Tuesday, June 18, 2024

Sanfilippo Syndrome Treatment NP3011 Receives FDA Fast Track Designation

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Novel Pharma, in collaboration with GC Green Cross, announced on the 10th that their jointly developed treatment for Sanfilippo syndrome type A (MPS IIIA), NP3011 (also known as GC1130A), has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA).

The FDA’s Fast Track program is designed to expedite the development and review of drugs that address serious conditions and fulfill an unmet medical need. With this designation, NP3011 will benefit from more frequent meetings with the FDA throughout the development process, along with various forms of support to accelerate its development.

NP3011 Targets Severe and Rare Genetic Disorder Sanfilippo Syndrome, Begins Phase 1 Trials

Sanfilippo syndrome is a severe and rare genetic disorder caused by a deficiency in the enzyme needed to break down heparan sulfate, leading to its accumulation and progressive damage to the central nervous system (CNS). Currently, there are no approved treatments for this condition, highlighting a significant unmet medical need. NP3011 aims to address this by directly delivering the deficient enzyme to the CNS through intraventricular injection (ICV).

Previously, the FDA approved Novel Pharma’s Investigational New Drug (IND) application for NP3011 in May, allowing the commencement of Phase 1 clinical trials. These trials will evaluate the safety and tolerability of NP3011 in patients with Sanfilippo syndrome. Given the rarity of the condition, the trials are expected to proceed quickly due to the limited number of patients.

Sanfilippo Syndrome

Novel Pharma Accelerates Development of First Sanfilippo Syndrome Treatment with FDA Fast Track Designation

A Novel Pharma representative stated, “With the FDA’s Orphan Drug Designation (ODD), Rare Pediatric Disease Designation (RPDD), and now Fast Track designation, we can accelerate the development of the world’s first treatment for Sanfilippo syndrome. We are preparing for multinational clinical trials, starting with Phase 1 in the U.S., and extending to Korea and Japan.”

In February, Novel Pharma presented promising preclinical results for NP3011 at the 2024 WORLD Symposium in San Diego. The results demonstrated significant reductions in heparan sulfate accumulation and improvements in brain lesions in a mouse model of Sanfilippo syndrome, garnering considerable attention. This series of designations and the ongoing clinical trials mark significant milestones in the development of NP3011, offering hope for effective treatment options for patients suffering from this debilitating condition.

 

Resource: Novel Pharma, June 10, 2024

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