Accountable care organizations and other value-based payment models are increasing in the US, policy experts wrote in a Health Affairs blog, and there is no longer a question of whether the CMS will continue to support value-based payments. "Public and private payers could refine payment models to provide a clearer path to success in taking on some financial risk and create a more coherent set of complementary payment reforms," the authors wrote.
Breakthrough therapy designation was given by the FDA to Genentech's Xolair, or omalizumab, indicated to prevent severe allergic reactions after accidental exposure to at least one trigger in patients with food allergies. The decision was based on seven studies that assessed the safety and efficacy of the drug as monotherapy or in combination with oral immunotherapy against several food allergens.
The FDA gave a rare pediatric disease designation to Cellectar Biosciences' CLR 131, being developed to treat patients with Ewing's sarcoma.
MeiraGTx's AAV-CNGA3, which is being developed for the treatment of achromatopsia caused by mutations in the CNGA3 gene, received orphan drug status from the FDA.
The FDA has given investigational device exemption approval for a pivotal trial of GI Dynamics' EndoBarrier device, a plastic gut sleeve intended for treating patients with obesity and type 2 diabetes. The study, which still needs Institutional Review Board approval, will have HbA1c reductions at one year as its primary endpoint and will initially enroll up to 67 patients, possibly expanding to 240 patients after the company submits an interim safety report to the FDA.
The FDA has given 7D Surgical 510(k) clearance for its machine-vision image guided surgery system for cranial surgery. The system captures virtual fiducial markers from a patient's anatomy using a camera-based technology and machine vision algorithms, enabling cranial registration in almost any position.
Antibody therapy Hemlibra, or emicizumab, which is being developed by Roche Holding, Genentech and Chugai Pharmaceutical, has gained Health Canada's approval as a treatment for the prevention or reduction of bleeding in Canadian patients with hemophilia A who have factor VIII inhibitors. Health Canada's decision was based on data from two late-stage studies that demonstrated Hemlibra's safety and efficacy in adolescents, adults and children with hemophilia A who have factor VII inhibitors.
UK biopharma Motif Bio's marketing application for antibiotic iclaprim, developed to treat acute bacterial skin and skin structure infections, was accepted by the FDA under priority review status. Forty-eight to 72 hours after starting treatment, the drug was not found to be inferior to current standard of care vancomycin.
Orchard Therapeutics, which is developing ex vivo gene therapies, raised $150 million from a new funding round. Orchard recently acquired a GlaxoSmithKline portfolio of rare disease medicines that includes Strimvelis, a gene therapy for the treatment of adenosine deaminase severe combined immunodeficiency, which is also known as "bubble baby" disease.
The comment period for the Environmental Protection Agency's proposed rule on 2019 renewable volume obligations under the Renewable Fuel Standard and 2020 renewable volume obligations for biomass-based diesel will end Friday. The regulations would require 19.88 billion gallons of biofuels to be blended into the country's fuel supply in 2019, and would set the 2020 RVO for biomass-based diesel at 2.43 billion gallons.