Landmark Month Sees Second Gene Therapy Approved and Other Advances
October was a landmark month for gene therapies with the approval of the second-ever gene therapy in the US, Yescarta (axicabtagene ciloleucel) from Kite and Gilead. The prior approval of Novartis’ Kymriah is more proof that gene therapies are set to shake up the pharma industry. Gene therapies saw other wins as Luxturna, Spark’s treatment for Leber congenital amaurosis, received the FDA’s recommendation for approval and Bluebird Bio’s interim data report for its gene therapy Lenti-D to treat cerebral adrenoleukodystrophy showed promising results.
Planning for Gene Therapy Success
With clinical trial successes bringing gene therapy closer to FDA approval and eventual commercialization, it's critical for companies developing gene therapies to understand the marketplace and plan accordingly.
RxC International has put together a white paper containing important information on the current state of gene therapies, companies driving the market forward, and what to consider when beginning the process of commercialization.
Gene Therapy Articles and Other Resources
- Gene Therapies: Pathways for Commercial Success: Commercial success for a gene therapy requires as much consideration as regulatory approval. In this article, RxC International provides key insights into the gene therapy market in conversation with ICER’s white paper for the commercial entrance of gene therapies.
GENE THERAPY: Understanding the Science, Assessing the Evidence, and Paying for Value: The Institute for Clinical and Economic Review (ICER) convened a policy summit in late 2016 to evaluate potential value and challenges of gene therapy treatments. This is a report of their findings and suggestions.