FDA Approves Pembrolizumab for Metastatic SCLC
June 18th 2019The FDA has granted an accelerated approval to single-agent pembrolizumab for the treatment of patients with metastatic small cell lung cancer who have disease progression on or after platinum-based chemotherapy and ≥1 other prior line of therapy.
Parkinson Disease Gene Therapy Demonstrates Continued Improvement in 6-Month Data
June 14th 2019Axovant reported positive safety data as well as improvements in a number of measurements of motor function and dyskinesias in patients with Parkinson disease who received treatment with their investigational gene therapy.
Outlining the Risks That Go Along With the Benefit of CAR T-Cell Therapy
June 5th 2019Though chimeric antigen receptor (CAR) T-cell therapy has been largely touted as one of the most important advances in cancer care in recent years, the therapy comes with the risk of severe toxicities as well as increased financial burden due to the high cost of the drugs.
FDA Approves Gene Therapy With $2.1M Price Tag for Spinal Muscular Atrophy in Pediatric Patients
May 24th 2019AveXis—a Novartis company—announced that it will work with payers to implement 5-year outcomes-based agreements and novel pay-over-time options. The company also said it will offer a patient program to support affordability and access.
Can Striatal CaV1.3 Genetic Silencing Prevent Levodopa-Induced Dyskinesias in Parkinson?
May 17th 2019New early-stage data suggest that vector‐mediated gene silencing of striatal CaV1.3 expression may hold promise for preventing the induction of levodopa-induced dyskinesias in Parkinson disease.
Ascending Dose Study Demonstrates Safety, Efficacy of Voxelotor in Sickle Cell Disease
May 15th 2019Voxelotor, a first-in-class oral therapy, is both safe and effective in sickle cell disease, according to a phase 1/2 randomized study assessing the drug. These findings were consistent across all doses, ranging from 500 to 1000 mg.
Early Trials Suggest New Path for CAR T-Cell Therapy in Solid Tumors
May 10th 2019Two novel CAR T-cell therapies designed to attack solid tumors are showing signs of antitumor activity and tolerability in early clinical trial findings, fueling optimism about expanding this emerging form of immunotherapy beyond hematologic malignancies.
New Innovations and Cures Require Overhauling Insurance and Benefit Design
May 5th 2019Healthcare is very comfortable with treating a disease, but it’s not as good with handling cures. However, the advent of gene therapy and precision medicine means more and more expensive cures are coming down the pipeline, said panelists on the last day of Asembia’s 15th annual Specialty Pharmacy Summit, held April 29 to May 2 in Las Vegas, Nevada.