Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec). Liver injury is a known possible side effect of the product, first approved by the U.S. FDA in June 2023 for DMD, as well as other AAV-mediated gene therapies, and the potential problem is highlighted in Elevidys’ prescribing information. Read More
Leading pharma companies have pitched into Sofinnova Partners’ new accelerator fund, which has exceeded the target and closed at €165 million (US$180 million). In what is said to be the largest pan-European biotech accelerator fund, Amgen Inc., Bristol Myers Squibb Co. and Pfizer Ventures will get an inside track on startups formed around academic research, as the nascent companies are shaped up for formal investment rounds. Read More
The U.S. foreign aid cuts and freezes that are taking place under President Donald Trump are putting at risk the global public health gains that have been made against diseases such as malaria, HIV and tuberculosis over the past two decades, World Health Organization (WHO) Director-General Tedros Adhanom Ghebreyesus said at a March 17 media briefing, as he called on other countries to step up and fill the gap. Read More
China’s National Medical Products Administration (NMPA) approved the country’s first injectable teprotumumab biosimilar from Innovent Biologics Inc. under the brand name of Sycume (teprotumumab N-01) to treat thyroid eye disease on March 14. The NMPA on March 15 accepted Simcere Pharmaceutical Group Ltd.’s NDA of deunoxavir marboxil (ADC-189) tablets, a product originally developed by Jiaxing Andicon Biotech Co. Ltd. to treat influenza A and B. Read More
Japanese-founded Regcell Inc. has raised $45.8 million and is redomiciling to the U.S. to accelerate clinical development of its pioneering regulatory T-cell (Treg) platform for autoimmune diseases and transplantation. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Allarity, Astrazeneca, Avidity, Capricor, Dyne, Greenwich, Incyte, Orca, Sutro, Zentalis. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Astrazeneca, Bayer, Grin, Idorsia, Nurix, Pep-Therapy, Travere, Vtv, Xeris. Read More
