In Ionis Pharmaceuticals Inc.’s second U.S. FDA approval in under a year, the agency approved Dawnzera (donidalorsen) as a prophylactic therapy in the rare and genetic disease hereditary angioedema (HAE). The approval came as scheduled as the NDA had a PDUFA date of Aug. 21. Dawnzera now joins a market with previously approved drugs for the rare, genetic, life-threatening condition, as well as other companies with HAE drugs in development. Read More
Showing similar results as the first phase III trial completed in 2022, Nicox SA’s nitric oxide-donating bimatoprost eye drop, NCX-470, met the primary endpoint in the phase III Denali trial for open-angle glaucoma or ocular hypertension, clearing the way for regulatory filings in both the U.S. and China. Read More
An investigation of the epidemiology and clinical characteristics of neuropathic pain in the UK Biobank has led to the discovery of a new pain gene and potential analgesic drug target in the peripheral nervous system. The gene, SLC45A4 (solute carrier 45A4), codes for a transporter that is involved in trafficking polyamines known to be involved in pain, across the cell membrane. Read More
No matter what level of tariff the Trump administration ends up imposing on biopharmaceuticals across the world, drugs and their precursors originating in the EU will face no more than a 15% tariff when imported to the U.S., according to an Aug. 21 EU-U.S. joint statement regarding the trade framework agreement between the two trade partners. Read More
South Korea’s Ministry of Food and Drug Safety (MFDS) launched a new industry-academia-research consortium to support the nation’s organoid industry, expected to grow in light of the U.S. FDA’s shift away from animal testing in the development of novel drugs. Read More
The BioWorld Biopharmaceutical Index (BBI) showed signs of recovery after hitting its lowest point of the year in late May, when it closed the month down 7.02%. While still negative overall, the index improved to -5.11% by the end of June and finished July down 3.57%. Similarly, the Nasdaq Biotechnology Index and the Dow Jones Industrial Average bottomed out at the end of May but posted stronger rebounds, closing July up 3.49% and 3.73%, respectively. Read More
Researchers at the Massachusetts Institute of Technology have developed a generative AI model that was able to generate novel antibiotic structures from either chemical fragments or de novo, starting from ammonia, methane, water or no starting point at all. In a study that was published online in Cell, the team tested two dozen of more than 10 million structures that were proposed as potential antibiotics by the model. Read More
The Medicare Payment Advisory Commission said in a recent report that Medicare spending in the U.S. is poised to nearly double to $1.9 trillion over the coming eight years, a figure that is likely to apply ever-increasing price pressures on makers of devices, diagnostics and pharmaceuticals. Read More
Biopharma happenings, including deals and partnerships, and other news in brief: ARC, Dong A-ST, Enanta, Jazz, Mural, Pfizer, Redhill, Saniona, Xoma, Xtalpi. Read More
Regulatory snapshots, including global drug submissions and approvals, and other regulatory decisions and designations: Madrigal, Rocket, Xspray. Read More
