Aug 19 (Reuters) – The U.S. Food and Drug Administration approved Ultragenyx Pharmaceutical’s gene therapy to treat a rare metabolic disorder, the company said on Wednesday.
The drug, branded as Genglycos, is approved for glycogen storage disease type Ia (GSDIa), also known as Von Gierke disease, a rare genetic disorder that impairs the body’s ability to release glucose from stored glycogen, causing dangerously low blood sugar levels.
Ultragenyx’s Genglycos is the first FDA-approved therapy to address the underlying cause of the inherited disorder, which affects 1 in 100,000 infants in the United States, according to National Institutes of Health data.
Several experimental treatments are also in development, including those from Moderna and Beam Therapeutics, though the candidates remain in early stages of clinical testing.
(Reporting by Padmanabhan Ananthan and Sneha S K in Bengaluru; Editing by Leroy Leo and Shinjini Ganguli)
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