An FDA advisory committee voted 13-0 to back the approval of GW Pharmaceuticals' Epidiolex, a cannabidiol oral solution for the treatment of Lennox-Gastaut and Dravet syndromes in patients ages 2 and older. In clinical trials, Epidiolex reduced monthly seizures in patients by 40%, and the FDA is expected to make a decision on the drug by late June.
FDA Commissioner Scott Gottlieb conceded that the agency is partly to blame for high drug prices because it could have done more to promote competition in the generic pharmaceutical market. Speaking at an event sponsored by pharmacy benefit managers, Gottlieb said improving the approval pathway for generic medicines and creating regulations to boost competition for complex brand-name drugs are some of the ways the agency could promote lower drug prices.
The CDC reported that the E. coli 0157:H7 outbreak linked to romaine lettuce grown in Yuma, Ariz., has expanded to 16 states, with 53 cases now reported, including at least 31 people hospitalized and five who developed kidney failure. Investigators have not yet identified a specific source of the lettuce.
Athenex's oral therapy Oraxol, a combination of paclitaxel and a nonabsorbable gastrointestinal tract P-glycoprotein pump inhibitor, was given orphan drug status by the FDA. The candidate is being developed as a treatment for patients with angiosarcoma.
The National Institute for Health and Care Excellence recommended Roche Holding and subsidiary Chugai's RoActemra, or tocilizumab, for one-year National Health Service funding as a treatment for adult patients with giant cell arteritis. The drug is also licensed to treat adult patients with moderate to severe active rheumatoid arthritis, polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis in patients as young as 2 years.
Krystal Biotech's gene therapy KB103, which is being developed as a treatment for dystrophic epidermolysis bullosa, received orphan status from the European Medicines Agency.
The FDA granted Arthrosurface 510(k) clearance for its stemless total shoulder system, OVOMotion Shoulder Arthroplasty System, for use with the company's Inlay Glenoid System. The shoulder system is intended for use in patients with severely disabled or painful shoulder joints due to avascular necrosis, arthritis or traumatic events.
European regulators granted Fountain Valley, Calif.-based HeartStitch CE mark approval for its CoAp Pro device, which is intended to help re-establish proper coaptation by facilitating remodeling of mitral valve leaflets. The device repairs the leaflets using sutures instead of a full prosthetic device.
The FDA granted Urotronic approval to conduct a pivotal trial of its Optilume drug-coated balloon, which is designed for treating men with narrowed urethras. The study, which will include up to 20 sites, will enroll at least 140 men who previously had at least two dilation treatments for the same urethral stricture and will randomize the participants to receive either the device or a standard of care treatment.
A follow-on offering brought in $140.3 million for Apellis Pharmaceuticals, which intends to launch a late-stage trial of APL-2 for the treatment of geographic atrophy. In a midstage trial, treatment with APL-2 yielded sustained, significant reductions in lesion growth in patients.
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