Drugs

BrainChild Breakthrough Status for Brain Tumor Therapy

FDA has granted BrainChild Bio a breakthrough therapy designation for its investigational CAR T-cell therapy, BCB-276, for treating diffuse intrinsic pontine glioma (DIPG), a rare and fatal pediatric brain tumor. The company says the designation was granted based on encouraging clinical data from the Phase 1 BrainChild-03 trial (NCT04185038) that was conducted at Seattle Children’s Hospital. The study, recently published in Nature Medicine, showed a promising survival benefit in patients treated with the autologous B7-H3-targeting CAR T-cell therapy, it says.

DIPG affects roughly 300 children annually in the U.S., most of them between ages five and 10 years, BrainChild says. The condition currently has no cure and a median survival time of just 11 months following diagnosis. Existing treatments, like focal radiation, are palliative rather than curative, it says.

CAR T-cell therapy offers a new approach by using a patient’s own immune cells to target cancer. BrainChild Bio’s method involves delivering engineered T-cells directly into the cerebrospinal fluid, bypassing the blood-brain barrier and allowing direct access to the tumor, according to the company.

BrainChild says it is planning to launch soon a pivotal Phase 2 multi-center trial to support eventual FDA approval of BCB-276 through a BLA.

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