PTC Therapeutics Inc. said a “highly competitive process with several parties involved” led to the deal centered on the firm’s Huntington’s disease program with Novartis AG, an arrangement that brings $1 billion up front along with as much as $1.9 billion in development, regulatory, and sales milestone rewards. Read More
Following a late-cycle review meeting with the U.S. FDA in September and the agency’s decision to skip the advisory committee meeting, expectations were high heading toward the PDUFA date for Applied Therapeutics Inc.’s priority NDA for govorestat in galactosemia. So the complete response letter issued by the FDA just ahead of the Nov. 28 PDUFA date, citing deficiencies in the clinical application, caught nearly everyone off guard. Read More
Sichuan Kelun-Biotech Biopharmaceutical Co. Ltd. won local approval of the first China-made trophoblast cell-surface antigen 2 (TROP2)-targeted antibody-drug conjugate (ADC), sacituzumab tirumotecan, as a third-line therapy for advanced breast cancer. Read More
The U.S. FDA said it is investigating the risk of hematologic malignancies associated with Bluebird Bio Inc.’s Skysona (elivaldogene autotemcel), approved in 2022 as a one-time gene therapy for treating early active cerebral adrenoleukodystrophy in boys, ages 4 to 17. Read More
Hangzhou Jiuyuan Gene Engineering Co. Ltd. raised HKD$485.4 million (US$62.38 million) in a November 28 IPO on the main board of the Hong Kong Stock Exchange (HKEX:2566). Read More
Researchers at the University of Rochester have described a neuroimaging-based biomarker that could identify individuals with early psychosis, and improved their identification when it was added to a standard neurocognitive diagnostic test. In a group of roughly 160 participants in the Human Connectome Early Psychosis Project, individuals who were in the early stages of psychosis had stronger connections from the thalamus (a midbrain sensory processing area) to the cortex, but weaker connections between different cortical areas, than controls. Read More
A proposed rule to implement the five-year-old Medicaid Services Investment and Accountability Act would expand the U.S. Health and Human Services’ (HHS) permissive exclusion authority to biopharma manufacturers that misclassify outpatient drugs supplied under agreements with federal health care programs. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Acadia, Adcendo, Agenus, Alector, Duality Biologics, Empyrean, Eos, Idorsia, Kronos, PTC, Saniona. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Advanz, Anavex, Aop Orphan, Beigene, Cevomed, Exelixis, Gsk, Kelun-Biotech, Lin, Novartis, Oncopeptides, Satsuma, Tuhura, Valneva. Read More
