Sept 17 (Reuters) – The U.S FDA said on Thursday it has approved Ultragenyx’s gene therapy for the treatment of a rare, fatal disease. The approval makes Fayuvi the first therapy for Sanfilippo syndrome Type A, caused by a missing enzyme that stops the body from breaking down a complex sugar molecule called heparan sulfate. […]
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US FDA approves Ultragenyx’s gene therapy for rare disorder
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Sept 17 (Reuters) – The U.S FDA said on Thursday it has approved Ultragenyx’s gene therapy for the treatment of a rare, fatal disease.
The approval makes Fayuvi the first therapy for Sanfilippo syndrome Type A, caused by a missing enzyme that stops the body from breaking down a complex sugar molecule called heparan sulfate. Ultragenyx shares were up nearly 6% in afternoon trading.
The therapy is designed to address the underlying sulfamidase (SGSH) enzyme deficiency responsible for abnormal accumulation of heparan sulfate in the brain that results in progressive cell damage and neurodegeneration.
The U.S. Food and Drug Administration had declined to approve the therapy last year citing manufacturing concerns.
(Reporting by Sriparna Roy and Kamal Choudhury in Bengaluru; Editing by Maju Samuel and Shilpi Majumdar)