BioMarin Pharmaceutical's hemophilia A gene therapy candidate valoctocogene roxaparvovec appeared to increase factor VIII levels more than a gene therapy from Spark Therapeutics, according to a study in The New England Journal of Medicine and data presented at a meeting of the American Society of Hematology.
Data presented at the meeting of the American Society of Hematology last week by bluebird bio and Celgene researchers on their chimeric antigen receptor T-cell therapy candidate bb2121 showed the therapy achieved a 94% response rate from 18 patients with advanced multiple myeloma, with 56% maintaining remission 40 weeks after treatment on average.
Janssen filed a supplemental new drug application to the FDA for the use of Xarelto, or rivaroxaban, in reducing the risk of acute limb ischemia in patients with peripheral artery disease and of major cardiovascular events in patients with chronic coronary and/or peripheral artery disease. The application was supported by data from a late-stage study, which found that compared with aspirin use alone, the drug reduced cardiovascular events in patients by 24%, death by 22%, stroke by 42% and heart attack by 14%.
AstraZeneca disclosed details on data from its clinical trial to assess the efficacy of its drug candidate Calquence as a treatment for mantle cell lymphoma, which showed its efficacy and resulted in fewer side effects compared with the current standard of care. GSK was also scheduled to publish a study on the effectiveness of its multiple myeloma antibody treatment GSK 2857916, according to presentations made at the American Society of Hematology last week.
The FDA has approved an investigational device exemption for Cerus' late-stage ReCePI study, allowing the firm to progress with the evaluation of its Intercept blood system for red blood cells to be used in patients receiving complex cardiac surgery. Development of the system is supported by the Biomedical Advanced Research and Development Authority.
The CMS' clinical lab fee schedule for 2018 would make cuts to 75% of all tests, creating "new barriers to patient access to care," AACC says. "One unintended consequence of the proposed CLFS is that it may force many laboratories to stop or significantly curtail their testing, particularly rural hospitals, small health clinics, and physician office laboratories," AACC warned the CMS.
Luke Miels, the new head of pharmaceuticals at GlaxoSmithKline, said the company will focus on buying or licensing early-stage cancer and immunology drugs. After gaining recent approvals for three new products -- for shingles, HIV and chronic obstructive pulmonary disease -- the company will not launch others until after 2020.
The Medicare Payment Advisory Commission says the CMS' Merit-based Incentive Payment System should be replaced by a voluntary program under which clinicians could choose to participate in a value-based reimbursement system scored on population-based measures and claims data. The proposal comes after many health care providers said they are unprepared for the financial and administrative requirements of the Medicare Access and CHIP Reauthorization Act's Quality Payment Program.