The European Medicines Agency (EMA) has granted approval for Dupixent, a therapy developed by Sanofi and Regeneron, to treat eosinophilic esophagitis (EoE) in children as young as one year old. This decision marks a critical step in addressing a condition that has left young patients and their families with limited options. With this approval, Dupixent […]
In a significant development for hereditary angioedema (HAE) patients, Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted therapy designed to prevent HAE attacks in individuals aged 12 and older. The FDA has set a Prescription Drug User […]
Sangamo Therapeutics experienced a significant surge in its stock price, jumping by as much as 53%, after the U.S. Food and Drug Administration (FDA) indicated support for an accelerated approval pathway for its gene therapy drug, isaralgagene civaparvovec (ST-920), designed to treat Fabry disease. The FDA’s guidance permits Sangamo to proceed with a streamlined clinical […]
In a major leap for hemophilia patients, Pfizer Inc. (NYSE: PFE) announced that the U.S. Food and Drug Administration (FDA) has granted approval for HYMPAVZI (marstacimab-hncq) to prevent or reduce bleeding episodes in individuals aged 12 and above with hemophilia A or B without inhibitors. This marks a groundbreaking advancement in the treatment landscape for […]
The U.S. Food and Drug Administration (FDA) has granted priority review status to Deciphera Pharmaceuticals, Inc.’s New Drug Application (NDA) for vimseltinib, a drug developed to treat tenosynovial giant cell tumor (TGCT). The FDA’s target action date for this review is February 17, 2025. This development follows the European Medicines Agency’s (EMA) acceptance of Deciphera’s […]
Novartis has reached a pivotal moment in the treatment of rare kidney diseases with the US Food and Drug Administration (FDA) granting accelerated approval for Fabhalta (iptacopan), a novel therapy designed to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN). This rare, progressively debilitating condition affects the kidneys by attacking the glomeruli, the […]
In a significant development for the pharmaceutical industry, Dr. Reddy’s Laboratories Ltd. has received a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) regarding its proposed biosimilar, DRL_RI, also known as ITUXREDI. This endorsement could pave the way for the launch of this promising biosimilar across European […]
Amgen (NASDAQ: AMGN), a global biotechnology leader, has announced its plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for teprotumumab. This treatment, a fully human monoclonal antibody and targeted inhibitor of the insulin-like growth factor-1 receptor (IGF-1R), is aimed at adults suffering from moderate to severe Thyroid Eye Disease […]
Bristol Myers Squibb (NYSE: BMY), a global leader in biopharmaceuticals, has reached a significant milestone with the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending approval of Opdivo in combination with cisplatin and gemcitabine. This recommendation is for the first-line treatment of adult patients with unresectable or metastatic […]
Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT), a leading global clinical-stage messenger RNA medicines company, has achieved a significant milestone in the development of treatments for infectious diseases and rare liver and respiratory conditions. The European Commission (EC), influenced by a favorable review from the European Medicines Agency (EMA), has recently granted orphan medicinal product designation […]