Three months ahead of schedule, the FDA approved Kalydeco (ivacaftor), a new cystic fibrosis drug from Vertex Pharmaceuticals Inc., of Cambridge, Mass. The drug targets the G551D mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene in cystic fibrosis, which affects about 4 percent of patients with the disease, or about 1,200 patients in the U.S. Read More
While it didn't exactly blast the ball out of the park, Biogen Idec Inc. reported solid 2011 revenues, reaching the $5 billion milestone for the first time. Read More
Only two weeks after disappointing news on its Alzheimer's disease candidate, Dimebon (latrepirdine), Medivation Inc. had better news to share. Data from its Phase III AFFIRM trial of prostate cancer drug MDV3100, which will be presented later this week, were highlighted in a press conference put on by the American Society of Clinical Oncology 2012 Genitourinary Cancers Symposium Tuesday. Read More
WASHINGTON – Recognizing social media as a powerful tool that small start-ups can use to attract investors, an SEC advisory committee is advising the commission to consider an exemption from its registration requirements to permit crowd-funding. Read More
LONDON – There's been another twist in the gene therapy saga, with the European Commission refusing to give its usual rubber stamp to the European Medicines Agency's (EMA) decision not to grant marketing authorization to Glybera, a treatment for inherited disorder lipoprotein lipase deficiency (LPL). Read More
• MannKind Corp., of Valencia, Calif., said it intends to offer and sell $50 million worth of units in an underwritten public offering, with each unit consisting of one share of common stock and a warrant to purchase 0.6 of a share. Read More
• Argos Therapeutics Inc., of Durham, N.C., presented a high resolution immune monitoring analysis of Phase II study AGS-003-006, testing Arcelis immunotherapy AGS-003 in combination with Sutent (sunitinib, Pfizer Inc.) in patients with advanced renal cell carcinoma (RCC), with results showing a statistically significant correlation between antitumor memory T-cell responses and overall survival. Read More
• Transparency Life Sciences LLC, of New York, launched operations with the introduction of a prototype of the company's crowdsourced web platform designed to allow patients, physicians, researchers and other stakeholders to contribute to the design of clinical studies. Read More
