disease Type C (NPC) is a rare (1:100,000 live births), prematurely fatal, autosomal recessive, lysosomal storage disorder. 6 The disease presents with systemic, psychiatric, and neurological symptoms, including cerebellar ataxia. NPC is chronic and progressive in nature and is characterized by rapid degeneration of the cerebellum and major organ systems which severely impacts the quality of life. 3,4,7
About IB1001-301
IB1001-301 (NCT05163288) is a multinational, randomized, placebo-controlled, crossover trial that evaluates the safety and efficacy of IB1001 (AQNEURSA, levacetylleucine) in pediatric and adult patients with NPC. Patients aged 4 years and older were screened at trial sites in Australia, Europe, the United Kingdom, and the United States.
Patients were assessed during a baseline period and then randomly assigned (1:1) to receive orally administered IB1001 or placebo for 12 weeks. At the end of the 12-week treatment period, patients crossed over and initiated therapy with the alternate study drug (IB1001 or placebo) over the subsequent 12-week period. Patients who completed the study had the option to participate in an open-label Extension Phase, with some patients having been dosed for over 5 years.
About IntraBio
IntraBio Inc. is a global biopharmaceutical company that develops and commercializes targeted therapies for rare and common neurological and neurodevelopmental diseases. IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide, and leverages the expertise of its scientific founders from the University of Oxford and the University of Munich.
For more information about IntraBio, please visit the company's website at intrabio.com and follow on LinkedIn (@IntraBio-Inc).
References
1. Bremova-Ertl T, et al. J Neurol. 2022;269(3):1651-1662
2. Patterson, Marc C., et al. "Disease-modifying, neuroprotective effect of N-acetyl-l-leucine in adult and pediatric patients with Niemann-Pick disease type C." Neurology 105.1 (2025): e213589.
3. Geberhiwot T, et al. Orphanet J of Rare Dis. 2018;13:50
4. Patterson MC, et al. Orphanet J Rare Dis. 2013;8:12; 3. NORD. NPC Signs & Symptoms. Published Dec 12, 2023. Accessed May 19, 2024.
5. AQNEURSA. Prescribing Information. IntraBio Inc
6. Burton BK, Ellis AG, Orr B, et al. Estimating the prevalence of Niemann-Pick disease type C (NPC) in the United States. Mol Genet Metab
7. Vanier MT. Orphanet J Rare Dis. 2010;5:16.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Business Wire
22 minutes ago
- Business Wire
Aetna Marks 3 Years of Type 2 Diabetes Reversal through Virta Health, Thousands See Results
PHOENIX--(BUSINESS WIRE)-- Banner|Aetna, a joint venture health plan owned by Banner Health and Aetna, a CVS Health company, celebrates three years of investing in members by offering an evidence-based diabetes reversal initiative. In 2022, the insurer was an early adopter of Virta Health for diabetes reversal – the process of reducing blood glucose levels to below the threshold used to diagnose diabetes. 1 Since then, the program enrollment has grown 290% and thousands of members have achieved sustainable health transformations while reducing their need for expensive weight loss medications, like Wegovy and Ozempic. 2 The flexible app-based program uses a virtual model to address the root cause of metabolic disease through individualized nutrition plans that fit members' unique lifestyles, cultural background, dietary preferences, and budget. Thanks to the personalized approach and one-on-one expert support, participants are experiencing significant and lasting outcomes. Recent data shows that Banner|Aetna members saw meaningful metabolic health improvements after 365 days of program enrollment, including the following. Blood sugar reduction: On average, members saw a 0.97% reduction in A1C blood sugar levels. 3 Every one-point decrease in A1C reduces the risk of long-term diabetes complications by up to 40%. Prescription reduction: Members eliminated 57% of diabetes medications (excluding metformin), including a 66% reduction in insulin dosages. Of those on insulin, 44% eliminated it completely. 4 Clinically significant weight loss: 61% of members experienced clinically significant weight loss (5% or more); on average, members lost 8% of their body weight. 5 Clinically significant weight loss can lead to better sleep, reduced inflammation, improved blood pressure, reduced risk of heart disease and positive impacts on arthritis and fatty liver disease. In addition to the diabetes reversal program demonstrating outstanding clinical results, Banner|Aetna's omnichannel outreach approach achieves 3x higher member engagement compared to industry averages. 6 Through deep integration with Banner Health—one of Banner|Aetna's parent companies and Arizona's largest healthcare delivery system—the insurer leverages the Banner Health clinical and care management teams to refer eligible members into a diabetes management program. 