With Dr. Marks’ (Director, Center for Biologics Evaluation and Research), recent perspective on FDA’s Effort to Advance the Development of Gene Therapy, he thoughtfully highlights the recent successes of approved gene therapies, highlights the expert resourcing within FDA, CBER and NIH being applied towards this therapeutic specialization and details the innovative programs and committees (Novel and Exceptional Technology and Research Advisory Committee (NExTRAC), Regenerative Medicine Advanced Therapy (RMAT) designation and the INTERACT program) being instituted and enacted by the FDA. In addition, he highlights the critical issue facing success of this scientific endeavor, is quality manufacturing capacity but attentively details FDA action of awarding grants dedicated to industry stakeholders for the advancement of manufacturing technologies.
At Eurofins BioPharma Services, we feel this FDA press release, as a Christmas gift that came early! After working on multiple protocols, across multiple clients, we couldn't be happier to see the continued evolution of treatment solutions for this rare disease (around 2 cases per million). Prior to the first treatment being released in 2007, life expectancy of patient diagnosed with PNH was only ten years, with poor quality of life throughout that time. With the significant resource dedication of years of effort and capital, our peers and colleagues throughout the clinical trial industry have allowed patients to have:
After being in the draft stages for approximately three years and continuing to receive industry feedback, the FDA CDER has just released final Guidance for Industry Title Testicular Toxicity: Evaluation During Drug Development in October 2018. There are numerous challenges with conducting multi-center clinical trials focusing on testicular toxicity and yet remaining within the realm of guidance from the FDA per their statement: