IB1001

IB1001 is an orally administered, modified amino acid (N-Acetyl-L-Leucine). Its safety and tolerability profile has been studied extensively in clinical trials.
 


Given the urgent, unmet medical need, IB1001 is being developed for orphan indications where there are no FDA-approved therapies: GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) and Ataxia-Telangiectasia. This development is based on existing pre-clinical and clinical data demonstrating safety and efficacy in these disorders. 

Details on IntraBio’s clinical trials with IB1001 are available here.

Regulatory History
IntraBio has been granted fifteen Orphan Drug Designations (US Food and Drug Administration)/ Orphan Medicinal Drug Designations (European Commission) for the IB1000 Series for the treatment of NPC, GM1 Gangliosidosis, GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), Spinocerebellar Ataxias (of which there are over 40 known subtypes), Ataxia-Telangiectasia, Ataxia Oculomotor Apraxia type 4 (AOA4), as well as Multiple Systems Atrophy (MSA).
 


IntraBio has been granted three Rare Pediatric Disease Designations for IB1000s by the FDA for the treatment of NPC, GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and Ataxia-Telangiectasia. These Rare Pediatric Disease Designation makes IB1000s eligible for, and expedites the request of, a Rare Pediatric Disease Priority Review Voucher (PRV) granted at the time of marketing approval.
 


IntraBio has also been granted Fast Track Designation for IB1001 by the FDA for NPC and GM2 Gangliosidosis.
 

In September 2024, U.S. Food and Drug Administration (FDA) approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. 

IntraBio has filed a Marketing Authorization Application with the European Medicines Agency that is currently under review with an opinion expected H2 2025.

AQNEURSA is not currently approved for any other indication or by any other jurisdiction.

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.

IMPORTANT SAFETY INFORMATION
Do not take AQNEURSA if you:

• have not had a negative pregnancy test. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments

Before taking AQNEURSA, discuss with your doctor if you:

• are pregnant or planning to become pregnant. Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments.
• require the need for effective contraception during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
• are breastfeeding or plan breastfeed. It is not known if AQNEURSA or its metabolites pass into human or animal milk or effects on breastmilk production
• are taking P-gp substrate. Your doctor will monitor you for related adverse reactions
• have any other medical conditions
• are taking any medications and supplements, including N-acetyl-DL-leucine or N-acetyl-D-leucine, as some of these may impact the effectiveness of AQNEURSA

The most common side effects of AQNEURSA include abdominal pain, dysphagia, upper respiratory tract infections and vomiting. Speak with your doctor if these side effects persist or worsen.

Take AQNEURSA exactly as prescribed by your doctor.

If a dose of AQNEURSA is missed, skip the missed dose and take the next dose at the scheduled time.

Do not take 2 doses at the same time to make up for a missed dose.

AQNEURSA can be taken with or without food.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please click here for Full Prescribing Information for AQNEURSA