Case study:
End-to-end regulatory support for a gene therapy MAA submission
Background:
Boyds was engaged by a US-based biotechnology company to provide regulatory consultancy services for the preparation and submission of a Marketing Authorisation Application (MAA) for a novel gene therapy in the European Medicines Agency (EMA) region. The company required full-service support to coordinate all pre-submission activities, regulatory interactions, and technical documentation to achieve a successful outcome.
Boyds approach
Our regulatory experts delivered comprehensive support across the entire MAA preparation and submission process, ensuring strategic alignment, technical rigour, and adherence to EU requirements.
We led the preparation of all key pre-submission documentation, including the Letter of Intent, legal basis and eligibility request, the EMA pre-submission meeting request, supporting documents, and the product overview. We also completed the application form and annexes, drafted product information (SmPC, labelling, package leaflet) using the latest QRD template, and reviewed labelling mock-ups and the Patient Information Leaflet (PIL) for compliance.
To ensure a robust safety profile, our team reviewed the Risk Management Plan (RMP) and pharmacovigilance (PV) system documents, and coordinated compliance checks across labelling and clinical summaries. We compiled the regulatory Modules 1 and 2, including scientific advice, orphan designations, and declarations, and prepared the cover letter, ATC code request, and invented name submission. We also supported the company’s ATMP classification and request for accelerated assessment.
Boyds managed all regulatory interactions, including planning and participating in pre-submission meetings with the EMA and Rapporteurs, developing briefing documents, maintaining and transferring orphan designations, and managing SME communications such as fee exemption requests.
Our team conducted compliance checks on paediatric requirements, provided detailed review and feedback on Module 2 documents, and supported submission and validation of the finalised MAA. Throughout the 15-month engagement, we provided ongoing project management, ensuring seamless coordination between stakeholders, regulatory authorities, and internal teams to maintain project momentum.
Impact and Outcomes
Boyds provided end-to-end regulatory support tailored to the gene therapy’s specific requirements, ensuring timely preparation for EMA engagements and submission readiness.
The MAA was successfully submitted and approved, with Boyds delivering continuity, strategic insight, and technical depth across all scientific, operational, and regulatory activities.