FDA accelerated approval makes Genglycos the first treatment for GSDIa, based on a 31% reduction in daily cornstarch intake that must now be confirmed through long-term follow-up.
INTerpath-001 met recurrence-free and distant metastasis-free survival endpoints in 1,137 patients, giving individualized mRNA cancer therapy its first positive Phase 3 readout.
UroGen Pharma will invest up to $7 million in IntraGel while securing an option on TumoCure and potential rights to three additional SRGel oncology programmes.
FDA Fast Track designation could increase regulatory interaction around safusidenib while Nuvation Bio tests the IDH1 inhibitor across several glioma settings.
FDA grants LTI-03 Fast Track status as Rein advances its 120-patient Phase 2 IPF trial toward a closely watched second-half 2026 readout.
PleoPharma secures $6.5M NIDA funding as a 420-patient Phase 3 trial tests PP-01 for cannabis withdrawal in adults with cannabis use disorder.
ProCare Injury Specialists adds Waco as its 14th Texas clinic, extending its integrated accident-care network across Central Texas.
Guardian Pharmacy heads to Jefferies after raising 2026 guidance as IRA pricing masks stronger resident growth and margin gains.
Ingenia Therapeutics gains dual Prix Galien nominations as its KOSDAQ debut puts MK-8748, IGT-303 and the broader TIE2 pipeline under scrutiny.
Soft Bones urges South Korea to widen HPP reimbursement as age and radiographic criteria restrict treatment access for some patients.