Drugs

FDA Gives Orchard ‘Advanced’ Status for Gene Therapy

FDA has granted Orchard Therapeutics a Regenerative Medicine Advanced Therapy designation for the company’s ex vivo autologous hematopoietic stem cell-based gene therapy (OTL-103) for treating Wiskott-Aldrich Syndrome (WAS). The designation was based on data from an interim analysis of a registrational trial in patients with severe WAS. The data show that the gene therapy assisted with the “recovery of the immunological and platelet abnormalities associated with WAS, with a consequent significant reduction in the major complications of the disease,” the company says. “Following treatment with OTL-103 gene therapy, patients with follow-up ranging from 0.5 to 5.6 years showed a decrease in the frequency of severe infections. In addition, severe bleeding episodes were eliminated and moderate bleeding episodes were greatly reduced.”

WAS is a life-threatening inherited immune disorder characterized by autoimmunity, eczema, abnormal platelet number and function. The company says WAS manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. “Without treatment, the median survival for WAS patients is 14 years of age and treatment with stem cell transplant carries significant risk of mortality and morbidities,” it says.

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