Biologics

BEAM-101 Gets Advanced Therapy Designation

FDA has granted Beam Therapeutics a Regenerative Medicine Advanced Therapy designation for BEAM-101, an investigational gene-edited cell treatment for sickle cell disease. The designation follows the agency’s orphan drug status awarded in June for the therapy.

Updated clinical data from the BEACON Phase 1/2 clinical trial of BEAM-101 were presented in June at the European Hematology Association Congress showing that 17 patients treating with BEAM-101 experienced sustained increases in fetal hemoglobin (HbF) and decreases in sickle hemoglobin, along with improved markers of blood health, according to the company. No vaso-occlusive crises occurred after patients’ blood cells recovered, it adds. Beam plans to present additional BEACON trial data by the end of 2025.

BEAM-101 uses base editing to modify a patient’s own stem cells, increasing production of HbF, which resists sickling, BEAM says. The therapy aims to mimic naturally occurring genetic variants that protect against the disease.

Sickle cell disease, the most common inherited blood disorder in the U.S., affects about 100,000 Americans and roughly eight million people worldwide, according to the company.

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