INSIGHTS ON CELL & GENE REGULATORY ISSUES

• The 505(b)(2) Pathway: Getting To The Clinic Faster

  Discover the advantages of the 505(b)(2) pathway, look at common misconceptions, and offer practical advice on its application in the real world.

• Aligning For 505(b)(2) Success: Oncology Combination Product First-In-Class Treatment Success

  Discover why it is necessary to have the right regulatory and commercial plan in place early on based on sound science and medical, regulatory, and commercial assessments.

• Improving NDA Approval Odds For New Dosage Forms Of Approved Products

  Learn why providing a more convenient and/or faster-acting dosage form of a well-chosen drug provides significant benefits for patients.

• CGT’s Top Regulatory Challenges Broken Down By Segment

  Our recent Cell & Gene Live, Tackling Cell & Gene Therapies’ Top 3 Regulatory Impediments, featuring BridgeBio's Dr. Adora Ndu and REGENXBIO's Dr. Nina Hunter, delivered just under 60 minutes of exceptionally valuable information. We also know that your time is likely not your own, which is why we’ve broken down the full-length version into digestible segments that you can view on your own time.

• Navigating Regulatory Pathways For Digital Therapeutics

  This blog post will help sponsors gain clarity regarding what is and what is not considered a DTx and how to ensure they are following the correct regulatory pathway for their device.

• How Do Cell & Gene Therapy Requirements Differ Between FDA & EMA?

  This article examines the similarities and differences between how the FDA and the European Medicines Agency (EMA) approach cell and gene therapies. Where do the regulatory perspectives align and where do they diverge?

• Expert Regulatory Filings To See Your Regulatory Strategy Through

  Veristat’s regulatory publishing team offers extensive expertise across the full scope of regulatory requirements.

• FDA Releases Final Guidance On Early-Phase Cell & Gene Therapy Trials

  The cell and gene therapy development process involves screening different variants of a product type. To aid this process and to ensure that suitable evidence is gathered so that effective variants of the product are taken through to later-stage trials, the FDA has produced a final guidance, Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial.

• EMA's Revised GMP Annex 1 Addresses Common Global Challenges for Sterile Products

  The new EMA GMP Annex 1, “Manufacture of Sterile Medicinal Products," is in line with other international regulations, e.g., ICH and PIC/S. FDA involvement in its writing is a clear indication that the contents will be the FDA position in general terms. Fortunately, the annex contains many checklists to help you assess and remedy your systems.

• Considerations For Developing Rare Disease Treatments

  When developing a new therapy for a rare disease, learn why a thoughtful, strategic approach early in the development process supports a well-designed study and agreement to your approach from regulatory authorities.