IntraBio Inc announced today that the U.S. Food and Drug Administration has accepted its New Drug Application (NDA) for IB1001, intended for treating Niemann-Pick disease Type C (NPC). The application has received Priority Review and is expected to undergo action by September 24th, 2024, under the Prescription Drug User Fee Act (PDUFA).

The NDA's acceptance follows positive results from the Pivotal Phase 3 trial (IB1001-301), involving adult and pediatric NPC patients. This trial successfully met primary and key secondary endpoints, demonstrating significant improvements in neurological signs and symptoms, functioning, and overall quality of life compared to a placebo. These findings were published in the New England Journal of Medicine on February 1st, 2024.

Additionally, the NDA included data from a multinational Phase IIb trial of IB1001 for NPC, which also met primary and secondary endpoints, showing improvements in symptoms, functioning, and quality of life in pediatric and adult patients. This trial indicated that the drug was well-tolerated.

Sean Kassen, Director of the Ara Parseghian Medical Research Fund, expressed optimism about IB1001's potential to address the critical medical needs of NPC patients, emphasizing its potential to improve their quality of life.

Mallory Factor, Executive Chairman of IntraBio, highlighted the significance of FDA's acceptance of the NDA for IB1001, affirming the company's commitment to addressing unmet medical needs and delivering effective treatments to patients.

Professor Elizabeth Berry-Kravis from Rush University Medical Center welcomed the news, anticipating the availability of new treatment options for NPC patients and expressing intentions to administer IB1001 to eligible patients, closely monitoring their long-term progress.

In conjunction with this development, IntraBio announced the closure of an equity financing round, securing over $40 million US, to support the commercialization and launch of IB1001 upon FDA approval.

IB1001, an orally administered therapy, utilizes monocarboxylate transporters for uptake, facilitating its distribution across tissues, including the blood-brain barrier. Its multi-modal mechanism of action targets key disease drivers in both rare and common neurological disorders, offering promising therapeutic potential.

 

Source: accesswire.com

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