Trending News Today: Gene-Silencing Drug Raises Concerns About Safety in Late-Stage Trial

New late-stage trial data on Alnylam’s gene-silencing drug, givosiran, sparked concerns about its safety despite the treatment’s efficacy, Reuters reported. According to the article, givosiran met the main goal of reducing the yearly number of attacks in patients with acute intermittent porphyria (AIP), but many patients experienced serious adverse effects, such as renal impairment and elevated liver enzymes in the trial. The study included patients with acute hepatic porphyria, a family of rare diseases that affects the liver, with the most common subtype being AIP.

The health care company formed by Amazon, Berkshire Hathaway, and JP Morgan Chase & Co. will be named Haven and will focus on priorities such as improving access to primary care, lowering prescription drug costs, and simplifying insurance coverage, STAT reported. The company also launched its website, which describes its intentions to use new technologies and data analysis to improve the health care system. The company will operate as a nonprofit focused on the employees of its 3 founders, but said it hopes to eventually create models to benefit other companies and patients, the article reported.

The resignation of FDA Commissioner Scott Gottlieb, MD, has raised questions as to whether his ambitious policy proposals, such as the crackdown on youth vaping, will continue after he steps down, The Hill reported. According to the article, Gottlieb has proposed actions aimed at curbing the youth vaping epidemic, including limiting in-store sales of flavored e-cigarettes, but no guidances have been released yet. Gottlieb told The Hill that he’s confident the FDA will continue with this policy over the next month and that the agency will remain focused on the youth vaping issue after his departure, the article reported.