Novartis AG, which previously announced positive interim trial results for the company’s experimental gene therapy for spinal muscular atrophy, said an investigation is underway into whether a second trial death could be related to the treatment.
Current health systems built to evaluate and pay for traditional drugs are going to struggle with gene and other unusual therapies – but manufacturers can take steps before launch to define how manufacturers assess and value these new products.
Regeneron Pharmaceuticals is investing $800 million in Alnylam Pharmaceuticals to develop treatments for eye and central nervous system diseases using gene-silencing RNA interference technology.
Biogen Inc. should slash the price of the spinal muscular atrophy (SMA) drug Spinraza, and the $4 million to $5 million Novartis has said the company’s experimental gene therapy Zolgensma for the disease is worth is excessive, an independent U.S. organization that reviews the value of medical treatments said.
The U.S. Food and Drug Administration approved ADMA Biologics’ Asceniv to treat primary humoral immunodeficiency disease (PIDD or PI) in adults and adolescents.
An advisory committee of the EMA recommended conditionally approving a drug from Akcea Therapeutics and Ionis Pharmaceuticals that aims to treat a rare genetic disease.
Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.
Here are seven trends most likely to be hitting the news cycle in 2019 according to BioSpace.
Akcea Therapeutics Inc. planned to cut its workforce by about 10 percent after the U.S. health regulator declined to approve the company’s Waylivra drug for treating a genetic disease that causes fat accumulation in blood.
The U.S. FDA declined to approve a drug from Akcea Therapeutics and Ionis Pharmaceuticals that aims to treat a genetic disease that causes fat accumulation in the blood.
U.S. health regulators expanded the use of Vertex Pharmaceuticals Inc.’s cystic fibrosis drug Kalydeco to include patients aged between 12 months and 24 months.