A successful Initial Targeted Engagement for Regulatory Advice on CBER products (INTERACT) meeting with CBER paved the way for the early product manufacturing activities and very early preclinical safety and efficacy evaluations. A pre-Investigational New Drug (Pre-IND) meeting provided additional clarity on the IND-enabling activities needed to support an initial IND submission in support of a Phase 1 first-in-human (FIH) study.
The FDA published a new draft guidance on August 10, 2018 entitled, Expansion Cohorts: Use in First-In-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics. The comment period for the draft guidance closed on October 12, 2018 and approximately 20 comments were submitted to the docket. The guidance provides sponsors with recommendations for designing and conducting first-in-human (FIH) multiple expansion cohort trials within their oncology development programs.
CMOs are positioned to play a leading role in standardizing control strategies for gene therapy because so many gene therapy products are funneled through CMOs for commercial scale production.
The regenerative medicine sector is at a remarkable moment. Transformative products are now on the market and accessible to greater numbers of patients every day. Dozens of additional therapies are in late stage studies. The regulatory and policy environment has evolved rapidly alongside the science, enabling a surge of incoming innovation.
A number of challenges are hindering efforts to develop cell and gene therapies to treat rare diseases. While these challenges may seem common across other drug markets, in the case of rare diseases, they are exacerbated by limited patient populations
The FDA has been issuing guidance documents addressing gene therapy development issues for approximately 20 years — a remarkable dedication of resources to an area that did not have a licensed product until 2017. Of the six gene therapy-related draft guidances the agency issued last month, two represent the first of the “suite of disease-specific guidance documents on the development of specific gene therapy products” Commissioner Scott Gottlieb promised in Dec. 2017.
Cell and gene therapies: Will their use become ubiquitous, changing the landscape forever as many have promised? Or will applications be confined to rare diseases as they have been thus far?
The FDA recently issued six draft guidance documents. This article discusses the guidance Human Gene Therapy for Hemophilia.
This is the fifth article in a six-part series summarizing each of the FDA's gene therapy draft guidance documents. It will discuss the draft guidance Human Gene Therapy for Retinal Disorders.
The FDA recently issued for public comment six draft guidance documents. This is the fourth article in a series and will discuss the guidance Human Gene Therapy for Rare Disease.