Regulatory and Standard Setting Organizations

Nathan Edwards of Arya Consulting moderates a discussion with Elisabeth Gardiner of Tevard Biosciences and Donny Xu of the RTW Institute about rare disease treatment development and navigating the FDA’s Rare Pediatric Disease Priority Review Voucher Program.

Antisense oligonucleotides are reshaping drug design, from RNase H gapmers to inhaled delivery; this FAQ explains how they work and why trials still fail.

The FDA approved Roche's satralizumab (Enspryng), an IL-6 receptor-targeting monoclonal antibody, for adults with thyroid eye disease based on the Phase 3 SatraGO-1 and SatraGO-2 trials, giving patients the first at-home subcutaneous treatment option.

AVT29, a proposed aflibercept biosimilar to Eylea 8 mg, could extend retinal disease treatment intervals to 16 weeks, versus 8 weeks today.

The FDA has accepted Pierre Fabre Pharmaceuticals’ resubmitted BLA for tabelecleucel, an allogeneic T-cell immunotherapy for EBV+ post-transplant lymphoproliferative disease. The FDA’s target action date is March 31, 2027.

FDA has accepted for review the biologics license application for FYB206, a proposed pembrolizumab biosimilar developed without a comparative phase 3 efficacy trial. A decision could come in the third quarter of 2027.

MT027, T-MAXIMUM's B7-H3 CAR-T therapy, earns a second FDA-cleared phase 2 trial, now testing lung and breast cancer brain metastases.