EMA grants orphan drug status to Sanofi’s Efdoralprin Alfa for rare lung disease

Efdoralprin alfa, a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, showed superior efficacy to standard plasma-derived therapy in adults with AATD

Gilead’s new HIV therapy shows promising Phase 3 results

The study found BIC/LEN to be statistically non-inferior to BIKTARVY, Gilead’s current standard-of-care therapy

Eli Lilly reports positive Phase 3 results for oral obesity drug Orforglipron

At one year, the drug met its primary and all key secondary endpoints, delivering significantly better weight maintenance than placebo

Merck’s KEYTRUDA–Padcev combo delivers survival gains in Phase 3 bladder cancer trial

Astellas & Pfizer report breakthrough Phase 3 results in muscle-invasive bladder cancer

The combination also outperformed chemotherapy on another important secondary endpoint

FDA nod to Johnson & Johnson’s AKEEGA for aggressive prostate cancer

Patients with BRCA mutations often face aggressive disease and poor prognosis

Ascletis Pharma reports positive Phase I results for oral IL-17 inhibitor

The randomized, double-blind, placebo-controlled single ascending dose (SAD) study evaluated safety, tolerability, pharmacokinetics, and target engagement in 46 healthy participants

Zenas BioPharma and InnoCare hit key milestone in Lupus drug trial, secures China Phase 3 nod

The study also showed a clear dose-dependent trend, with the 75 mg QD group outperforming the 50 mg QD cohort

FDA nod to first oral single-dose treatment for gonorrhea

The approval of Nuzolvence (zoliflodacin) follows a collaborative development effort with the Global Antibiotic Research and Development Partnership

Neuren partner Acadia bags FDA go-ahead for new DAYBUE STIX powder for Rett Syndrome

The new powder formulation gives children and adults living with Rett syndrome greater flexibility and choice in dose volume and taste