Confluence Pharma inks deal with AOP Orphan for Fragile X syndrome drug

Privately-held USA-based Confluence Pharmaceuticals and Austria’s AOP Orphan Pharmaceuticals have signed an agreement to co-develop and market Confluence’s lead asset, a product for the treatment of Fragile X Syndrome in Europe and the Middle East.

Confluence has received Orphan Designation in both Europe and the USA for its lead product. With Orphan Designation and pre-Investigational New Drug (IND) guidance, the two companies anticipate an accelerated regulatory path to approval for Fragile X syndrome. In addition, the companies may qualify for several important benefits such as administrative and procedural assistance, smaller clinical trial sizes, fee reductions and exemptions from administrative expenses, prolonged market exclusivity upon approval and facilitated market access based on Health Technology Assessments of Orphan Drugs.

No approved treatment for the condition at present