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Denali Therapeutics Announces FDA Has Selected DNL126 (ETV:SGSH) for MPS IIIA (Sanfilippo Syndrome Type A) for START Pilot Program Intended to Accelerate Development of Rare Disease Therapies
START is a pilot program newly launched jointly by the FDA CDER and CBER divisions to further accelerate the development of novel drug and biological products
About this update from Denali Therapeutics Inc.
[{"type":"text","content":"START is a pilot program newly launched jointly by the FDA CDER and CBER divisions to further accelerate the development of novel drug and biological products for rare diseasesDNL126 was selected as one of three CDER-regulated products based on eligibility criteria including the potential for clinical benefit for a rare neurodegenerative disease and the drug sponsor’s ability to advance development towards a marketing application Participation in START is expected to facilitate and accelerate development of DNL126 through more rapid and ad hoc communication with FDA review staff SOUTH SAN FRANCISCO, Calif., June 03, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative and lysosomal storage diseases, today announced that the U.S. Food and Drug Administration (FDA) has selected DNL126 for participation in the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. DNL126 is an investigational enzyme replacement therapy designed to cross the BBB for the potential treatment of MPS IIIA (Sanfilippo syndrome type A). The FDA announced the START Pilot Program on September 29, 2023, with the stated purpose to further accelerate the pace of development of novel drug and biological products that are intended to address an unmet medical need as a treatment for a rare disease. Selected participants of the START Pilot Program are provided opportunities to obtain frequent advice and engage in more rapid ad hoc communication with FDA review staff to address product-specific development issues. The START pilot and metrics are milestone driven and agreed upon by the FDA and sponsor. Initial selection for START planned to include up to six eligible programs (three each) from the FDA’s Center for Biologics Evaluation and Research's (CBER) Office of Therapeutic Products and Center for Drug Evaluation and Research's (CDER) Office of New Drugs.1 “We are thrilled to be selected by the FDA for participation in START and see this as another important opportunity to work together to solve challenges unique to rare disease drug development,” said Carole Ho, M.D., Chief Medical Officer of Denali. “It is an exciting time to be part of the collective...