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Aqneursa (levacetylleucine)

Ataxia-telangiectasia

Also known as IB1001
Manufacturer
IntraBio
Last updated
206 sources

Product information and disease descriptionProduct descriptionProduct information

Generic, brand name and therapeutic class of product

7 evidence topics · 8 sources

Generic name

“AQNEURSA® (levacetylleucine) for oral suspension” (opens the prescribing information at this quote)

Drug nameActive ingredientsStrengthDosage form/routeMarketing status
“AQNEURSA”“LEVACETYLLEUCINE”“1GM/PACKET”“FOR SUSPENSION;ORAL”“Prescription”
Brand name

“AQNEURSA® is a registered trademark of IntraBio.” (opens the prescribing information at this quote)

Manufacturer: IntraBio

“Approval was granted to IntraBio Inc.” (opens the source at this quote in a new tab)

“Distributed by: IntraBio Inc., Austin TX 78701” (opens the prescribing information at this quote)

“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.” (opens the source at this quote in a new tab)

Development code and synonyms

“The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.” (opens the source at this quote in a new tab)

“We evaluated the safety and efficacy of N-acetyl-l-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, for the treatment of Niemann-Pick disease type C.” (opens the source at this quote in a new tab)

FieldQuoted record
Name of medicine“Aqneursa”
Active substance“L-acetylleucine”
International non-proprietary name (INN) or common name“L-acetylleucine”

“Aqneursa contains the active substance levacetylleucine.” (opens the source at this quote in a new tab)

Therapeutic class: modified amino acid

“AQNEURSA (levacetylleucine) for oral suspension contains the drug substance levacetylleucine, a modified amino acid.” (opens the prescribing information at this quote)

“AQNEURSA® (levacetylleucine) is a first-in-class, chemically modified amino acid approved by the FDA for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg and for ataxia in in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg.” (opens the source at this quote in a new tab)

“It is taken orally as a suspension.” (opens the source at this quote in a new tab)

Chemical description

“Levacetylleucine is slightly soluble in aqueous solutions.” (opens the prescribing information at this quote)

“The chemical name is 2-acetamido-4-methylpentanoic acid.” (opens the prescribing information at this quote)

“The empirical formula is C8H15NO3 and the molecular weight is 173.21.” (opens the prescribing information at this quote)

Enantiomer: distinction from N-acetyl-DL-leucine and N-acetyl-D-leucine

“Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine and N-acetyl-D-leucine.” (opens the prescribing information at this quote)

“The D-enantiomer, N-acetyl-D-leucine, competes with levacetylleucine for monocarboxylate transporter uptake, which may reduce the levacetylleucine efficacy.” (opens the prescribing information at this quote)