Biologics

Bluebird Sickle Cell Gene Therapy BLA Accepted

FDA has accepted for priority review a bluebird bio BLA for lovotibeglogene autotemcel (lovo-cel), a gene therapy for patients with sickle cell disease ages 12 years and older who have a history of vaso-occlusive events. The agency has set a user fee review action target date of 12/20.

The submission is based on data from 36 patients in the HGB-206 Group C cohort with a median 32 months of follow-up and two patients in the HGB-210 study with 18 months of follow-up each, according to the company. It also includes safety data from 50 patients treated across the entire lovo-cel program, including six patients with six or more years of follow-up. Previously, the agency granted the products an orphan drug designation, fast track designation, regenerative medicine advanced therapy designation, and rare pediatric disease designation.

The gene therapy is a one-time treatment that is designed to add functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells, the company says. Once patients have the targeted gene, their red blood cells can produce anti-sickling hemoglobin that can reduce sickled red blood cells, hemolysis, and other complications, 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