'The U.S. is facing a metabolic disease crisis. One in 10 people lives with diabetes, and most have type 2. One in three has prediabetes,' explains Ed Clarke, MD, Banner|Aetna Medical Director and VP, CMO Banner Plans & Networks. 'These conditions take a toll on the physical, emotional and financial health of millions of people in Arizona. That's why Banner|Aetna collaborated with Virta to offer members a holistic, non-pharmacological approach that puts disease reversal within reach. The feedback from members who have completed the program shows its dramatic impact on their lives. Not only do they gain a better understanding of disease management, but more importantly how they can take control health of their health.' The results speak for themselves as members are achieving notable clinical outcomes helping them reclaim their lives, while also having a positive effect that ripples out to their families and the broader community. For Banner|Aetna member Shelby*, learning to use food as medicine has led to 'amazing' changes in her life, including increased energy, better sleep, and weight loss. 'I have learned how my body reacts to a wide array of foods and what I can eat to help it flourish,' she explains. 'I am very thankful for the support my coach provides every step of the way, and her encouragement that I am worth the hard work.' Banner|Aetna currently offers type 2 diabetes reversal to eligible members of fully insured and Administrative Services Only groups. Additionally, Banner|Aetna self-funded employer groups can access Virta's prediabetes and obesity reversal offerings, which use the same innovative care model. About Banner|Aetna Banner|Aetna is a health insurance company focusing on bettering member outcomes at a lower cost, all while improving the overall member experience for employers and consumers in Arizona. The collaboration combines Banner Health's quality, local providers and delivery systems with Aetna's health-plan experience, care management and health information technology. Banner|Aetna is the brand name used for products and services provided by Banner Health and Aetna Health Insurance Company and Banner Health and Aetna Health Plan Inc. For more information about Banner|Aetna, visit About Banner Health Banner Health is one of the largest, secular nonprofit health care systems in the country. In addition to 33 hospitals, Banner also operates an academic medicine division, Banner – University Medicine, and Banner MD Anderson Cancer Center, a partnership with one of the world's leading cancer programs, MD Anderson Cancer Center. Banner's array of services includes a health insurance division, employed physician groups, outpatient surgery centers, urgent care locations, home care and hospice services, retail pharmacies, stand-alone imaging centers, physical therapy and rehabilitation, behavioral health services, a research division and a nursing registry. To make health care easier, 100% of Banner-employed doctors are available for virtual visits and patients may also reserve spots at Banner Urgent Care locations and can book appointments online with many Banner-employed doctors. Headquartered in Arizona, Banner Health also has locations in California, Colorado, Nebraska, Nevada and Wyoming. For more information, visit About CVS Health CVS Health is a leading health solutions company building a world of health around every consumer, wherever they are. As of March 31, 2025, the Company had more than 9,000 retail pharmacy locations, more than 1,000 walk-in and primary care medical clinics, a leading pharmacy benefits manager with approximately 88 million plan members, and a dedicated senior pharmacy care business serving more than 800,000 patients per year. The Company also serves an estimated more than 37 million people through traditional, voluntary and consumer-directed health insurance products and related services, including highly rated Medicare Advantage offerings and a leading standalone Medicare Part D prescription drug plan. The Company's integrated model uses personalized, technology driven services to connect people to simply better health, increasing access to quality care, delivering better outcomes, and lowering overall costs. 1 Riddle MC, Cefalu WT, Evans PH et al. Consensus Report: Definition and Interpretation of Remission in Type 2 Diabetes. Diabetes Care 1 October 2021; 44 (10): 2438–2444. 2 Virta Health & Banner|Aetna Clinical Outcomes. March 31, 2025. 3 Virta Health & Banner|Aetna Clinical Outcomes. February 27, 2025. 4 Ibid. 5 Ibid. 6 Engagement data reported by Virta Health. January 2023-May 2025. * The member's uncompensated testimonial reflects the enrollee's experience with Virta Health.
