European client initiating US development of its gene therapy product.
- Company beginning first discussions with the FDA regarding IND development and submission of intra-cerebral gene therapy product
- Intent to gain agreement on completed nonclinical program to support first in human study
- Intent to gain agreement on the use of historical controls to support clinical development in rare disease space.
Regulatory strategy, dossier support, and pre-IND meeting attendance.
- Assisted client with review and submission of pre-IND meeting materials
- Regulatory lead for FDA meeting discussions and management of all follow up activities
- Scope of Biopharma Excellence activities:
- Lead regulatory strategy for US based development incl. review of FDA proposed questions, meeting request letter, briefing dossier
- Preparation and attendance at FDA Pre-IND meeting.
Productive Pre-IND meeting with FDA to support IND submission.
- Gained FDA guidance on completed and intended nonclinical program to support first in human study
- Received FDA feedback on CMC requirements for manufacturing transfer and comparability process
- Collaborative discussions regarding clinical design relative to patient population, endpoints, and natural history controls