Case Studies

From FDA CRL to BLA Readiness: NGT Leads Regulatory Turnaround for Rare Disease Candidate

The Challenge

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NGT Approach

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Outcome / Impact

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Client Overview

A small biotech company developing bentracimab, a novel biologic, sought regulatory support after receiving a Complete Response Letter (CRL) from the FDA on its original BLA submission.

The Challenge

The FDA identified significant gaps in analytical methods, process validation, stability data, and documentation. The client needed to clarify CRL observations, align technical and regulatory strategies, and prepare for a Type A FDA meeting to secure clear guidance for resubmission.

NGT Approach

Reviewed the original BLA and information requests (IRs) to pinpoint root causes and technical-regulatory gaps.

Reframed CRL responses to ensure alignment with FDA expectations and drafted a compliant Type A briefing book meeting all format and content requirements.

Conducted multiple mock Type A meetings in Washington DC to prepare the team, with NGT consultants role-playing FDA regulators.

Participated in the in-person Type A meeting at FDA headquarters, clarifying agency feedback and unspoken expectations.

Developed strategies to ensure technical study designs and data packages fully met regulatory and ICH guidelines (Q1, Q2, Q5, M4Q).

Provided forward-looking advice to optimize lifecycle management via comparability protocols and future-proof the filing.

Outcome / Impact

1

Achieved a successful Type A meeting resulting in FDA alignment on the BLA resubmission strategy.

2

The client gained clear regulatory guidance on addressing key CRL issues, enabling the development of a robust, compliant, and data-driven resubmission plan.

3

Strengthened CMC documentation and manufacturing readiness, ensuring inspection preparedness and future submission consistency.

4

Following the positive outcome of the FDA meeting, NGT was entrusted to lead the development of the comprehensive BLA resubmission, taking ownership of the strategy, structure, and authoring process. Working closely with the Sponsor, CDMOs, and investment stakeholders, NGT guided cross-functional collaboration to deliver a cohesive, compliant, and submission-ready BLA package—reflecting regulatory best practices and stakeholder alignment.