Rare Disease Report

EMA Grants Orphan Medicinal Product Designation to SB-525 for Hemophilia A

JUNE 07, 2017
Mathew Shanley
Earlier today, it was announced by Sangamo Therapeutics, Inc. and Pfizer, Inc. that their clinical stage cDNA gene therapy candidate for hemophilia A, SB-525, was granted orphan medicinal product designation (OMPD) by the European Medicines Agency (EMA).
Hemophilia A, a monogenic, rare bleeding disorder in which the blood does not clot normally, is caused by mutations in the F8 gene. The gene encodes Factor VIII clotting protein that helps the blood clot and stop bleeding when blood vessels are injured. Per the Centers for Disease Control and Prevention, hemophilia affects an estimated 1 in every 5,000 male births, with an estimated 20,000 males in the U.S. living with the disorder.
Typically, those with this particular mutation in the F8 gene experience bleeding episodes after injury, and impulsive bleeding episodes that can lead to additional joint disease and disorders, like arthritis.
SB-525, which was granted Fast Track Designation and Orphan Drug Designation by the U.S. Food and Drug Administration in May, is a single infusion therapy that delivers a human Factor VIII cDNA construct and proprietary, synthetic liver-specific promoter to the nucleus of liver cells by way of a recombinant adeno-associated virus (rAAV).
An Investigational New Drug application for this program was also cleared by the FDA, and a Phase 1/2 clinical trial assessing SB-525 in adult hemophilia A patients is now open and screening subjects for enrollment.
Initial data from this study are anticipated in the last quarter of 2017 or first quarter of 2018. Under the collaboration and license agreement between the Sangamo and Pfizer, the latter company will be responsible for any following clinical trials and the commercialization of SB-525.

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