Business Imperatives

  • An Italy-based organization encountered challenges with the US FDA due to stringent expectations for their biological products used in treating orphan diseases.
  • With the right skill set and resources, Freyr enabled seamless Regulatory pathways and simplified future action plans for handling FDA queries.

Objectives

The objective was to prepare and submit Biologics License Applications (BLA) for enzyme replacement therapy products in the USA for US FDA Approval.

Problem Statement

  • Lacked in-house expertise for BLA submissions for orphan products.
  • The US FDA identified insufficient scientific data in the application.
  • Faced increased complexity due to a change in the Drug Product manufacturing site, which had not been previously inspected by the agency.
  • Required coordination with multiple CMOs to gather source data and prepare submissions.

Freyr Solutions and Services

Freyr Solutions and Services
  • Freyr managed the procurement of source documents, preparation of dossier sections, and finalization of BLA modules.
  • Developed Regulatory submission strategies and a submission planner to ensure full compliance.
  • Conducted gap analysis on source documents.
  • Coordinated with CMOs to secure the timely availability of necessary documents.
  • Prepared BLA sections with the required information.
  • Recognized the lack of in-house expertise in BLA submissions for orphan products.
  • Ensured timely availability of documents.
  • Managed complexities due to the change in the Drug Product manufacturing site not previously inspected by the US FDA.
  • No FDA refusals of BLAs.
  • Trained the client's resources according to the Regulatory action plan.
  • Transferred knowledge to the client to handle future FDA issues independently.
  • Prepared and submitted Biologics License Applications (BLA) for enzyme replacement therapy products, resulting in US FDA approval.