Spark Therapeutics Inc. has priced its new gene therapy for a rare form of blindness at $850,000. That may not seem like much of a bargain, but it starts to sound better when you consider that the industry expected a cost of $1 million. The company said the price tag for voretigene neparvovec-rzyl (Luxturna)…
The FDA has approved a label update for secukinumab (Cosentyx, Novartis) that includes data on treating moderate-to-severe scalp psoriasis, a difficult-to-treat form of the disease that affects approximately half of all psoriasis patients. The label update is based on the results of a dedicated phase…
An FDA advisory committee has declined to recommend approval of a proposed new oral testosterone replacement therapy (Tlando, Lipocine Inc.). Lipocine said the Bone, Reproductive, and Urologic Drugs Advisory Committee (BRUDAC) voted six in favor and 13 against the benefit–risk profile of Tlando, which…
The FDA has approved a lidocaine topical system (ZTlido, Sorrento Therapeutics, Inc.), 1.8%, for the relief of pain associated with post-herpetic neuralgia (PHN) that Sorrento bills as “a major advancement in analgesics” because of improved adhesion. “ZTlido was designed to solve a problem that…
A new study at Columbia University Irving Medical Center (CUIMC) challenges the popular notion that psychiatric medications are overprescribed in U.S. children and adolescents. When the researchers compared prescribing rates with prevalence rates for the most common psychiatric disorders in children,…
ASP0113 (Astellas Pharma Inc./ Vical Incorporated), an investigational DNA vaccine being developed for cytomegalovirus (CMV)-seropositive hematopoietic stem cell transplant recipients, did not significantly improve overall mortality and CMV end-organ disease through the first year following the transplant,…
The flu-related deaths of 22 more children were reported during the week ending February 10, federal officials say, bringing the pediatric toll to 84 for this grim flu season. Nearly 20,000 people have been hospitalized so far, with weeks to go. Several of the childhood deaths occurred weeks earlier…
Positive results have been announced from ACHIEVE I (UBR-MD-01), the first of two pivotal phase 3 clinical trials evaluating the efficacy, safety, and tolerability of Allergan’s orally administered ubrogepant 50 mg and ubrogepant 100 mg compared with placebo in a single migraine attack in adults.…
A U.S. map of flu activity issued Friday by the Centers for Disease Control and Prevention (CDC) was a sea of red: the color marking the highest levels of outpatient visits for an illness that continues to worsen this season. According to this week's CDC report, covering the week of January 7 to 13,…
Cabozantinib (Cabometyx, Exelixis, Inc./Ipsen) improved overall and progression-free survival among previously treated liver cancer patients in the pivotal phase 3 CELESTIAL trial, according to results being presented at the 2018 ASCO-GI Symposium in San Francisco. At the planned second interim analysis…
A one-month antibiotic regimen to prevent active tuberculosis (TB) disease was at least as safe and effective as the standard nine-month therapy for people living with human immunodeficiency virus (HIV) infection, according to the results of a large international clinical trial. Adults and adolescents…
Hospitalizations blamed on the flu may be on track for a record in this increasingly challenging flu season, federal officials said Friday, and 16 more pediatric deaths were reported during the week. “This is a very difficult season. The hospitalization rate is the highest we’ve seen,” said…
Positive results have been announced from the phase 3 PROSPER trial in patients with nonmetastatic castration-resistant prostate cancer (CRPC). The results show that the use of enzalutamide (Xtandi, Astellas Pharma/Pfizer) plus androgen deprivation therapy (ADT) significantly reduced the risk of developing…
The FDA has rejected the new drug application (NDA) for Linhaliq (Aradigm Corporation) as a treatment for non-cystic fibrosis bronchiectasis (NCFBE) patients who have chronic lung infections with Pseudomonas aeruginosa. Aradigm said it received a complete response letter (CRL) stating that the agency…
Researchers at Massachusetts Institute of Technology (MIT) and Brigham and Women’s Hospital have developed a capsule that can deliver a week’s worth of human immunodeficiency virus (HIV) drugs in a single dose. This advance could make it much easier for patients to adhere to the strict schedule…
