Drugs

FDA Clinical Hold on 4D Molecular Fabry Drug

FDA has placed a clinical hold on 4D Molecular Therapeutics’ 4D-310 drug being studied for treating Fabry Disease Cardiomyopathy after the company reported three patients experiencing atypical hemolytic uremic syndrome, a condition that affects kidney function and causes blood clots to form. The company says that it stopped enrollment in the trial last month and that the program will be evaluated in the second half of this year after 12-month clinical data are collected on the s currently enrolled patients, including ongoing safety and cardiac endpoint assessments for a potential pivotal trial.

In communicating the hold, FDA told the company to continue long-term follow up of treated patients under the current IND, and noted that it would provide feedback on 4D-310 within 30 days.

Additionally, 4D Molecular reported improvements in all cardiac endpoints in patients (n=3) who reached 12 months follow up, and the “single available cardiac biopsy was positive for widespread genome delivery and transgene expression from 4D-310.” The company intends to present detailed safety, cardiac biopsy and cardiac efficacy data at the WORLD Symposium meeting on 2/25.

"4D-310 utilizes the targeted and evolved C102 vector to deliver a functional copy of the GLA gene and was designed for a unique dual mechanism of action after a single IV administration," the company says. "The product is designed to generate both high sustained blood levels of AGA for systemic cross-correction of tissues, as well as for a complementary high local production of AGA directly within critically affected organs, including heart, blood vessels and kidney."

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

Read more

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