Business Wire
an hour ago
- Business Wire
New Health Affairs Article Reveals How Medicare Payment Rules Violate Biological Laws, Worsen Kidney Care Disparities
WASHINGTON--(BUSINESS WIRE)--The National Minority Quality Forum (NMQF) today announced the release of the second article in its groundbreaking Health Affairs series on the Physical Laws Framework (PLF)—a scientific approach that evaluates healthcare policies based on whether they align with the fundamental biological laws governing human health and disease. The new article, " Payment Systems Violate the Physics of Life: How ESRD Bundle Policies Disrupt Biological Order in Minority Communities," demonstrates how Medicare's payment structure for end-stage renal disease (ESRD) directly contradicts the biological requirements of kidney function, creating measurable harm particularly in Black and Hispanic communities. What is the Physical Laws Framework? The Physical Laws Framework (PLF) recognizes that human biology operates according to immutable natural laws — just as gravity governs physics, specific biological principles govern how diseases progress, how organs function, and how treatments must be timed. PLF provides a scientific standard for evaluating whether healthcare policies work with or against these biological realities. When policies violate these laws, they don't just create inefficiencies—they accelerate disease progression and shorten lives. "The kidneys maintain a precise biochemical balance that keeps us alive. This isn't negotiable—it's physics," said Dr. Frita Fisher, nephrologist and co-author of the article. "When payment policies prevent patients from getting phosphate binders when their bodies need them, we're not just delaying care—we're disrupting fundamental biological processes." Key Findings: Biology vs. Bureaucracy The article reveals how Medicare's decision to bundle oral phosphate therapies into dialysis center payments creates a direct conflict with biological necessity: Biological Reality: Phosphate levels must be controlled continuously, not just during dialysis sessions. Patients need immediate access to medications when biochemical imbalances occur. Policy Contradiction: The bundling policy forces patients to obtain critical medications only through dialysis centers, eliminating pharmacy access and creating dangerous gaps in treatment. Equity Impact: Black Americans comprise 35% of dialysis patients but face the greatest barriers under this system, as they are more likely to rely on pharmacies and long-term care facilities now excluded from dispensing these life-sustaining medications. Innovation Blocked: Advanced treatments like XPHOZAH, which can dramatically improve medication adherence and phosphate control, become effectively inaccessible under the bundled payment model. "This is what happens when economic models ignore biological laws," said Dr. Janice Desir, Co-Chair of NMQF's Nephrologists for Equitable Kidney Care. "The policy assumes patients can wait for their next dialysis appointment to get medications, but elevated phosphate levels don't wait—they cause immediate, cumulative damage to blood vessels and organs." A Scientific Path Forward "The PLF gives us an objective, scientific standard for healthcare policy," said Gary A. Puckrein, PhD, President and CEO of NMQF. "Just as engineers must respect the laws of physics when building bridges, healthcare policymakers must respect the laws of biology when designing payment systems. This isn't about opinion—it's about measurable biological reality." The article is part of a 15-part Health Affairs series running through March 2026. Each installment applies PLF to different healthcare challenges, demonstrating how aligning policy with biological laws can reduce disparities and improve outcomes. Join the National Dialogue NMQF invites healthcare leaders, policymakers, clinicians, and patient advocates to engage with the Physical Laws Framework: Visit to: Download the complete Physical Laws Framework Join discussion forums with healthcare leaders Register for upcoming webinars and LinkedIn Live sessions Access resources for implementing PLF principles Explore the Series: Health Affairs: National Minority Quality Forum Sponsored Series Upcoming Events: August 13: LinkedIn Live discussion with article authors August (tentative): Webinar: "Applying Biological Laws to Payment Policy" November 5-6: Cancer Equity Summit featuring PLF applications About NMQF: Founded in 1998, the National Minority Quality Forum (NMQF) is a United States-based health care research, education, and advocacy organization whose mission is to reduce patient risk and advance health equity by ensuring optimal care for all. NMQF utilizes data and research to support and mobilize healthcare organizations, leaders, policymakers, and patients in advocating for biologically-aligned care that reduces hospitalizations, disabilities, and deaths while promoting high-quality, long lives, particularly for the most vulnerable. For more information, visit
