Here are the latest stories being discussed in biopharma today:
The FDA has granted accelerated approval to Madrigal Pharmaceuticals’ NASH drug, resmetirom, also known as Rezdiffra. This marks the first approved treatment for the liver disease, anticipated to be available by April. The medication intends to treat patients with stage 2 and 3 fibrosis, though pricing details have not been disclosed yet.
Geron’s anemia treatment for specific myelodysplastic syndrome (MDS) patients, imetelstat, received a favorable vote from the FDA’s Oncologic Drugs Advisory Committee. Despite safety concerns regarding low levels of white blood cells or platelets, the committee voted 12 to 2 in favor of the drug, stating the risks seemed manageable. The drug met its primary endpoint in a Phase III trial, resulting in eight-week red blood cell-transfusion independence.
Chinese biopharmaceutical company, BeiGene, is establishing an additional API source outside of China and is about to open an $800 million biologics production facility in the US by July. BeiGene aims to lessen its reliance on China-based API manufacturers and in turn, expanding its manufacturing capacities to adhere to international standards.
The UK’s National Institute for Health and Care Excellence (NICE) is not prepared to recommend Vertex and CRISPR Therapeutics’ Casgevy for sickle cell disease, stating it requires further efficacy data, and clearer commercial benefits. Casgevy, also known as exa-cel, is the first approved treatment created with CRISPR gene editing.
Senate Finance Committee leaders urge the passing of reforms to pharmacy benefit managers (PBMs) before the March 22 deadline for the next round of appropriations bills. With unanimous support, the finance committee aims for greater regulation in PBMs practices, ensuring efficient patient care and minimized health expenses.
A recent Future of Healthcare Report by CMI Media Group predicts a gradual shift from mainstream TV to personalized and outcome-driven strategies for pharma advertising. Cited as an “attitudinal shift” across generations, more proactive and personalized approaches continue to gain traction in pharmaceutical marketing.
