IntraBio Announces Submission of Variation Application to the European Medicines Agency for AQNEURSA® for Ataxia-Telangiectasia

If approved, AQNEURSA® would become the first approved therapy for Ataxia-Telangiectasia in the European Economic Area

AUSTIN, Texas--(BUSINESS WIRE)--IntraBio Inc. today announced the submission of a variation application to the European Medicines Agency (EMA) to expand the approved Marketing Authorization Application (MAA) for AQNEURSA® (levacetylleucine) to include the treatment of Ataxia-Telangiectasia (A-T).

This submission represents the first regulatory application submitted to the EMA seeking approval of a therapy for the treatment of A-T, a rare, progressive, inherited neurodegenerative disorder. The variation application is subject to EMA validation before the start of formal scientific review.

In the United States, IntraBio’s supplemental New Drug Application for AQNEURSA for A-T has been accepted for review by the U.S. Food and Drug Administration and granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of September 19, 2026.

The variation application seeks to expand the label of AQNEURSA® to include A-T. AQNEURSA® is currently approved in the European Economic Area for the treatment of neurological manifestations of Niemann-Pick disease type C in adults and children aged 6 years and older weighing at least 20 kg, either in combination with miglustat or as monotherapy in patients who cannot tolerate miglustat, and in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg.

About Ataxia-Telangiectasia

Ataxia-Telangiectasia (A-T) is a rare, inherited, progressive neurodegenerative disorder that typically begins in early childhood. A-T is estimated to affect 1 in 40,000-100,000 people. It is characterized by degeneration of the cerebellum, resulting in worsening loss of coordination, impaired speech, abnormal eye movements, and eventual wheelchair dependence. Many patients also develop telangiectasia, immune deficiency with recurrent and potentially life-threatening infections, lung disease, and a markedly increased risk of cancer. There are currently no approved therapies for A-T.

About AQNEURSA®

EMA Indication
AQNEURSA® is authorized in the European Union for the treatment of neurological manifestations of Niemann-Pick disease type C in adults and children aged 6 years and older weighing at least 20 kg, either in combination with miglustat or as monotherapy in patients who cannot tolerate miglustat. EMA Indication and Important Safety Information

U.S Indication
AQNEURSA® (levacetylleucine) is approved in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg.

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 US Prescribing Information for AQNEURSA: https://www.aqneursahcp.com/wp-content/prescribing-information.pdf

About IntraBio

IntraBio Inc. is a global biopharmaceutical company headquartered in Austin, Texas, focused on developing and commercializing targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio’s platform technologies are built on decades of scientific research and collaboration with leading universities and institutions worldwide, including the University of Oxford and the University of Munich.

For further information, please contact:
Cass Fields
Vice-President of External Affairs
ccfields@intrabio.com
www.intrabio.com