ALBANY, N.Y. & SHANGHAI, China — Drug Farm today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to DF-003, the company’s investigational small molecule inhibitor targeting ALPK1, for the treatment of patients with ROSAH syndrome, a rare, genetically driven systemic autoinflammatory disease with...
treatment News
GUILFORD, Conn. — Drug Farm, a clinical-stage biopharmaceutical company announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s alpha-kinase 1 (ALPK1) inhibitor, DF-003, to treat patients with ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache ) Syndrome. DF-003...
San Antonio, Texas – A drug effective in treating breast cancer shows new promise in addressing breast cancer with brain metastases or recurrent glioblastoma, as reported by results of a prospective window-of-opportunity trial at the University of Texas Health Science Center at San Antonio (UT Health San Antonio). The window...
New York, NY – A drug currently in clinical trials as a cancer therapy can also stimulate pancreatic beta cells to secrete insulin, revealing a previously unknown mechanism for insulin regulation in type 2 diabetes, according to a new study by Weill Cornell Medicine investigators. The preclinical discovery, reported Nov....
Durham, North Carolina – Scientists from Duke-NUS Medical School (Duke-NUS) have developed a new approach using the Zika virus to destroy brain cancer cells and inhibit tumor growth, while sparing healthy cells. Using Zika virus vaccine candidates developed at Duke-NUS, the team discovered how these strains target rapidly proliferating cells...
Approval based on pivotal results showing improvements in sustained disease remission and reductions in itch and oral corticosteroid use compared to placebo in adults with BP BP is a chronic, debilitating, and relapsing rare skin disease affecting approximately 27,000 adults in the US whose disease is uncontrolled by systemic corticosteroids...
TARRYTOWN, N.Y. and PARIS, France — Regeneron Pharmaceuticals, Inc. and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for the treatment of pediatric patients aged 1 to 11 years, weighing at least 15 kg, with eosinophilic esophagitis (EoE). Dupixent is now the first...
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi announced positive pivotal Phase 3 results from a trial evaluating Dupixent® (dupilumab) in adults with uncontrolled prurigo nodularis, a chronic type 2 inflammatory skin disease that causes extreme itch and skin lesions. The trial met its primary and all key secondary endpoints, showing...
Abstract Background: Advanced systemic mastocytosis (AdvSM) is a rare, life-threatening myeloid neoplasm predominantly driven by KIT D816V mutations. Avapritinib is a selective and potent KIT D816V inhibitor approved for the treatment of AdvSM. Methods: PATHFINDER (NCT03580655) is a clinical study evaluating avapritinib in adults with AdvSM. This analysis reports long-term...
STRASBOURG, France and PHILADELPHIA – Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today that the US Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for DYN101, an...