Rare Disease Report


BioMarin Chief Executive Officer Jean-Jacques Bienaime spoke about the company’s development of valoctocogene roxaparvovec, a gene therapy in development for the treatment of Hemophilia A.
Global Blood Therapeutics, Inc. was granted Breakthrough Therapy Designation from the U.S. Food and Drug Administration for voxelotor for the treatment of sickle cell disease.
Catalyst Biosciences announced the initiation and open enrollment of the Phase 2 part of its Phase 2/3 program of marzeptacog alfa (activated), a highly potent, subcutaneously administered Factor VIIa therapy in development for the treatment of hemophilia A and B with inhibitors.
This morning, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to Novatris’ eltrombopag (Promacta), a first-line treatment for severe aplastic anemia (SAA).
The U.S. FDA has accepted for review the New Drug Application for Azedra (iobenguane I 131) to be indicated for patients with malignant, recurrent and/or unresectable pheochromocytoma and paraganglioma.
Novartis announced that its combination therapy Tafinlar (dabrafenib) + Mekinist (trametinib) was granted Priority Review by the U.S. FDA for the adjuvant treatment of stage 3 BRAF V600 mutation-positive melanoma.
Last week, Novartis announced that the U.S. FDA approved the inclusion of Treatment-free Remission data in Tasigna (nilotinib).
This morning, the U.S. FDA granted regular approval to hydroxyurea for the treatment of pediatric patients from 2 years of age and older with sickle cell anemia with recurrent moderate to severe painful crises.
Pfizer has announced that the U.S. FDA approved a supplemental New Drug Application for Bosulif (bosutinib) to include patients with newly-diagnosed chronic phase Philadelphia chromosome-positive chronic myelogenous leukemia.
At the ASH Meeting and Exposition Martin Hutchings, Ph.D., M.D. discussed whether Adcetris is worth being investigated as a combination therapy.
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