Regulatory Strategy with Scientific Input
Regulatory Operations
Need
Asian biotechnology company with a Central Nervous System (CNS) compound originally developed to treat a neurological disorder
Preclinical pharmacology studies revealed potential for additional neurological indication
Assistance requested with the preparation and filing of a new Investigational New Drug (IND)
Approach
Reviewed original IND filing and provided a detailed gap analysis and regulatory strategy for the new indication
Prepared and filed the new IND to FDA on time within an aggressive target timeline
FDA placed the clinical program on a partial clinical hold due to nonclinical data
Developed a regulatory strategy and assisted in the design of a pivotal nonclinical toxicology study to provide conclusive data to FDA for removing the partial clinical hold
Interacted with an academic consortium of leading Neurology experts to design and implement a novel, Phase IIa clinical proof-of-concept study, which allowed initial patient cohorts to be treated while under a partial clinical hold
Submitted a complete response to the partial clinical hold and initial safety data on the Phase IIa study within 90 days of being placed on partial hold
FDA removed the hold 30 days after submission of the complete response, thereby allowing full development of the compound to proceed
Benefits
Leveraged existing nonclinical and Phase I safety and PK data to allow an initial proof-of-concept study as the IND-opening clinical trial
Obtained removal of the partial clinical hold and effective FDA input into the design of the proof of concept clinical study allowing the client to meet all critical milestones
Completion of the multi-site, proof-of-concept trial on time , with the positive results supportive of the new target indication
Quality ICH-consistent Clinical Study Report prepared on time
