Nov 25, 2019
FDA approves novel treatment to target abnormality in sickle cell disease
Nov 21, 2019
FDA takes second action under international collaboration, approves new treatment option for patients with chronic lymphocytic leukemia
Nov 20, 2019
FDA approves first treatment for inherited rare disease
Nov 15, 2019
FDA approves first targeted therapy to treat patients with painful complication of sickle cell disease
Nov 14, 2019
FDA approves new antibacterial drug to treat complicated urinary tract infections as part of ongoing efforts to address antimicrobial resistance
Oct 21, 2019
FDA approves new breakthrough therapy for cystic fibrosis
Oct 08, 2019
FDA awards two grants for natural history studies in rare diseases
Oct 08, 2019
FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases
Oct 08, 2019
Vast Therapeutics Receives Qualified Infectious Disease Product Status for BIOC11
Sep 27, 2019
FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels
Sep 17, 2019
FDA takes first action under new international collaboration with Australia and Canada designed to provide a framework for concurrent review of cancer therapies, approving treatment for patients with endometrial carcinoma
Aug 16, 2019
FDA approves treatment for patients with rare bone marrow disorder
Aug 15, 2019
FDA approves third oncology drug that targets a key genetic driver of cancer, rather than a specific type of tumor
Aug 13, 2019
Windtree Announces FDA Fast Track Designation for Istaroxime
Aug 05, 2019
Provention Bio Announces Breakthrough Therapy Designation for Teplizumab (PRV-031) for the Prevention or Delay of Clinical Type 1 Diabetes in At-Risk Individuals
Aug 02, 2019
FDA approves first therapy for rare joint tumor
Jun 21, 2019
FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older
Jun 04, 2019
BioMarin Announces Approval of Vimizim® (elosulfase alfa) in China for Treatment of Morquio A Syndrome
May 24, 2019
FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality
May 06, 2019
FDA approves first treatment for children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder