Aqneursa
(Levacetylleucine)PreclinicalSmall molecule
This medication is used to treat neurological symptoms in people with Niemann-Pick disease type C, a rare inherited disorder where fatty substances build up in the body and damage the nervous system and brain. The exact way it works is not fully understood, but it helps manage the nerve and brain-related symptoms of this disease.[1] Aqneursa is currently being investigated across 1 indication[3], with its most advanced program in Preclinical.[1]
Aqneursa is one of 7 small molecule programs targeting Ataxia-telangiectasia. It is currently in Preclinical.
1
Indications
1
Total Trials
0
Active Trials
6
Competitors
1
FDA Application
NDA/BLA/ANDA regulatory filings
3
Patents
Pipeline
Status by Indication[3][1]
Indication
Competition
Competitive Landscape
8 competitors in Ataxia-Telangiectasia
#DrugCompanyPhaseTrials
1Actos
TakedaApproved
Glucophage
EMD SeronoApproved
N-Acetyl-L-Leucine
IIntraBio Inc
Phase 3
Nicotinamide Riboside
UUniversity of Oklahoma
Preclinical
Prevenar
GSKPreclinical
Aqneursa
IIntraBio Inc
Preclinical
Pneumovax
NNational Institute of Allergy and Infectious Diseases (NIAID)
Preclinical
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Clinical
Trials by Indication[1]
Regulatory
FDA Approval Timeline[4]
Sep 24, 2024
AQNEURSA
NDA NDA219132
Sources
References
- [1]
- [2]
- [3]Open Targets Platform — Drug–disease associations for Levacetylleucine. EMBL-EBI & Wellcome Sanger Institute.
- [4]
- [5]
- [6]