Rare Disease Report


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).
Novartis has received U.S. FDA approval for lutetium (177Lu) oxodotreotide (Lutathera) for the treatment of somatostatin receptor positive GEP-NETs.
The ipsogen JAK2 RGQ PCR Kit  received clearance from the U.S. FDA for additional use in the diagnosis of all myeloproliferative neoplasms.
BERG announced this morning that the U.S. FDA has granted orphan drug designation to the company’s leading product candidate BPM31510 (ubidecarenone) for the treatment of pancreatic cancer.
New research published in Annals of Internal Medicine shows that the rate of arterial and venous thrombosis was significant in patients with myeloproliferative neoplasms (MPNs).
Last night, the U.S. FDA approved the supplemental New Drug Application to add overall survival data from the Phase 3 head-to-head ENDEAVOR trial to the Prescribing Information for Kyprolis (carfilzomib).
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