Curant Rare and IntraBio Expand Collaboration Following FDA Approval of AQNEURSA® for the treatment of Ataxia in patients with Ataxia-Telangiectasia

ATLANTA, GA – October 5, 2026 – Curant Rare has announced the expansion of its collaboration with IntraBio following U.S. Food and Drug Administration approval of AQNEURSA® (levacetylleucine) for the treatment ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥ 15 kg. As the exclusive rare disease pharmacy for AQNEURSA, Curant Rare will continue providing comprehensive patient support, access services, and therapy fulfillment.  

The approval marks an important advancement for individuals living with A-T, offering a new treatment option for ataxia associated with this rare and debilitating neurodegenerative disorder. It also expands Curant Rare’s support of the AQNEURSA patient community and reinforces the organization’s commitment to helping patients access innovative therapies for rare diseases for patients with A-T. 

“As the exclusive pharmacy for AQNEURSA, it is an honor to deliver the personalized care, education, and access support patients and families need throughout their treatment journey. Our continued collaboration with IntraBio reflects a shared dedication to improving the lives of individuals living with rare diseases,” said Marc O’Connor, Chief Business Officer of Curant Health. 

A-T is a rare, inherited disorder that affects multiple organ systems and is characterized by progressive ataxia, challenges with coordination and movement, immune dysfunction, and an increased risk of serious health complications. Patients often face complex care needs that require coordinated support among healthcare providers, caregivers, and specialty pharmacy teams. 

Through its specialized rare disease infrastructure, Curant Rare will continue working closely with healthcare providers and patient families to facilitate access to treatment, provide ongoing therapy support, and help patients navigate challenges related to care and adherence. 

The expanded indication underscores the shared commitment of IntraBio and Curant Rare in addressing unmet needs within the rare disease community. Together, the organizations remain focused on improving access to treatment and supporting patients living with  Niemann-Pick Disease Type C (NPC) and A-T.  

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 

• In Niemann-Pick disease type C, the most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting. 

• In ataxia-telangiectasia, the most common adverse reactions (incidence ≥5% and greater than placebo) are fall, skin laceration, and urinary tract infection. 

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. 

INDICATION 

AQNEURSA® (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. 

AQNEURSA® (levacetylleucine) is indicated for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg. 

Please click here for Full Prescribing Information for AQNEURSA. 

References:

1. AQNEURSA. Prescribing information. IntraBio. 2. Bremova-Ertl T, Ramaswami U, Brands M, et al. Trial of N-acetyl-L-leucine in Niemann-Pick disease type C. N Engl J Med. 2024;390(5):421-431. doi:10.1056/NEJMoa2310151 3. MIPLYFFA. Prescribing information. Zevra Therapeutics Inc; 2024. 4. Geberhiwot T, Moro A, Dardis A, et al; International Niemann-Pick Disease Registry (INPDR). Consensus clinical management guidelines for Niemann-Pick disease type C. Orphanet J Rare Dis. 2018;13(1):50. doi:10.1186/s13023-018-0785-7 5. 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. 2021;134:182-187. doi:10.1016/j.ymgme.2021.06.011 6. Kassen S, Parseghian C, Andrews P, et al. Niemann-Pick Type C Patient and Caregiver Voices: Externally-led, Patient-focused Drug Development Meeting. The Ara Parseghian Medical Research Fund at Notre Dame; 2019. 

About Curant Rare 

Curant Rare is a rare disease pharmacy dedicated to transforming the treatment experience for patients living with rare and complex conditions. Combining deep rare disease expertise with highly personalized, hands-on support, Curant Rare helps patients and families overcome barriers to therapy, navigate complex treatment journeys and stay supported throughout their care. Through dedicated care teams, clinical expertise and close coordination with healthcare providers and biopharmaceutical organizations, Curant Rare delivers the access, guidance and continuity patients need.  

About IntraBio 

IntraBio Inc., a US biopharmaceutical company, is focused on the development of novel drugs addressing rare and common neurological diseases. IntraBio’s platform technologies result from decades of research and collaboration with universities and institutions worldwide. Its clinical programs are based upon the expertise in lysosomal function and intracellular signaling of its scientific founders from the University of Oxford and the University of Munich. 

About AQNEURSA® (levacetylleucine)  

AQNEURSA is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.  

AQNEURSA is indicated for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg.  

Visit https://aqneursa.com/ to view the Full Prescribing Information, including Important Safety Information. 

Contact Information: 

Tim Griffin 

Brand Manager, Curant Rare 

TGriffin@CurantHealth.com