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 12 small molecule programs targeting Ataxia-telangiectasia. It is currently in Preclinical.
1
Indications
1
Total Trials
0
Active Trials
11
Competitors
1
FDA Application
NDA/BLA/ANDA regulatory filings
3
Patents
Pipeline
Status by Indication[3][1]
Indication
Competition
Competitive Landscape
20 competitors in Ataxia-Telangiectasia
#DrugCompanyPhaseTrials
1Actos
TakedaApproved
Glucophage
EMD SeronoApproved
Amantadine Sulphate
SSheba Medical Center
Approved
Lioresal
NovartisPhase 1
N-Acetyl-L-Leucine
IIntraBio Inc
Phase 3
IB1001
MMedexus Pharma, Inc.
Phase 2
Conjugated Pneumococcal Vaccine
IInstitute of Child Health
Phase 3
Decadron
QQuince Therapeutics S.p.A.
Preclinical
Prevenar
GSKPreclinical
Nicotinamide Riboside
UUniversity of Oklahoma
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]
Details
Drug Info
Quick Facts
- Modality
- Small molecule[2]
- Highest Phase
- Preclinical[1]
- Company
- IIntraBiotics Pharmaceuticals