The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application for IB1001 for the treatment of Niemann-Pick disease type C (NPC).

IB1001 is an orally administered, modified amino acid (N-acetyl-L-leucine) with a multimodal mechanism of action. The application is supported by data from the phase 3 IB1001-301 trial (ClinicalTrials.gov Identifier: NCT05163288), which evaluated the efficacy and safety of IB1001 in patients 4 years of age and older with NPC (N=60). Study participants were randomly assigned 1:1 to receive IB1001 for 12 weeks followed by placebo for 12 weeks, or placebo for 12 weeks followed by IB1001 for 12 weeks.

Mean baseline Scale for the Assessment and Rating of Ataxia (SARA) total scores were 15.88 and 15.68 prior to receiving the first dose of IB1001 and placebo, respectively. The primary endpoint was the change in SARA total scores, with lower scores indicating improved neurologic status.

Results showed the mean change from baseline in the SARA total score was -1.97 ± 2.43 points after 12 weeks of receiving IB1001 compared with -0.60 ± 2.39 points after 12 weeks of receiving placebo (least-squares mean difference, -1.28 points; [95% CI, -1.91, -0.65]; P <.001). Treatment with IB1001 also improved neurological signs and symptoms, functioning, and quality of life compared with placebo (key secondary endpoints).

“The FDA’s acceptance of IntraBio’s NDA submission for IB1001 and granting of Priority Review brings us one step closer to our ultimate goal of delivering new, effective treatments to patient communities like NPC who have extremely high unmet medical needs,” said Mallory Factor, IntraBio’s Executive Chairman. “IntraBio remains dedicated to advancing therapies and addressing unmet medical needs, and we are optimistic IB1001 will be approved and made rapidly available for all NPC patients.”

N-Acetyl-L-Leucine Granted Priority Review for Niemann-Pick Disease Type C

Want to read more?