Overview

G&L Healthcare Advisors was engaged by an EU-US gene therapy company developing a hemophilia treatment.

The client required full-service regulatory and medical writing support for their gene therapy program in Japan, aiming to launch the product in parallel with the US and EU.

This engagement required authoring of key documents for the Pharmaceutical and Medical Devices Agency (PMDA) Consultation Meetings and the submission of a Japanese Clinical Trial Notification (CTN).

Challenges

The client faced significant challenges in preparing for the Japanese market:
Local regulatory expertise: The client needed in-country support to ensure that all submissions complied with Japanese regulatory requirements.
Complex gene therapy submission: The submission process for gene therapies is particularly intricate, requiring highly specialized documentation, including clinical bridging studies and safety data.
Document translation and alignment: The team needed to manage English-Japanese translations and ensure alignment across global teams, while developing documents for PMDA consultation.

G&L’s Approach and Solution

G&L provided a dedicated team of experts with experience in Japanese gene therapy submissions. Key activities included:
In-country team engagement: G&L’s Japanese affiliate team led all clinical development and regulatory activities, including writing the Briefing Book and other documents needed for the PMDA consultation meetings.
Translation management: The team managed English-Japanese translations and worked closely with EU and US-based SMEs to ensure accuracy and consistency.
PMDA meeting preparation: G&L led the preparation for successful PMDA Japanese Agency meetings, covering non-clinical, clinical, Chemistry, Manufacturing and Controls (CMC), and Cartagena disciplines.

Impact and Outcomes

As a result of G&L’s involvement:
Successful consultation meetings: The PMDA Consultation Meetings were successful, with positive outcomes across safety, clinical, and CMC discussions.
Timely CTN submission: G&L’s support enabled the client to submit their CTN on schedule, positioning the product for launch in Japan.
Ongoing support: The client continues to rely on G&L’s expertise for ongoing regulatory submissions and future clinical development in Japan.