14 January 2025 – This week in clinical research brings two critical updates reflecting both therapeutic advances and regulatory modernization.

1. ViGeneron’s VG901 Earns FDA Rare Pediatric Disease Designation
   The U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation (RPDD) to VG901, an innovative AAV-based gene therapy targeting retinitis pigmentosa (RP) caused by CNGA1 mutations. VG901 is currently under investigation in a Phase 1b clinical trial (NCT06291935).

This designation underscores VG901’s potential for addressing an area of high unmet need in visual impairment—and may qualify the therapy for priority review and eventual eligibility for a Rare Pediatric Disease Priority Review Voucher.

2. EMA’s CTIS Newsflash Spurs Clinical Trial Transition to CTR
   On 14 January 2025, the European Medicines Agency released its latest CTIS Newsflash, urging sponsors to transition ongoing clinical trials expected to continue beyond 30 January 2025 from the outdated Clinical Trials Directive (CTD) to the newer Clinical Trials Regulation (CTR)

The EMA emphasized:

• Submissions under CTD must be wrapped up swiftly.
• Sponsors should utilize provided resources such as fee structures, templates, and updated FAQs.
• The Accelerating Clinical Trials in the EU (ACT‑EU) initiative is surveying academia and SMEs to tailor clinical trial training, with responses due by 11 February 2025

Strategic Importance

ViGeneron’s VG901 move is a vital milestone in gene therapy for inherited retinal disorders—RPDD not only recognizes the therapy’s promise but may also accelerate its development and enhance investor and patient interest.

At the same time, EMA’s CTR transition mandate marks a critical shift in the European clinical trial landscape. By enforcing a deadline and providing support tools, the Agency aims to improve transparency, regulatory efficiency, and harmonization across the EU. The ACT-EU survey further suggests an inclusive approach—aiming to close skill gaps across diverse stakeholders.

Together, these developments highlight how clinical research is evolving—from breakthrough therapeutic pathways to levers of regulatory agility that underpin trial design and execution.