CSL Behring Germany has announced that the first haemophilia B patient in Germany was treated with the gene therapy Hemgenix (etranacogene dezaparvovec) at the Hemophilia Treatment Center at Hannover Medical School. Hemgenix is the first one-time gene therapy approved in Europe for the treatment of adults with severe… Read More
The Food and Drug Administration (FDA) has authorised Spinogenix to begin its Expanded Access Program (EAP) for SPG302 in the United States for people with amyotrophic lateral sclerosis (ALS) that meet programme eligibility criteria. SPG302 is being developed as the first synaptic regenerative approach to treating ALS,… Read More
DDW Editor Reece Armstrong speaks to Christine MacCracken, Executive Director of Patient Strategies & Solutions at Johnson & Johnson Innovative Medicine, about expanded access strategies and the company’s decision to create a guide to help reduce barriers to investigational medicines for seriously or terminal ill patients. RA: Why are expanded… Read More
Suzanne Aitken, Senior Vice President of Managed Access at Clinigen, discusses the benefits of unlicensed medicines (ULMs) and why regulators and the industry need to do more to improve patient access to these therapies. The pharmaceutical industry is constantly changing. However, an unwavering problem is the constant unmet need… Read More
One-third (32%) of rare disease patients are not aware they can access unlicensed medicines through Early Access Programmes, new research has revealed. However, two-thirds (68%) of patients are comfortable receiving medicines before they’re licensed through their country’s healthcare system. The new data was released… Read More
England will become the first country in Europe to roll out darolutamide (Nubeqa) to patients whose prostate cancer has spread to other parts of the body. The drug works by blocking androgen receptors in cancer cells, which in turn blocks the effect of testosterone that allows the… Read More
