Confluence 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.
“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.
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."
About Fragile X Syndrome
Fragile X Syndrome (FXS) is associated with an array of intellectual and emotional disabilities impacting 180,000 individuals in the U.S. and 213,000 in Europe. 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 (FMRP). FXS is the most common inherited form of mental impairment and the most known cause of Autism.
About Confluence Pharmaceuticals, Inc.
Confluence Pharmaceuticals, an Indianapolis, IN based company is developing novel treatments for core impairments associated with Fragile X Syndrome and Autism Spectrum Disorders. Dr. Craig A. Erickson while at Indiana University conducted the foundational work and the initial IP was exclusively licensed to Confluence by Indiana University. Confluence has assembled a team of industry experts experienced in drug development, clinical trials and regulatory affairs to accelerate the development of novel drugs, and formulations for FXS and ASD.
About AOP Orphan Pharmaceuticals AG
AOP is a pharmaceutical company specializing in the development and marketing of medicines for rare and complex diseases in Haematology/Oncology, Cardiovascular Disease, Metabolic Disorders and Neurology. The company is represented in all Central European countries and the Middle East. AOP also commercializes its products in South East Asia, South Africa and South America with a series of distribution partners. The Company is headquartered in Vienna, Austria.