New Drug Approved for Gaucher Disease

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An inherited metabolic disorder

TUESDAY, Aug. 18 (HealthDay News) -- A new drug can be used to treat patients with a rare genetic disorder called Gaucher disease before the drug gets full marketing approval, according to the U.S. Food and Drug Administration.

The Associated Press reported that the agency approved a treatment protocol that allows Protalix BioTherapeutics Inc. to give prGCD to patients in a clinical trial during a shortage of an older drug called Cerezyme, made by Genzyme Corp. That company had to discard most of its ingredients for Cerezyme after an FDA inspection of the manufacturing facility.

Under the treatment protocol, the patients in the clinical trial will receive prGCD for free until the drug receives full FDA approval, the AP reported.

Gaucher disease can cause liver and neurological problems.

More information

To learn more about Gaucher disease, visit the U.S. National Institute of Neurological Disorders and Stroke.



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