Overview

A US-based gene therapy company sought G&L Healthcare Advisors' expertise to navigate the complex regulatory landscape in Japan for its hemophilia gene therapy program.

Intending to simultaneously launch the product in Japan, the US, and the EU, the company needed full regulatory support, local market insights, and strategic planning to meet the requirements of Japan’s Pharmaceuticals and Medical Devices Agency (PMDA).

Challenges

The company faced several key challenges in bringing its gene therapy program to the Japanese market:
In-country regulatory support: They required a local Japanese team to manage the regulatory filings and PMDA interactions.
Gap analysis: A thorough review of the global development program was necessary to ensure that the data and CMC (Chemistry, Manufacturing, and Controls) were acceptable to Japanese authorities.
Local study requirements: They needed guidance on Japanese bridging studies and Clinical Trial Notification (CTN) applications.
PMDA meetings: Successfully navigating the PMDA’s regulatory process required precise planning and expertise in Japanese regulatory affairs.

G&L’s Approach and Solution

In collaboration with its Japanese affiliate, G&L provided comprehensive regulatory support and developed a strategic plan tailored to Japan’s market. This included:
Feasibility assessment: G&L’s Japanese affiliate conducted a feasibility study, evaluating the potential for the gene therapy’s entry into the Japanese market, and addressing key regulatory considerations.
Gap analysis of CMC and data: G&L performed a detailed gap analysis of the available CMC, non-clinical, and clinical data for the therapy, which was undergoing Phase III trials. This analysis identified any discrepancies or additional requirements for the Japanese submission.
Development of a regulatory pathway and clinical plan: A comprehensive clinical and regulatory development plan was designed to guide gene therapy through the Japanese Marketing Authorization Application (MAA) process, ensuring compliance with local requirements.
PMDA consultation leadership: G&L led the client through a series of critical PMDA consultation meetings. These meetings were crucial in obtaining the necessary regulatory designation and approval pathway for the gene therapy product. G&L’s Japanese team, along with non-clinical, clinical, and CMC experts, managed all aspects of the PMDA interactions, including:
Development of Briefing Books for the meetings.
Designing the study protocol for the Japanese bridging study.
Leading discussions on regulatory and clinical strategies.
CTN application preparation: G&L supported the client in preparing the CTN for Japan, ensuring that all documentation and protocol requirements met the regulatory standards of the PMDA.

Impacts and Outcomes

Through G&L’s expert guidance and local regulatory knowledge, the client successfully navigated the complex requirements of the Japanese PMDA for its gene therapy program. The key outcomes included:
Clear regulatory pathway: G&L’s Japanese team established a clear and actionable regulatory pathway for the gene therapy’s MAA filing in Japan, setting the stage for a successful market entry.
Detailed project plan: A comprehensive project plan was developed, outlining the steps for ongoing PMDA engagement, local study design, and regulatory compliance.
Bridging study design: A localized study protocol was created to meet the specific requirements of the Japanese market, ensuring compliance with all clinical trial standards.
Regulatory strategy alignment: The client’s global development program was effectively aligned with Japan’s regulatory requirements, allowing for a smoother transition into the Japanese market without major delays.