The U.S. Food and Drug Administration granted various designations for Sanofi’s avalglucosidase alfa for Pompe disease, Sanofi’s rilzabrutinib for immune thrombocytopenia, and ViiV Healthcare’s cabotegravir for HIV prevention.
Biogen’s muscle disease treatment Spinraza has been deemed too expensive for use on Britain’s state-run health service, even after a price discount offered by the U.S. drugmaker.
Biogen Inc. reported far better-than-expected second-quarter 2018 profit, driven by strong demand of the company’s Spinraza muscle disease treatment, and the U.S. biotech company raised its full-year profit forecast.
Investor’s Business Daily looked at three small biotech companies that appear to be leading the CRISPR race.
A Phase 3 study evaluating Ultragenyx Pharmaceuticals’ aceneuramic acid extended release in patients with GNE Myopathy did not achieve its primary endpoint.