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US FDA Approves IntraBio Aqneursa to Treat Niemann-Pick Disease Type C

28 Sep 2024
Admin
News Article

US FDA Approves IntraBio Aqneursa to Treat Niemann-Pick Disease Type C

IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, announced that the US Food and Drug Administration (FDA) has approved Aqneursa (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and paediatric patients weighing =15 kg. Aqneursa is the only FDA-approved stand-alone therapy indicated for the treatment of NPC.

Satemet from the CEO: IntraBio

“IntraBio has been dedicated to bringing novel treatments to patients with extremely high unmet medical needs like NPC, and today we celebrate a major milestone in this tremendous effort,” said Mallory Factor, president and chief executive officer of IntraBio.
“Patients and families in the NPC community have long awaited an effective, FDA-approved treatment, and we are proud to bring hope to those affected by this devastating disease. We remain committed to ensuring that all patients who can benefit from this novel treatment will have the opportunity to do so. Based on our clinical research, we believe that Aqneursa may hold potential for treating other rare and common neurodegenerative and neurodevelopmental disorders, and we will continue to rapidly develop Aqneursa for these additional indications.”

About NPC

NPC is a rare, inherited lysosomal disease that occurs in about 1 in 100,000 live births.
Patients with NPC typically experience systemic, neurological and psychiatric symptoms that can be debilitating and significantly impact functional abilities.
Until now, current treatment approaches have not addressed the debilitating effects of NPC on patients’ daily lives.

From National Niemann-Pick Disease Foundation

“The FDA approval of Aqneursa marks a significant breakthrough for those living with Niemann-Pick disease type C,” commented Laurie Turner, Family Services Manager at the National Niemann-Pick Disease Foundation.
“For too long, our community has been without an approved therapy for the treatment of NPC. Today we celebrate this tremendous milestone for individuals and families living with NPC. We are immensely thankful for the dedication to innovative research that has led to this approval, and we are ready to help families embark on this new chapter of treatment.”

Data Behind the Approval

The FDA approval is based on data from the IB1001-301 multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial (NCT05163288).
It evaluated the impact of Aqneursa on neurological symptoms and functioning in paediatric (aged 4 years and older) and adult patients (n=60) with a confirmed diagnosis of NPC.

Primary Efficacy Endpoint

The trial met the primary efficacy endpoint and all secondary endpoints across all cohorts receiving Aqneursa.
Results from the study showed Aqneursa significantly improved neurological signs and symptoms and demonstrated functional benefits important to everyday life that were evident within 12 weeks.
These findings were published in the February 1, 2024 issue of the New England Journal of Medicine.

About Primay Outcomes

The primary outcome assessed by the FDA was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (fSARA).
SARA is a clinical assessment tool that assesses gait, stability, speech, and upper and lower limb coordination across eight individual domains.
fSARA consists only of gait, sitting, stance, and speech disturbance domains of the original SARA with modifications to the scoring responses.
The results in patients who received Aqneursa compared to placebo showed a greater improvement in fSARA score with a mean treatment difference of -0.4 (95% CI: -0.7, -0.2) with a two-sided p-value of <0.001.
Results on the fSARA were supported by consistent results demonstrated on the original SARA.

Aqneursa Tolerance

Aqneursa was well tolerated in the trial with the most common adverse reactions (incidence =5% and greater than placebo in Period I of the trial) being abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.

Aqneursa: Indication

Aqneursa (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and paediatric patients weighing =15 kg.

IntraBio’s Support Programs

IntraBio offers support programs to eligible patients through their Patient Support Service, Aqneursa Cares.
This program includes financial support to reduce or eliminate out-of-pocket costs for qualifying patients and also connects patients with third-party resources.
Aqneursa Cares includes access to financial and educational resources and a dedicated team of specialists.
The team is available to help with individuals’ unique challenges including starting treatment, questions about taking the medication, and navigating insurance coverage.
Contact 866-200-0419 to speak to an Aqneursa Cares team representative to seek assistance with any questions or concerns about access to Aqneursa.

About the Company: IntraBio Inc.

IntraBio Inc., a US biopharmaceutical company, is focused on the development of novel drugs addressing rare and common neurological diseases.
IntraBio’s platform technologies result from decades of research and collaboration with universities and institutions worldwide.