EU SUBMISSION READINESS FOR GLOBAL PHARMA

Our regulatory experts reviewed the full non-clinical and clinical development packages, assessing whether the existing development strategy, conducted primarily in China, was consistent with EU biosimilar guidelines and scientific expectations to ensure positive outcome.

Scientific Advice Preparation and Procedure Handling

For three EMA Scientific Advice procedures, regenold:

• Conducted a detailed review of briefing documents to ensure alignment with EU biosimilar standards
• Provided strategic input on clinical, PK/PD, and analytical comparability questions
• Supported the client throughout the EMA process, including request submission, procedural handling, and follow-up via IRIS portal
• Helped the client understanding the EMA scientific advice received optimizing the outcome of the interaction

The Outcome

Through early regulatory engagement, technical review and hands-on Scientific Advice support, regenold helped the client establish a development path that is fully aligned with EU requirements, providing a clear basis for future MAA preparation and Europe-wide biosimilar launch planning.

The biosimilar program included a large, three-arm clinical study comparing the new biosimilar candidate with both EU and US reference products. regenold GmbH contributed regulatory insight to ensure that the study design met EU requirements for:

• Selection of the appropriate EU reference product
• Harmonization of EU and US expectations w. r. t. PK/PD and immunogenicity
• Endpoint alignment based on current EMA guidelines and past precedents

Outcome

The resulting clinical design met EMA expectations and positioned the client to generate a robust evidence package acceptable for EU assessment. By aligning EU and US requirements for PK/PD methodology and immunogenicity endpoints, the program advanced with a clear regulatory foundation and reduced risk of later redesign.

Client Profile

A pharmaceutical company developing a paediatric therapy across two rare disease indications, competing directly with another developer for overlapping orphan incentives.

The Challenge

With a New Chemical Entity (NCE) and two rare indications, the company aimed to maximise EU regulatory exclusivity while keeping pace with competitor timelines. Key objectives included:

• Securing sequential orphan designations
• Structuring the PIPs to maximize exclusivity for both orphan indications
• Managing submissions with precise timing to maintain competitive advantage

Our Approach

regenold designed and executed a tailored regulatory roadmap maximizing results of clinical trials vs. competitors in the same orphan space:

• Obtained approval for orphan Indication 1 for a broader patient population
• Supported the development of a PIP strategy negotiated with PDCO enabling extended orphan exclusivity
• Secured timely approval of orphan Indication 2 with +1 year exclusivity with successful demonstration of significant clinical benefit
• Managed lifecycle regulatory submissions to ensure favorable timing vis-à-vis the competitors

Regulatory Milestones Achieved

1. Marketing Authorisation for orphan Indication 1 in a broader population
2. Confirmation of full PIP compliance warranting extension of orphan exclusivity
3. Timely approval for Indication 2 with orphan maintenance and +1 year market protection
4. Competitor’s orphan application for Indication 1 rejected due failed demonstration of significant benefit over existing therapies

Outcome

regenold’s strategic coordination of ODD, PIP, timelines, and procedural actions enabled the client to:

• Maximize market exclusivity
• Reach MA earlier than the competing product
• Secure broader patient coverage
• Establish a strong competitive position in a narrow orphan landscape

This case demonstrates how precise regulatory planning — particularly around PIP compliance, orphan strategy, and timing — can be decisive in securing long-term EU market advantages.

Client Profile

US biopharmaceutical company developing a novel therapy for an inherited neurodegenerative disorder, preparing for a First-in-Human (FiH) clinical trial in the EU.

The Challenge

The client intended to submit a FiH Clinical Trial Application (CTA) via CTIS. During the initial review, regenold identified scientific and regulatory gaps that led to a shared understanding that the timing of the CTA carried high risk.

The client proceeded and the CTA was rejected by the authorities for the reasons we had anticipated.

Our Approach

regenold provided a structured regulatory redirection:

• Recommended initiating EMA Scientific Advice (SA) to address unresolved scientific and regulatory issues.
• Prepared the full SA briefing package, ensuring alignment with EMA expectations.
• Managed agency interactions and consolidated feedback into a revised development plan.
• Re-shaped the CTA content based on agreed scientific positions.

The updated CTA, now aligned with regulatory expectations, was approved in CTIS, enabling the FiH trial to begin.

Ongoing Collaboration

Following the successful turnaround, regenold was retained to support broader development activities, including:

• Preparation and submission of the Paediatric Investigation Plan (PIP)
• Orphan Drug Designation (ODD) strategy in the EU and respective documentation
• Support for a second CTIS CTA for an additional (non-orphan) indication
• Establishment of the pharmacovigilance system

Outcome

regenold’s early intervention, scientific insight, and hands-on execution transformed a rejected CTA into an approved clinical program. The client now advances multiple regulatory pathways with a clearer strategy, reduced risk, and a development plan grounded in formal EMA feedback.