Drugs

Alnylam Pharmaceuticals Plans NDA for Patisiran

Alnylam Pharmaceuticals says it is planning an NDA submission late this year for patisiran, indicated for treating transthyretin-mediated amyloidosis with cardiomyopathy after reporting positive data from the APOLLO-B Phase 3 study. The study met the primary endpoint of change from baseline in the Six-Minute Walk Test at 12 months compared to placebo, according to the company. It also met the first secondary endpoint of change from baseline in quality of life compared to placebo, which was measured by the Kansas City Cardiomyopathy Questionnaire.

On the safety side, Alnylam says five patients (2.8%) on patisiran and eight patients (4.5%) on placebo died. “Furthermore, the number of deaths in the all-cause mortality efficacy analysis was 4 (2.2%) in the patisiran arm and 10 (5.6%) in the placebo arm, determined in accordance with the pre-defined statistical analysis plan, which excluded death due to Covid-19, and treated cardiac transplant as a death event consistent with other studies in the field,” it says. “The patisiran and placebo arms had similar frequencies of adverse events (91.2% and 94.4%, respectively) and serious adverse events (33.7% and 35.4%, respectively).”

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