Regulatory snapshots, including global drug submissions and approvals, and other regulatory decisions and designations: Alessa, Aquestive, Kezar, PDS, Uniqure.
Biopharma happenings, including deals and partnerships, and other news in brief: Acepodia, Anaptys, Avista, Biocytogen, Cytoreason, Dewpoint, GSK, Iktos, Link Cell Therapies, Madrigal, Medilink, Nona, Pfizer, Pierre, Roche, Sanofi, Tesaro.
A second complete response letter issued by the U.S. FDA for Atara Biotherapeutics Inc.’s allogeneic T-cell immunotherapy Ebvallo (tabelecleucel) for Epstein-Barr virus positive post-transplant lymphoproliferative disease is “a complete reversal of position” by the agency, which had previously confirmed the single arm Allele trial was enough to support a BLA under the accelerated approval pathway, the company said.
After raising AU$29 million (US$19.44 million) in a series A round, Rage Biotech Pty Ltd. is beginning phase I trials of its lead candidate, RB-042, an inhaled splice-switching oligonucleotide for treating chronic obstructive pulmonary disease (COPD) and other inflammatory lung diseases.
Moving away from a one-size-fits-all approach, the U.S. FDA's CBER released details Jan. 11 about how it’s leveraging its growing experience with cell and gene therapies (CGTs) to exercise greater regulatory flexibility in chemical, manufacturing and control requirements for the products.
Stoke Therapeutics Inc.’s speeded-up timeline for zorevunersen, the antisense oligonucleotide in development with Biogen Inc. as a first-in-class potential disease-modifying treatment for Dravet syndrome, put the rare, severe form of lifelong epilepsy in the spotlight. The news involved completion of enrollment and a phase III data readout from the Emperor study, as officials said signups of 150 patients are expected in the second quarter of the year, which puts the study on track for data in mid-2027.
It was a battle of the companies with drugs for transthyretin-mediated amyloidosis on the first day of the J.P. Morgan 2026 Healthcare Conference with Alnylam Inc., Bridgebio Pharma Inc. and Pfizer Inc. all presenting at the annual kickoff conference. One company disclosed 2025 sales as well as 2026 revenue guidance, the second only looked back, while the third was too big to do either for a specific drug.
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