The first therapies for several rare diseases were among medicines given the green light by European regulators at their monthly meeting. The EMA’s Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion for Sanofi SA’s Xenpozyme (olipudase alfa) for two types of Niemann-Pick disease and Eiger Biopharmaceuticals Inc.’s Zokinvy (lonafarnib) for children with Hutchinson-Gilford progeria syndrome or progeroid laminopathies. PTC Therapeutics Inc.’s Upstaza (eladocagene exuparvovec), the first medicine for adults and children with aromatic L-amino decarboxylase deficiency, was also backed by the CHMP. Read More
Shares of Athersys Inc. fell sharply May 20 after its partner and top investor Healios K.K. reported that Multistem (invimestrocel), the experimental cell therapy they've co-developed since 2016, missed the primary endpoint of a Japanese phase II/III ischemic stroke study called Treasure. Read More
Atara Biotherapeutics Inc. is keeping the faith and figuring out next steps with its next-generation, mesothelin-directed CAR T-cell therapies after Bayer AG ended their exclusive worldwide licensing agreement, signed in late 2020. The setup included funding and development of ATA-3271, an armored allogeneic T-cell immunotherapy, and an autologous version, ATA-2271, for high mesothelin-expressing tumors such as malignant pleural mesothelioma and non-small-cell lung cancer. Read More
In the wake of a patient’s death, the U.S. FDA has placed a partial clinical hold on Foghorn Therapeutics Inc.’s phase I study of FHD-286 in treating relapsed and/or refractory acute myelogenous leukemia (r/r AML) and myelodysplastic syndrome. Read More
Zhejiang Acea Pharmaceutical Co. Ltd. failed to obtain marketing approval from China’s NMPA for its third-generation EGFR tyrosine kinase inhibitor, abivertinib. The NDA was based on a phase II trial of abivertinib in patients with EGFR Thr790Met point mutation-positive non-small-cell lung cancer with disease progression from prior EGFR inhibitors. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Aileron, Asahi, Brickell, Caladrius, Chinook, Mind Medicine, Nimbus, Ose, Surrozen, Xylocor. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Akari, Almac, Aslan, Biogen, Blue Water, Comera, Hitgen, Immix, Kymera, Smart Immune, Tenaya and Zymeworks. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Abbvie, Biontech, Eiger, Eli Lilly, Indaptus, Karyopharm, Merck, Mersana, Mirati, Novartis, Pfizer, Poxel, PTC, Saniona, Sanofi, Siga, Simcere, Tallac, Tetra, Umecrine, Valneva. Read More
