Biologics

Vertex Pharma Gets 2nd OK for Gene Therapy

FDA has approved a Vertex Pharmaceuticals BLA for Casgevy (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy for treating transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. This is the second approval granted for Casgevy; the first approval was granted 12/9/2023 for treating sickle cell disease (see story).

The company says that because administering Casgevy requires experience in stem cell transplantation, it is working with experienced hospitals to establish a network of authorized treatment centers (ATCs). Currently, nine such ATCs are able to offer the cell therapy to eligible patients with TDT and sickle cell disease.

TDT is described as a serious, life-threatening genetic disease that requires frequent blood transfusions and iron chelation therapy throughout a person’s life. “TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity,” the company 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