Rare Disease Report


This morning, Cellectar Biosciences announced that the U.S. FDA has granted orphan drug designation to CLR 131 for the treatment of neuroblastoma.
Phase 2 clinical and immunological data presented at the 44th Annual EBMT Meeting this morning revealed that galinpepimut-S is safe and effective in high-risk multiple myeloma.
At the ENDO Annual Meeting in Chicago, Progenics presented biochemical tumor marker data from its pivotal Phase 2 trial of iobenguane I 131 (Azedra) in patients with pheochromocytoma and paraganglioma
Top-line results from the pivotal Phase 3 GLOBE study of ofranergene obadenovec in combination with bevacizumab in patients with recurrent glioblastoma were reported today, and the study did not meet its primary endpoint of overall survival.
Rigel Pharmaceuticals announced that data from its FIT Phase 3 extension study of fostamatinib in patients with chronic ITP will be presented at the Biennial Summit of the Thrombosis and Hemostasis Societies of North America.
Kazia Therapeutics has received orphan drug designation by the U.S. FDA for GDC-0084, intended for the treatment of glioblastoma multiforme.
Nativis, Inc. announced this morning that its Voyager Pediatric system has been granted a Humanitarian Use Designation from the U.S. FDA.
Imara has announced that it has dosed the first patient in its Phase 2a clinical trial of IMR-687 in adult patients with sickle cell disease.
Amgen announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for recommending a label variation for Kyprolis (carfilzomib).
Emmaus Life Sciences has announced that it has a Medicaid Drug Rebate Agreement with the Centers for Medicare and Medicaid Services, allowing coverage of Endari (L-glutamine oral powder).
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