RARE Daily

FDA Grants 4D Molecular Therapeutics Fast Track Designation for Fabry Disease Gene Therapy

August 13, 2020

Rare Daily Staff

The U.S. Food and Drug Administration granted 4D Molecular Therapeutics Fast Track designation for its experimental gene therapy 4D-310 for Fabry disease, a debilitating lysosomal storage disorder.

Fabry disease is a rare, genetic disorder in which a deficiency of the lysosomal α‑galactosidase‑A enzyme results in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls throughout a person’s body. The abnormal storage of Gb3 increases with the ultimate consequences of Gb3 deposition ranging from episodes of pain and impaired peripheral sensation to end-organ failure – particularly of the kidneys, but also of the heart and the cerebrovascular system.

4D-310 holds promise for the treatment of Fabry by using a proprietary and optimized AAV vector to deliver a functional copy of the GLA gene, resulting in AGA production in target tissues. The proprietary vector in 4D-310 is designed for efficient, low-dose intravenous delivery to key affected tissues in Fabry disease, including cardiac tissue, kidney, and vascular smooth muscle tissues.

“We’re pleased that the FDA has granted Fast Track designation for 4D-310,” said David Kirn, co-founder, chairman and CEO of 4DMT. “Patients with Fabry currently have significant unmet medical needs, despite enzyme replacement therapies (ERT). In contrast to ERT and other medicines in development, 4D-310 is designed to drive high level AGA enzyme expression directly within the diseased target tissues themselves, including the heart, kidney and blood vessels, in addition to driving high and stable blood concentrations. We believe this differentiation has the potential to benefit a broad range of patients with Fabry disease, including those with significant cardiomyopathy, the leading cause of death in these patients.”

The FDA’s Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.

Photo: David Kirn, co-founder, chairman and CEO of 4DMT

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