Mathew Shanley

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.

Adamas Pharmaceuticals, Inc. announced the full commercial launch of Gocovri (amantadine) extended release capsules for the treatment of dyskinesia in patients with Parkinson’s disease receiving levodopa-based therapy, with or without concomitant dopaminergic medications.

Last week, Pfizer announced a new partnership with Sangamo. As part of the agreement, the sides will team to develop a potential gene therapy to treat ALS, or Lou Gehrig’s disease.

Two months after Ionis submitted its NDA to the U.S. FDA, the company has announced that the regulatory agency has accepted its investigational drug inotersen for Priority Review.

This morning, it was announced that Retrophin, Inc. and Censa Pharmaceuticals are partnering to continue development and evaluate CNSA-001 for the treatment of phenylketonuria (PKU).

Mallinckrodt plc has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA), as per an official announcement from the company yesterday.

Last night, the U.S. FDA posted a warning letter addressed to American CryoStem Corporation for marketing an adipose derived stem cell product without FDA approval and for significant deviations from current good manufacturing practice requirements.

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.

Asthma patients woke up less often and improved faster with FF/VI

Ambulatory oxygen improved symptoms, performance, and recovery time in fibrotic ILDs.