Rare Disease Report


In this Rare Connections program, Martha Raymond; Founder of the Raymond Foundation, Executive Director of Michael’s Mission, and the Co-Chair of the GI Cancers Alliance meets with Sarah Bennett, a cholangiocarcinoma survivor to highlight the space.
At the 36th Annual J.P. Morgan Healthcare Conference, uniQure announced that in 2018, the company intends to advance its gene therapy AMT-061 into a pivotal study for hemophilia B.
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.
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