AQNEURSA
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.
AQNEURSA is the only FDA-approved stand-alone therapy for the treatment of NPC and is available now.
For more information visit aqneursa.com
AQNEURSA Cares Program Information
Latest News
U.S. FDA approves AQNEURSA™ (IB1001) for the Treatment of NPC
We are pleased to share the U.S. Food and Drug Administration (FDA) has approved has AQNEURSA™ for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.
Read IntraBio Letter to the NPC Community
Read IntraBio Press Release
Read FDA Press Release
09/25/2024
Update from IntraBio: IntraBio is pleased to announce that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease Type C (NPC). The application has been granted Priority Review and was given a Prescription Drug User Fee Act (PDUFA) target action date of September 24th, 2024.
Read the complete announcement.
03/26/2024
Update from IntraBio: IntraBio is pleased to share that The New England Journal of Medicine (NEJM) has published a “Science Behind the Study” Expert Perspective Editorial on N-acetyl-L-leucine (IB1001) for the treatment of various neurodegenerative disorders, including all neurodegenerative lysosomal storage diseases.
Read the complete announcement.
Read the published article.
02/07/2024
Update from IntraBio: IntraBio is pleased to share that The New England Journal of Medicine (NEJM) has published the detailed results of the IB1001-301 Phase 3, Pivotal study with N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC).
Read the complete announcement.
Read the published article.
02/1/2024
Update from IntraBio: IntraBio Inc. is pleased to share that the New Drug Application (NDA) for N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease type C (NPC) was submitted to the US Food and Drug Administration (FDA) in January 2024. Read the complete update.
01/31/2024
IntraBio Announces Positive Pivotal Trial Results of IB1001 for the Treatment of Niemann-Pick Disease Type C. Read the complete announcement.
06/29/2023
IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301). Read the complete announcement.
12/05/2022
IntraBio Ltd. is pleased to share that IB1001-301 has enrolled over 70% of the target number of patients in the second month of recruitment. Recruitment is expected to be completed by December 2022. Read the complete announcement.
11/07/2022
IntraBio Ltd. is pleased to confirm the Phase III pivotal trial with N-acetyl-L-leucine (IB1001-301) is active and recruiting in the United States at the Mayo Clinic, MN. Read the complete announcement.
10/26/2022
IntraBio Reports Further Detail on Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C. Click here for complete announcement.
10/19/2020
IntraBio Reports Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C. Click here for complete Announcement.
09/30/2020
IntraBio Receives Fast Track Designation from FDA for IB1001 Treatment of NPC. Click here for complete Announcement.
03/25/2020
IntraBio Announces Extension Phase for NPC Accepted in the United States. Click here for complete Announcement.
03/05/2020
IntraBio Completes NPC Clinical Trial Enrollment. Click here for complete announcement.
01/17/2020
IntraBio IB1001-201 Clinical Trial for NPC Reaches 2/3 of their Recruitment Goal. Click here for complete announcement.
11/13/2019
IntraBio Clinical Trial IB1001-201 Open for US Recruitment at Mayo Clinic. Click here for complete announcement.
09/04/2019
IntraBio IB1001 Series Update
IntraBio is developing a number of small-molecule drug candidates that have broad applicability to rare and common neurological disorders, genetic, and inflammatory diseases. Their current lead drug candidate, IB1001, is initially being developed to treat three rare, genetic diseases where there are extremely high unmet medical needs: Niemann-Pick disease type C, GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and Ataxia-Telangiectasia (A-T; an inherited Cerebellar Ataxia). Click the link below for additional information.
Click here for IB1000 Series development history.
05/31/2019
NNPDF Industry Update Webinar Series featuring IntraBio, Inc. Click here for recording.
04/25/2019
IntraBio Clinical Trial Authorization Application Approved by the MHRA for the Treatment of NPC. Click here for complete announcement.
03/11/2019
IntraBio Investigational New Drug Application Approved by the FDA for the Treatment of NPC. Click here for complete announcement.
02/13/2019
IntraBio Receives Niemann-Pick disease Orphan Drug Designation from the FDA for IB1000 Series. Click here for complete announcement.