IB1001 FDA Approval Status

Last updated by Judith Stewart, BPharm on April 10, 2024.

FDA Approved: No
Brand name: IB1001
Generic name: N-acetyl-L-leucine
Company: IntraBio Inc.
Treatment for: Niemann-Pick Disease

IB1001 (N-acetyl-L-leucine) is a modified amino acid in development for the treatment of Niemann-Pick disease type C.

• Niemann-Pick disease type C (NPC) is a rare autosomal recessive neurodegenerative lysosomal disease characterized by multiple symptoms such as progressive cerebellar ataxia and cognitive decline.
• IB1001 contains the modified amino acid N-acetyl-leucine which has been associated with positive symptomatic and neuroprotective disease-modifying effects in NPC.
• The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for IB1001 for the treatment of NPC and the application has been granted Priority Review.  The NDA is based on the results of a Pivotal Phase 3 trial (IB1001-301) that met all primary and key secondary endpoints and showed IB1001 improved neurological signs and symptoms, functioning, and quality of life versus placebo. The NDA also included data from a positive multinational Phase IIb trial of IB1001 for NPC, which also met its primary and secondary endpoints and showed improvement in symptoms, functioning, and quality of life, with the drug being well-tolerated.
• The Prescription Drug User Fee Act (PDUFA) target action date is September 24, 2024.

Development timeline for IB1001

Further information

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