AOP Orphan and Confluence Pharmaceuticals, together for Fragile X Syndrome

AOP Orphan Pharmaceuticals Confluence Pharmaceuticals Fragile X

AOP Orphan Pharmaceuticals and Confluence Pharmaceuticals, signed today a definitive 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 is an American biopharmaceutical company focused on developing therapeutic treatments for Fragile X Syndrome and Autism Spectrum Disorders. The company has received Orphan Designation in both Europe and the United States for its lead product. With Orphan Designation and pre-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.

Commenting on today’s announcement, Dr. Rudi Widmann, Chief Executive Officer of AOP, said, “The cooperation between AOP and Confluence will allow AOP to further its mission for finding and developing therapies for rare diseases. AOP has a unique record of developing and distributing products for rare and complex diseases, and Confluence will benefit from AOP’s access to treatment centers and its long term expertise in commercializing drugs for rare and complex diseases.

AOP is an Austrian based company specialized in the development and marketing of medicines for rare and complex diseases as Fragile X Syndrome (FXS). The syndrome is associated with an array of intellectual and emotional disabilities from which 213,000 individuals in Europe suffer. FXS is often accompanied by anxiety, attention-deficit disorder, aggression, seizures, self-injurious behavior and physical deficits. The cause of FXS has been linked to a genetic mutation of the X Chromosome leading to a decreased or absent level of fragile X mental retardation protein. FXS is the most common inherited form of mental impairment and the most known cause of autism.

The relationship we have established with AOP is an exciting opportunity for Confluence to leverage AOP’s knowledge and expertise in Europe and the Middle East to accelerate this breakthrough treatment for the benefit of Fragile X patients,” said Confluence President and Founder Steven Johns. Today, Fragile X patients have no approved treatment for the core social and communication impairments.


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