Diamyd Medical accelerates Phase 3 Type 1 diabetes trial following FDA guidance

The interim efficacy readout, involving around 170 participants with 15-month data, remains on track for the end of March 2026

INOVIO’s DNA therapy for rare throat disease gets FDA review

INOVIO remains confident in the therapy’s benefits and intends to meet with the FDA to discuss maintaining eligibility under this pathway

SN Bioscience’s SNB-101 wins FDA orphan drug designation for gastric cancer

The drug, currently in a phase 1b/2 clinical trial for small cell lung cancer, is a polymer nanoparticle formulation of SN-38

US House passes Bill reauthorizing FDA rare disease incentive program

FDA okays AQVESME as first treatment for anemia in alpha- & beta-thalassemia

The approval makes AQVESME the only FDA-approved therapy for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia

FDA setback for Sanofi’s Tolebrutinib in progressive MS

Tolebrutinib had been provisionally approved in the United Arab Emirates in July 2025 for nrSPMS to slow disability accumulation independent of relapse activity

Amneal and mAbxience score FDA green light for two Denosumab biosimilars

Edwards Lifesciences’ SAPIEN M3 becomes first FDA-approved transseptal mitral valve replacement

The SAPIEN M3 transcatheter mitral valve replacement (TMVR) system is designed for patients with symptomatic moderate-to-severe or severe MR

Strides Pharma Inc's Chestnut Ridge facility gets 4 observations from USFDA

These observations are procedural in nature and the company does not anticipate any impact on supply of its commercial products

FDA nod to Boehringer Ingelheim’s JASCAYD for progressive pulmonary fibrosis in adults

The study found that nerandomilast effectively slowed lung function decline in patients, with discontinuation rates similar to placebo