Valneva SE was awarded Breakthrough Therapy Designation for the company’s single-shot chikungunya vaccine candidate, VLA1553, by the U.S. Food and Drug Administration.
The U.S. Food and Drug Administration approved Zolgensma, a gene therapy that Novartis called a life-changing medicine, for children younger than 2 years of age who have been diagnosed with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene.
Top 10 Pipelines To Watch: 2019 Annual Report
Analysts, Ankylosing Spondylitis (AS), Annual Reports, Antiretroviral Drugs, Atopic Dermatitis (Eczema), Autoimmune Diseases, B Cells, Big Pharma, Biologics, Biopharma, Biotechnology, Blockbusters, BRCA Gene, BRCA mutation, Breakthrough Therapy Designation, Business, Cancer, CAR-T Therapy, Cells, Chemotherapy, Clinical Data, Clinical Trials, Collaborations, Crohn's Disease, Cystic Fibrosis, Drug Discovery, EU, European Medicines Agency (EMA), Fast Track Designation, FDA, FDA/Regulatory, February 2019, Future Blockbusters, Gene Editing, Generalized Myasthenia Gravis (gMG), Genomics, HIV, Human Genome, Immune Cells, Immune System, Immune-Mediated Diseases, Immunotherapy, Inflammatory Diseases, Injectables, Innovation, Integrase strand transfer inhibitors, Issue Archives, Japan, M&A, Med Ad News, Monoclonal Antibodies, Multiple myeloma, Neoantigens, Neurology, Neuromyelitis Optica Spectrum Disorder (NMOSD), New Drug Applications, Non-Nucleoside Reverse Transcriptase Inhibitor (NNRTI), Non-Small Cell Lung Cancer (NSCLC), Nonalcoholic Steatohepatitis (NASH), Nuclear Medicine, Oncology, Orphan Drug Designation, Paroxysmal Nocturnal Hemoglobinuria (PNH), Precision Medicine, Prescription Drug User Fee Act (PDUFA), Priority Medicines (PRIME) Designation, Priority Review Voucher, Product Pipelines, Proteins, Psoriatic Arthritis, R&D, Radioligands, Rare Disorders, Research, Rheumatoid Arthritis, RNA Interference (RNAi), Sickle Cell Disease, Small Molecules, Special Reports, T-Cells, Top 10 Pipelines, Top 10 Pipelines To Watch, Tumors, Ulcerative Colitis, Ultra Rare DiseasesThe return on R&D investment for leading biopharmaceutical manufacturers fell to a nine-year low while the U.S. FDA approved a record-breaking amount of novel medicines during 2018.
Orchard Therapeutics Receives EMA PRIME Designation for OTL-300
European Medicines Agency (EMA), Gene Therapy, Hematopoietic Stem Cell Transplantation (HSCT), Metachromatic Leukodystrophy (MLD), Priority Medicines (PRIME) Designation, Rare Diseases, Transfusion-Dependent Beta-Thalassemia (TDT), Wiskott–Aldrich Syndrome (WAS), X-Linked Chronic Granulomatous Disease (X-CGD)Orchard Therapeutics announced that the European Medicines Agency granted Priority Medicines (PRIME) designation to OTL-300, an investigational autologous ex vivo lentiviral gene therapy for the treatment of transfusion-dependent beta-thalassemia.