Updates on the regulatory front are eagerly awaited by investors in the pharma/biotech sector as they impact the share price of the concerned company. Regulatory updates include events like filing of marketing applications, acceptance of these applications for review by the regulatory agencies, reviews by advisory panels and finally, a response from the regulatory agency regarding the approval status. Late last week, Horizon Pharma plc ( HZNP ) announced that the FDA has granted orphan drug status to Actimmune for the treatment of Friedreich's ataxia (FA). According to Horizon Pharma, FA is a degenerative neuro-muscular genetic disorder affecting approximately one in every 50,000 people in the U.S. Under the U.S. Orphan Drug Act, a drug or biological product is granted the special status if it is considered capable of treating a rare disease or condition that affects less than 200,000 patients in the U.S. On receiving orphan drug designation for a candidate, the company developing it is entitled to various incentives, including tax credits for qualified clinical testing. Moreover, the Prescription Drug User Fee Act (PDUFA) fees are waived off the marketing application for an orphan drug designated candidate. Additionally, this status should make Actimmune eligible for seven years of … Continue reading →

Contact Information Available for logged-in reporters only As Breast Cancer Awareness month continues, Dr. Bhuma Krishnamachari, an epidemiologist and genetics expert at NYIT College of Osteopathic Medicine, is available to talk about the changing landscape of genetic tests and what it means for patients and doctors. Genetic testing has changed from a single gene approach to a multiple gene approach, says Krishnamachari, who specializes in hereditary cancers. Yet, doctors may not understand the clinical significance of the gene mutations found in these more comprehensive tests. With more information comes more questions, says Krishnamachari. For patients, Krishnamachari offers several tips to navigate the often confusing world of genetic tests. Its important to find a doctor who is well-versed in hereditary factors and who involves many disciplines in patient care because the complexity of testing has increased, she says. Were moving toward more individualized treatments so the team should include genetic counselors, surgeons, oncologists, and primary care physicians. Krishnamachi recently led NYIT research teams that published three separate studies on the need for physicians to learn more about genetic testing so they can help patients who may have a high risk of developing breast, ovarian, and colorectal cancers. Physicians, she says, should … Continue reading →

Michael Scott, a ViaCyte vice president, holds the VC-01 device that holds progenitor cells that will mature to make insulin and other hormones. More than ever before, stem cell therapies appear poised to transform medicine potentially curing heart disease, diabetes and paralyzing injuries, among other ailments. But its also clear that such innovations will be very expensive. How the government, insurers and patients will pay for what could be a flood of these new treatments drew the attention of more than 700 biomedical and health-care executives Tuesday at the 2014 Stem Cell Meeting on the Mesa. The annual conference, held on La Jolla's Torrey Pines Mesa, will run through Thursday. It brings together the business and academic worlds of cell therapy, including but not limited to stem cell treatments. In California alone, 131 clinical trials are taking place with stem cells, according to Clinicaltrials.gov, a government website that tracks clinical trials. Patients are being treated for conditions such as blindness from retinal diseases, HIV, leukemia, sickle cell disease, stroke and aging of skin. The recent proliferation of clinical trials marks great progress toward the ultimate goal of getting new treatments to patients, said stem cell researcher Jeanne Loring, who directs … Continue reading →