Elypta announces the enrollment of the first patient in the newly initiated AURORAX-0093A (AUR93A) study. AUR93A is intended to explore Elypta’s metabolism-based liquid biopsy platform for the prognosis of muscle-invasive bladder cancer (MIBC) before neoadjuvant chemotherapy. The first patient was enrolled at San Raffaele Hospital, Milan, Italy. AUR93A In AUR93A,...
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HYOGO, Japan – JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that European Medicines Agency (EMA) has granted orphan drug designation to JR-171, an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier...
CASTELVECCHIO PASCOLI, Italy — Kedrion announces that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its investigational treatment for Congenital Aceruloplasminemia (ACP), a rare genetic disorder of iron metabolism. Aceruloplasminemia is an autosomal recessive disorder caused by mutations in the CP gene, leading to a deficiency or...
Seoul, South Korea – The European Medicines Agency (EMA) has granted orphan drug designation to YH35995, Yuhan Corporation’s investigational oral therapy for Gaucher disease. The decision follows a similar orphan drug designation from the U.S. Food and Drug Administration (FDA) in April, reinforcing the therapy’s potential as a treatment for...
New York, NY – OS Therapies, Inc. (NYSE American: OSTX) (“OS Therapies” or “the Company”), the world leader in gene-edited, listeria-based cancer immunotherapies, today announced that the European Medicines Agency (EMA)’s Committee for Advanced Therapy (CAT), in conjunction with the Committee for Medicinal Products for Human Use (CHMP) and Pharmacovigilance...
STOCKHOLM, Sweden – Sobi® (STO: SOBI) today announced that the European Medicines Agency (EMA) has validated an indication extension application for Tryngolza® (olezarsen) for the treatment of adult patients with severe hypertriglyceridemia (sHTG) ≥880 mg/dL (≥10 mmol/L). Patients with elevated triglyceride levels have substantially higher risks of all-cause mortality, atherosclerotic...
Blacksburg, VA – Every 65 seconds, someone in the United States develops Alzheimer’s disease, a devastating form of dementia that affects 6.2 million Americans. Though it was initially identified almost 120 years ago, Alzheimer’s disease is a progressive neurological disorder with no cure and few treatments. It starts out with minor...
ROCKLAND, Mass. — EMD Serono, the Healthcare business of Merck KGaA, Darmstadt, Germany, in the U.S. and Canada, today announced the presentation of two new post-hoc analyses of tertiary endpoints from the MAGNIFY-MS study, a Phase IV, prospective, open-label study evaluating the efficacy and safety of MAVENCLAD® (cladribine) tablets in...
Vancouver, Canada — The global orphan drug market size was USD 154.20 Billion in 2022 and is expected to register a rapid revenue CAGR of 12.3 % during the forecast period. Rising frequency of uncommon illnesses and increasing number of governments providing regulatory incentives for development of orphan drugs are...
LAS VEGAS — Muscular dystrophies encompass a range of muscle disorders resulting from genetic mutations. Over time, these conditions lead to muscle weakness, which hinders everyday activities. Muscular dystrophy takes various forms, each targeting specific muscle groups and manifesting at different ages with varying degrees of severity. It can either...