Preparation of EU PIP application

Challenge

• Small US-based speciality company focused on developing medicines for rare genetic diseases. The products are classed as ATMPs and are gene therapy products for inherited neurological and metabolic syndrome disorders
• Preparation and submission of an EU PIP application, including all EMA interactions
• The product is a gene therapy product that does not fit precisely into the traditional categories of products

Solution

• All support is provided remotely, except for required attendance at agency meetings
• Plan timeframes according to EMA published timelines
• Advise client regarding EMA PIP application requirements
• Prepare PIP application documents for client review
• Liaise with EMA re: validation issues and the adoption of PDCO opinion/PDCO request for modification

Result

• Successful submission of the PIP for this unusual gene therapy product to the EMA