Morning Briefing
Summaries of health policy coverage from major news organizations
Drug Companies Tap Into The 'Power Of The Patient'
Boston Globe: Biotechs Leverage Patient Groups In Drug Development
Ganot and other speakers at a panel hosted Wednesday by the Massachusetts Biotechnology Council cited the recent successful campaign to persuade the Food and Drug Administration to green-light Exondys 51, a Duchenne drug developed by Cambridge’s Sarepta Therapeutics Inc. Hundreds of patients and their families — including representatives of patient organizations that funded Sarepta’s clinical study — packed an FDA advisory committee meeting in April to offer emotional testimony in favor of the drug. The advisers and some FDA staffers contended the therapy had not been proved effective, but the agency overruled them and approved it. (Weisman, 9/29)
In other pharmaceutical news —
Boston Globe: Vertex Wins FDA Approval To Treat Children Between Six And 11 With Its CF Drug
Federal regulators on Wednesday approved Vertex Pharmaceuticals Inc.’s cystic fibrosis drug Orkambi to treat children from 6 to 11 years old. The decision by the Food and Drug Administration covers about 2,400 patients in the United States, expanding the domestic patient population eligible for Orkambi to nearly 11,000. (Weisman, 9/28)
The Wall Street Journal: Intra-Cellular Therapies Shares Plunge After Disappointing Drug Trial Results
Intra-Cellular Therapies Inc. said its lead drug candidate had a similar rate of effectiveness to placebo in a Phase 3 schizophrenia trial. The company’s shares fell 68% to $13.69 in after-hours trading Wednesday. (Beckerman, 9/28)
The Wall Street Journal: FDA Approves Medtronic’s New Automated Insulin Pump
The Food and Drug Administration approved for sale a significant advance toward a so-called artificial pancreas for Type 1 diabetes patients, a long-awaited insulin pump that aims to take some of the guesswork out of blood-sugar control. (Whalen and Linebaugh, 9/28)
The Star Tribune: Medtronic Gets FDA Approval For Automated Insulin Delivery Device
In a quick approval, the U.S. Food and Drug Administration on Wednesday gave a green light to the first medical device for type 1 diabetes that can automate varying doses of insulin 24 hours a day using a real-time sensor and an advanced computer program that learns a patient’s personal needs. Medtronic PLC’s MiniMed 670G “hybrid closed-loop” system received approval from the FDA just three months after the Minnesota-run device maker submitted its premarket application for approval. (Carlson, 9/28)
