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

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The 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.

Britain’s AstraZeneca said a potential medicine to prevent respiratory syncytial virus (RSV) in babies and infants had been granted special status by U.S. and European regulators, designed to speed up the development of novel and better drugs.

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.