Overview

A mid-tier pharmaceutical client developed its first biological therapeutic - an Antibody-Drug Conjugate (ADC) for the treatment of adults with unresectable or metastatic HER2-positive breast cancer.

This ADC represented a significant breakthrough for the client’s oncology portfolio.

As they prepared to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA), the company sought Chemistry, Manufacturing and Controls (CMC) regulatory strategic advice to ensure their submission met all requirements and would be approved with minimal queries.

Challenges

The client faced several issues, including:
Limited BLA experience: This was the client’s first biological therapeutic, meaning they lacked prior internal experience with BLA submissions for biologics.
Gap analysis requirements: The critical Module 3 (CMC) portion of the BLA required a detailed gap analysis to ensure compliance with FDA standards and prevent significant Agency concerns.
Timeframe urgency: The project had to be completed within tight timelines to secure priority review and approval from the FDA.

G&L's Approach and Solution

Within this engagement, G&L took the following steps to ensure a successful outcome for the client:
Formation of specialized team: G&L assembled a team of highly skilled biologics CMC Regulatory Affairs experts and a Project Manager to support the client. The team worked seamlessly across different time zones, coordinating with the client’s US- and Japan-based Regulatory Affairs (RA) teams.
Comprehensive gap analysis: The G&L team conducted a thorough gap analysis of the entire Module 3 (CMC) portion of the BLA. This in-depth review identified potential risks or gaps that could result in FDA queries, ensuring that the CMC dossier was aligned with current FDA expectations for biological products.
Collaborative and immersive support: G&L provided immersive, collegial support throughout the project, working closely with the client’s internal teams. The project was managed dynamically, with resources expanding or contracting to meet the client’s evolving needs over the year-long engagement.
Proactive remediation strategy: By identifying critical CMC issues early on, G&L worked with the client to remediate these concerns before submission. This proactive approach ensured that the BLA was compliant and high-quality.
Timely submission: G&L's expert guidance enabled the client to submit the BLA on time according to their internal deadlines, meeting all deliverable timelines while adhering to the FDA’s stringent review processes.

Impact and Outcomes

As a result of G&L's support, the following was achieved:
Timeline expectations exceeded: G&L’s support enabled the client to exceed their deliverable timeline expectations, ensuring timely submission.
Priority review and FDA approval: The BLA was granted priority review and was subsequently approved by the US FDA in December 2019.
Ongoing client engagement: Due to the success of this project, the client has continued to engage G&L for additional Regulatory Affairs consulting.