Discover
llllllllll Amondys 45 (casimersen) / Sarepta TherapSarepta Therapeutics Announces FDA Acceptance of Casimersen (SRP-4045) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45 (GlobeNewswire) - Aug 25, 2020 - "Sarepta Therapeutics...today announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045) and provided a regulatory action date of February 25, 2021. The FDA has indicated it does not currently plan to hold an advisory committee to discuss the application....received conditional approval of AMONDYS 45 as the brand name for casimersen...The NDA included data from the casimersen arm of the ESSENCE study (also known as Study 4045-301)..."llllllllll IMR-687 / Imara, IncImara Receives Orphan Drug Designation from the European Commission for IMR-687 for the Treatment of Sickle Cell Disease (GlobeNewswire) - Aug 25, 2020 - "Imara...announced that the European Commission granted Orphan Drug designation to IMR-687 for the treatment of patients with sickle cell disease (SCD)."llllllllll berubicin (RTA 744) / CNS Pharma, WPD PharmaCNS Provides FDA Update on IND Filing (PRNewswire) - Aug 25, 2020 - "CNS Pharmaceuticals, Inc...provided an update on the Company's plans for its lead product candidate, Berubicin, which is being studied for the treatment of glioblastoma multiforme (GBM). The company has taken significant strides in designing Phase 2 clinical trials and anticipates submitting an Investigational New Drug application (IND) to the U.S. Food and Drug Administration (FDA) this year and initiating clinical trials in early 2021...In preparation for submitting the IND, CNS also has completed the Clinical Study Report, or CSR, for a Phase 1 study for which the Company holds the rights....'We believe our efforts optimally position us to submit an IND for Berubicin in the fourth quarter of this year.'"llllllllll Tepmetko (tepotinib) / EMD SeronoFDA Accepts Filing of New Drug Application for Tepotinib for the Treatment of Patients with Metastatic NSCLC with METex14 Skipping Alterations (PRNewswire) - Aug 25, 2020 - "EMD Serono...announced that the US Food and Drug Administration (FDA) has accepted and granted Priority Review to the new drug application (NDA) for once-daily, orally-dosed tepotinib for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition exon 14 (METex14) skipping, as detected by an FDA-approved test...The application is based on results from the pivotal ongoing, single-arm Phase II VISION study..."llllllllll ivaltinostat (CG-745) / CrystalGenomicsCrystalGenomics’ anticancer candidate has FDA’s orphan drug status (Korea Biomedical Review) - Aug 25, 2020 - "CrystalGenomics said Monday that the U.S. Food and Drug Administration has designated its anticancer candidate Ivaltinostat as an orphan drug for liver cancer...The company plans to expand indications of Ivaltinostat for different types of cancers and enhance efficiency via combination therapy. It is preparing to conduct phase 1/2a trials of combined administration of Ivaltinostat and immune checkpoint inhibitors on patients with liver cancer in the U.S."
