Drugs

Ultragenyx Gene Therapy OK’d for Glycogen Storage Disease

FDA has granted Ultragenyx Pharmaceutical accelerated approval for its Genglycos (pariglasgene brecaparvovec-opnr) for adults and children eight years and older with glycogen storage disease type Ia (GSDIa). The action marks the first gene therapy approval for Ultragenyx and the company’s fifth FDA approval overall. Ultragenyx also received an FDA priority review voucher for gaining the approval.

Genglycos is indicated to reduce daily cornstarch intake as an adjunct to nutritional management in patients with GSDIa. The indication was approved based on the drug’s demonstrated ability to reduce daily cornstarch requirements, and continued approval is contingent on verification of clinical benefit in a confirmatory study.

The genetic metabolic disorder is caused by a deficiency of an enzyme required for the liver to release glucose into the bloodstream. The resulting inability to adequately regulate blood glucose can cause potentially life-threatening hypoglycemia and other complications, the company says.

As part of the approval, Ultragenyx says it has “agreed to provide two years of safety and efficacy clinical data from open-label commercial treatment of 50 patients and 20 control patients through enhancement of its existing GSDIa Disease Monitoring Program (DMP). The control group will consist of patients who sought commercial treatment but cannot be treated with Genglycos due to the presence of anti-AAV8 antibodies.”

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome