Orphan Therapeutics Accelerator licenses OTXL-101 for OTC deficiency, reviving a rare disease gene therapy with early human data and major hurdles ahead.
AbbVie seeks FDA approval for etentamig after Phase 3 myeloma results showed a 60% reduction in progression risk. Infection safety remains in focus.
Vincora gains FDA permission to study its NeXuS mitral repair system in the US, with procedural safety and lasting valve repair now in focus.
Omabit raises $2 million from Bright Ventures to connect clinical and genomic data for drug development decisions. Validation and adoption remain key.
Orbis names Joanne Smith-Farrell CEO after its $1.4 billion Novo Nordisk partnership. The next test is advancing AI-designed oral macrocycles.
Stevanato and Aptar will test prefilled syringe systems for biologics, examining plunger forces and container integrity under USP 382 expectations.
Avanzanite appoints Sarah O'Connell to expand UK rare disease operations. The move strengthens its European commercialisation and patient access strategy.
Enhertu wins EU approval after cutting invasive recurrence or death risk by 53% versus T-DM1. Lung toxicity remains a key consideration.
Pfizer wins FDA approval for TALZENNA plus XTANDI in earlier metastatic prostate cancer. See how a 52% progression-risk reduction changes treatment.
Bayer's LYNKUET showed improved self-reported sleep quality in a post-hoc Phase 3 analysis. The findings do not establish a separate sleep indication.