Mathew Shanley

NORD announced that it will commemorate the 35th anniversaries of both its formation and the creation of the Orphan Drug Act with a year-long commemoration.

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).

Recent data have found that the same gene editing platform can disable the defective gene responsible for amyotrophic lateral sclerosis in mice. The therapy, which extended the lifespan of the mouse models by 25%, delayed the onset of the muscle wasting which characterizes the disease.

Results from a phase 1-2, dose escalation study cof a single peripheral infusion of AAV5-hFVIII-SQ in 9 men with severe hemophilia A displayed that changes in the vector and gene resulted in successful gene transfer.

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.