Tag: safety profile

- The INDIGO study met its primary and all key secondary endpoints with statistically significant results -

Tokyo, Japan and Cambridge, UK, 2 June 2026 – Nxera Pharma Co., Ltd. (“Nxera” or the “Company”)　 announces that the Ministry of Food and Drug Safety (MFDS) of the Republic of Korea has granted Orphan Drug Designation (ODD) and Global Innovative Products on Fast Track (GIFT) designation to vamorolone for the treatment of Duchenne muscular dystrophy (DMD). The Company plans to submit a Marketing Authorization Application (MAA) for vamorolone in South Korea during 2026.

POWER1 Study in highly refractory patients with focal onset seizures did not meet its primary success measure

ROCKVILLE, Md. and SUZHOU, China, Aug. 17, 2025 (GLOBE NEWSWIRE) -- Ascentage Pharma (NASDAQ: AAPG; HKEX: 6855), a global, commercial stage, integrated biopharmaceutical company engaged in the discovery, development and commercialization of novel, differentiated therapies to address unmet medical needs in cancer, announced that it has received clearance by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to conduct GLORA-4 study (NCT06641414), a global registrational Phase III study of lisaftoclax (APG-2575), a proprietary Bcl-2 inhibitor, in combination with azacitidine (AZA), for the treatment of patients with newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS).

Tonmya is the first FDA-approved therapy for the treatment of fibromyalgia in over 15 years

First-in-human study evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics of BGE-102, an orally available small molecule with potential best-in-class profile

Results support potential of efruxifermin (EFX) to reduce risk of fibrosis progression in patients with pre-cirrhotic (F2-F3) MASH