About six months after Johnson & Johnson (J&J) returned rights to the compound, oral Tryvio (aprocitentan) won FDA clearance for Idorsia Ltd. Given once daily at a 12.5 mg dose, Tryvio is indicated for hypertension in combination with other blood pressure drugs in patients whose condition is not adequately controlled. Read More
Two days after the U.S. FDA announced approval of gene therapy Lenmeldy (atidarsagene autotemcel), making it the first treatment option for rare disease metachromatic leukodystrophy, Orchard Therapeutics and parent firm Kyowa Kirin Co. Ltd. disclosed the wholesale acquisition price of $4.25 million for the one-time treatment, which edges out hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) to become the world’s most expensive drug. Read More
After months of wrangling, the update of the EU pharmaceutical legislation passed an important milestone on March 19, when members of parliament on the health committee reconciled their opposing views and voted the file through. Read More
Although there’s bipartisan interest in the U.S. Congress to hold pharmacy benefit managers (PBMs) accountable for their contribution to the costliest drug prices in the world, the Biden administration ignored PBMs when it again focused on drug companies as the bad guys of pricing in its proposed 2025 budget. Read More
One of the building blocks for newly launched Clasp Therapeutics Corp. is making the right patient choices for treatment. If those who receive the company’s therapy are correctly identified, CEO Robert Ross told BioWorld, it will have a profound effect on outcomes. The missing link in cancer treatment, Ross added, was how to identify a patient, something he said Clasp is able to do. Read More
As part of its required series of guidances on using real-world evidence, the U.S. FDA released a draft guidance in response to sponsors’ growing interest in the potential use of observational studies to contribute to a demonstration of the effectiveness or safety of a drug or biologic. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Actitrexx, Alimera, Asieris, Axsome, Bayer, Bill & Melinda Gates Medical Research Institute, Cerus, Crinetics, Innovent, Inventiva, Jasper, Kiromic, Maggie’s Pearl, Merck, PMV, Seelos. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Boehringer Ingelheim, Cocrystal, Cosmo, CSL Vifor, Deverra, Forge, Hasten, Lib, Orchard, PTC, Revive. Read More
