Recognizing the potential legal challenges to U.S. President Donald Trump’s executive order calling for most-favored-nation (MFN) prescription drug pricing and the limits of that order, several congressional Democrats introduced a bill in both the House and Senate May 14 that could make MFN pricing the law of the land and extend it to both government health programs and private insurance.
Just two months after Inozyme Pharma Inc. cut its workforce by 25% and prioritized activities to focus on a BLA filing for INZ-701 for ENPP1 deficiency, interim phase III data from its Energy 3 trial showed consistent safety and immunogenicity and increased phosphate levels in patients treated with the rare disease enzyme replacement therapy.
Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.
The U.S. FDA granted Abbvie Inc. accelerated approval for antibody-drug conjugate (ADC) Teliso-V (telisotuzumab vedotin), newly branded Emrelis, making it the first treatment for previously treated advanced non-small-cell lung cancer with high c-Met protein overexpression.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Beam, Hyundai, Regenxbio, Sarepta.
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Abtherx, Affimed, AFT, Bayer, Endo, Galapagos, Hyloris, Intron, Leap, Mallinckrodt, Qliniq, Single Cell, Vaccinex.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Ascendis, Atea, Biorestorative, Cytodyn, Cytokinetics, Enterobiotix, Iteos, Karyopharm, Mediwound, Novo Nordisk, Roche.
U.S. Health and Human Services Secretary Robert Kennedy made his first appearance May 14 before the Senate Health, Education, Labor and Pensions (HELP) Committee since his January confirmation hearing. Chaos, testy arguments, accusations and surprising agreements ensued.
RSS
