Challenge:
Our client was a biotechnology company developing life-enhancing gene therapies designed to restore vision in patients suffering from blindness due to inherited retinal diseases. The company's approach to using optogenetics in vision restoration was based on pioneering, proprietary research conducted at leading academic institutions to develop adeno-viral products. In order to support clinical development of its lead therapuetic, the client requsted Alacrita's support in obtaining EU Orphan Drug approval.
Solution:
Alacrita's process involved shaping the EU orphan drug application from one that had already been approved in the US by:- Preparation of Application form- Review and adaptation of Sections A-E to meet EU requirements (including literature searches and drafting of text to support prevalence of condition in EU)- Co-ordinating translations of active ingredient and orphan indication- Compiling the bibliography- Publishing and submitting the final application.
Our Regulatory Affairs Expertise
Our regulatory affairs consultants can help you devise effective regulatory strategies to place you on the best path torward regulatory approval, helping you avoid regulatory delays and overcome critical regulatory hurdles. We also provide hands-on support, including assistance with regulatory applications and eCTD publishing. Our experts can work alongside and supplement your internal regulatory teams to solve bandwidth challenges. We can also provide interim regulatory affairs leadership during periods of transition. Additionally, our team conducts regulatory due diligence both for internal purposes as well as for external parties engaged in licensing, investment, and M&A activities.