Gilead Sciences Inc. won FDA approval to market Harvoni, the first oral therapy for people with genotype 1 hepatitis C virus (HCV) to eliminate the need for both interferon and ribavirin. Read More
With a global, confirmatory phase III trial under way as of June with Translarna (ataluren) in nonsense mutation cystic fibrosis (nmCF), plus a phase III experiment in nonsense mutation Duchenne muscular dystrophy (nmDMD) fully enrolled as of last month, PTC Therapeutics Inc. is raising $108.75 million through the sale of 3 million shares at $36.25 each. Read More
LONDON – Leading health experts said that having decided it is ethical to use non-approved products to help deal with the Ebola virus epidemic, it would be unethical if those drugs were administered in the context of randomized clinical trials. Read More
Understanding the genetic causes of diseases has allowed a better understanding what, from a cell biological perspective, is going wrong. "We're always defining things genetically, and that is something I subscribe to," Carl Ernst told BioWorld Today. Read More
Scientists committed to making a successful leap from lab to market are getting a leg up from a unique NIH pilot program teaching a relatively new model of entrepreneurship, that of the lean start-up. Read More
LONDON – Novartis AG is looking to the commercialization of its CAR T-cell cancer immunotherapies in a $104 million-plus royalties manufacturing deal with Oxford Biomedica plc, to use the gene therapy specialist's Lentivector technology to transfect patients' T cells. Read More
Ariad Pharmaceuticals Inc., of Cambridge, Mass., said the Pharmacovigilance Risk Assessment Committee (PRAC) of the EMA concluded its review of Iclusig (ponatinib) and has recommended that the drug continue to be used in Europe in accordance with its already approved indications for the treatment of adult patients with chronic phase, accelerated phase or blast phase chronic myeloid leukemia who are resistant to Sprycel (dasatinib. Read More
Isis Pharmaceuticals Inc., of Carlsbad, Calif., updated data from its ongoing open-label phase II studies testing ISIS-SMNRx in infants and children with spinal muscular atrophy (SMA) at the International World Muscle Society Congress in Berlin. Read More
Janssen-Cilag International NV, of Beerse, Belgium, a unit of Johnson & Johnson, said a type II variation was filed with the EMA seeking approval of Stelara (ustekinuab) for the treatment of moderate to severe plaque psoriasis in pediatric patients, ages 12 to 17, who are inadequately controlled by, or are intolerant to, other systemic therapies or phototherapies. Read More
Ipsen SA, of Paris, reported positive results at the European Association of Urology Central European Meeting in Cracow, Poland, from the phase III study of tritorelin pamoate administered subcutaneously in patients with locally advanced or metastatic prostate cancer. Read More
Muscle weakness is a common problem in aging, affecting more than half of individuals older than 80. Now, scientists from Columbia University have shown they were able to improve skeletal muscle function and increase exercise in aged mice by increasing the activity of an antioxidant enzyme specifically in the mitochondria, the energy powerhouses of the cell. Read More
