Biologics

FDA Questions Safety/Efficacy of Bluebird’s Eli-Cel

FDA says it is challenged to determine the overall benefit-risk profile of Bluebird bio’s eli-cel (elivaldogene autotemcel) gene therapy, indicated for treating patients less than 18 years of age with early cerebral adrenoleukodystrophy who do not have an available and willing human leukocyte antigen-matched sibling hematopoietic stem cell donor. In an advisory committee briefing document released in advance of a 6/9 panel meeting to review the therapy, FDA says the benefit-to-risk ratio is “difficult to characterize because of the uncertain benefit and the uncertain magnitude of the risk of MDS” [myelodysplastic syndrome], a life-threatening malignancy which occurred in three subjects.

The advisory committee will be asked to discuss whether the efficacy data support the presence of a clinically meaningful benefit related to eli-cel, and, if so, in what population. Additionally, panel members will be asked to discuss whether there should be increased concerns about MDS occurrence in eli-cel because of similar events seen in the company’s related product, lovo-cel, for treating sickle cell disease, which was placed on clinical hold by FDA.

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