Evicenter
P&T meetings

Aqneursa (levacetylleucine)

Ataxia-telangiectasia

Also known as IB1001
Manufacturer
IntraBio
Last updated
206 sources

Product information and disease descriptionProduct descriptionProduct information

Indication

4 evidence topics · 4 sources

U.S. indication: ataxia in ataxia-telangiectasia

“AQNEURSA is indicated for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg.” (opens the prescribing information at this quote)

“This Prior Approval sNDA provides for use of Aqneursa as a treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia weighing ≥ 15 kg.” (opens the source at this quote in a new tab)

U.S. indication: signs and symptoms that ataxia comprises

“Ataxia may be comprised of multiple signs and symptoms, including gait ataxia, truncal ataxia, oculomotor ataxia, and difficulties with balance, speech, and hand coordination.” (opens the prescribing information at this quote)

U.S. indication: Niemann-Pick disease type C

“AQNEURSA® is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.” (opens the prescribing information at this quote)

European Union therapeutic indication and status of the A-T extension

“Aqneursa is indicated for the treatment of neurological manifestations of Niemann-Pick type C (NPC) disease, in combination with miglustat, or as a monotherapy in patients where miglustat is not tolerated, in adults and children aged 6 years and older and weighing at least 20 kg.” (opens the source at this quote in a new tab)

“C.6: Extension of indication to include treatment of Ataxia-telangiectasia for AQNEURSA based on results from study IB1001-303.” (opens the source at this quote in a new tab)