Tag: safety profile

Press Release - Regulated Information

Zurich, July 29, 2025 – MetrioPharm AG, a pharmaceutical company developing drugs for inflammatory and infectious diseases, announced today that the European Medicines Agency has granted Orphan Drug Designation (ODD) to its lead compound, MP1032 for the treatment of children affected by Duchenne muscular dystrophy (DMD). This milestone follows the U.S. FDA's ODD, underscoring MP1032's potential as a first-in-class therapy that could minimize reliance on high-dose corticosteroids, which cause severe side effects like osteoporosis, growth stunting, and metabolic issues.

WALTHAM, Mass., July 28, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI^® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States.¹

SOUTH SAN FRANCISCO, July 25, 2025 (GLOBE NEWSWIRE) -- Recognify Life Sciences, a clinical-stage biotech company focused on developing treatments for cognitive impairment, today announced that its randomized, double-blind, placebo-controlled Phase 2b clinical trial evaluating inidascamine (formerly RL-007) in patients with cognitive impairment associated with schizophrenia (CIAS) did not meet its primary endpoint.

VYJUVEK approved for the treatment of DEB from birth with flexible administration options including home dosing and the option for administration by the patient or their family

NEW YORK and MAINZ, GERMANY, July 25, 2025 — Pfizer Inc. (NYSE: PFE, “Pfizer”) and BioNTech SE (Nasdaq: BNTX, “BioNTech”) announced today that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for the companies’ LP.8.1-adapted monovalent COVID-19 vaccine (COMIRNATY^® LP.8.1) for active immunization to prevent COVID-19 caused by SARS-CoV-2 in individuals 6 months of age and older. The adaptation is based on the recommendation from the EMA’s Emergency Task Force (ETF) to update COVID-19 vaccines to target the LP.8.1 variant for the 2025-2026 season. The ETF stated that “targeting LP.8.1 will help maintain the effectiveness of the vaccines as SARS-CoV-2 continues to evolve.”²

Schlieren / Zurich, Switzerland, 25 July, 2025 – Memo Therapeutics AG ("MTx”), a late-stage biotech company translating unique immune responses into superior medicines to treat viral infections and cancer, today announced results from its Phase II clinical trial evaluating potravitug, a highly potent human BK polyomavirus (“BKPyV”)-neutralizing monoclonal antibody, for the treatment of BKPyV infection in kidney transplant recipients (“KTR”).