Gene Therapy Product Pipeline

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

Solution

• Acting on behalf of the company as their EU RA Department. Real Regulatory essentially runs all EU activities on behalf of the client
• Drive the regulatory portion of the project teams, provide and build required submission documentation with client input, manage all interactions with the EU agencies and drive the strategy for all EU regulatory development

Result

• Company is appended on the Real Regulatory EMA SME status to avail of fee incentives. Company has no EU-based legal company entity
• ATMP Classifications achieved for 4 products
• EU Orphan Medicinal Product Designation achieved for 2 products
• Scientific Advice meetings completed with Finland for 1 product & Germany for 2 products