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Denali Therapeutics Announces Fast Track Designation Granted by the U.S. FDA to ETV:IDS (DNL310) for the Treatment of Patients with Hunter Syndrome (MPS II)
SOUTH SAN FRANCISCO, Calif., March 11, 2021 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad
About this update from Denali Therapeutics Inc.
[{"type":"text","content":"SOUTH SAN FRANCISCO, Calif., March 11, 2021 (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 neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ETV:IDS (DNL310) for the treatment of patients with Hunter syndrome (MPS II). DNL310, Denali’s lead investigational brain-penetrant enzyme replacement therapy, is under evaluation in a Phase 1/2 study in patients with Hunter syndrome as a potential treatment for both central nervous system (CNS) and peripheral manifestations of the disease. “Hunter syndrome is a devastating disease for which current approved treatments fail to effectively cross the blood-brain barrier and therefore do not address CNS symptoms,” said Carole Ho, M.D., Denali’s Chief Medical Officer. “Using Denali’s Transport Vehicle technology, we have designed DNL310 to treat both body and brain in Hunter syndrome after intravenous administration. Receiving Fast Track designation is an important milestone in the DNL310 development program, and we look forward to our continued collaboration with the FDA to bring safe and effective treatments to patients with Hunter syndrome.” Fast Track is an FDA process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Denali expects that Fast Track designation may allow for early and frequent communication with the FDA regarding the development of DNL310 for the treatment of Hunter syndrome. This designation also enables rolling review, and potentially priority review, of the marketing application. About the DNL310 Development Program for the Potential Treatment of Hunter Syndrome (MPS II)Hunter syndrome (MPS II) is a rare neurodegenerative lysosomal storage disorder caused by a mutation in the gene that encodes for the enzyme iduronate-2-sulfatase (IDS). The resultant reduction or loss of IDS enzyme activity leads to accumulation of glycosaminoglycans (GAGs), which causes lysosomal dysfunction and neurodegeneration as well as progressive damage to multiple organs including bone, cartilage, heart and lung. Current standard of care enzyme replacement treatment does not address neuronopat...