Business Wire
an hour ago
- Business Wire
New Hospital Management Software PeriOptimization developed under the leadership of a VA-Affiliated Surgeon, shows major efficiency; set to Save Millions for the Veterans Administration Healthcare
MARLTON, N.J.--(BUSINESS WIRE)--After years of development and testing, PeriOptimization, a patient and staff management software system for hospitals, is poised to modernize and streamline operations across VA Healthcare settings. Based on initial feedback and pilot tests, it demonstrates significant success for improving overall efficiency and workflow. Early estimates project cost savings exceeding $200 million over 8 years and if broadly adopted over $500 million or more. At the forefront of this innovation is Dr. Robert C. Villare, a vascular and general surgeon and New Jersey resident. Dr. Villare served at the Lebanon Veterans Affairs Medical Center in Pennsylvania for eight years and brings first-hand experience to the operational challenges healthcare workers face every day. Unlike many health IT projects developed outside the clinical setting, PeriOptimization was conceived, designed, and tested by a surgeon working directly with software engineers and clinical staff. The result is a fully integrated Surgery and Dental Management Platform tailored to enable process improvement in VA surgical and dental services. 'This project was born out of a clear need to streamline workflows and reduce inefficiencies,' said Dr. Villare. 'By listening to end users, we've created a solution that is practical and powerful. We eliminated redundancies and rework and standardized to boost operational efficiency.' What is PeriOptimization? PeriOptimization is a modern web-based software solution designed to replace outdated scheduling and operational systems in surgery and dental services. It is: Customizable and scalable to fit various facility needs. Integrated with legacy platforms such as VistA. Cost-effective and built for rapid deployment. Focused on reducing inefficiencies and improving user experience. The Need for Change The VA pioneered electronic medical records in the 1970's with the creation of VistA. Many VA hospital systems still rely on fragmented outdated platforms that increase delays, adds administrative burdens, worsens staff burnout and reduces clinical efficiency. Importantly, many of the old forms and processes yield little to no value for patient care. Before PeriOptimization: Separate systems led to communication gaps. Manual scheduling consumed excessive time. Resource management was reactive. With PeriOptimization: Schedules and resources are automated and standardized. Staff spend more time on patient care. Departments coordinate in real-time. Data supports better, more immediate decision-making. Key Features & Benefits: Customizable & Scalable – Adaptable for facilities of all sizes and specialties. Real-Time Reporting – Enables faster, data-driven decisions. Seamless Integration – Built to work with existing VA infrastructure. Improved Staff Satisfaction – Reduces task redundancy and burnout. Improved Patient Care – Enhances continuity, speed, accuracy and safety. Cost – Estimated savings of $209 million over 8 years system-wide—actual savings may exceed $500 million or more. Next Steps Following successful testing over a 3-year period, PeriOptimization is under consideration by healthcare organizations, including the VA. At this time, no official endorsements or deployment decisions have been made by the U.S. Department of Veterans Affairs. 'This isn't just a software update,' Dr. Villare added. 'It's a culture shift—one that empowers providers and staff while honoring a commitment to patient care.' For interviews or a product demo, contact joe@
