Bio Roundup: Rare Disease Showdowns, Prices on TV, Dealmania & More
Summary
A few years ago, for instance, there were no drugs available for spinal muscular atrophy (SMA), an inherited and potentially lethal neuromuscular disease. Elsewhere, drug prices are coming to commercials near you, a new gene therapy is on the precipice of US approval, and a bunch of pharma companies whipped out their checkbooks. —The Wall Street Journal delved into healthcare system problems that Zolgensma could trigger if, as expected this month, it becomes just the second approved gene therapy in the U.S.. Analysts have projected a price tag of $2 million for a one-time dose. —AstraZeneca (NYSE: AZN ) spinout Viela Bio will seek FDA approval of experimental drug inebilizumab, which is aimed at the rare autoimmune disease neuromyelitis optica spectrum disorder. —The World Health Organization ramped up its Ebola vaccination efforts in the Democratic Republic of the Congo, where containment of an outbreak is being hampered by violence in the region.