WALTHAM, Mass. — Affinia Therapeutics said the European Medicines Agency has granted Orphan Drug designation to its investigational gene therapy AFTX-201 for the treatment of BAG3-associated dilated cardiomyopathy, a rare and often fatal inherited heart disease with no disease-modifying therapies currently available.
BAG3-associated dilated cardiomyopathy is caused by mutations in the BAG3 gene that lead to progressive loss of heart function at a young age. Despite standard treatments, nearly 25 percent of patients ultimately require a heart transplant. Affinia said AFTX-201 is designed to address the genetic root cause of the disease and has the potential to be a first-in-class and best-in-class therapy.
“BAG3 DCM is an inherited heart disease with a high mortality rate that results in loss of heart function at a young age,” said Fernando Dominguez, M.D., Ph.D., a cardiologist at the Heart Failure and Inherited Cardiac Diseases Unit, Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda in Madrid. “Despite current standard of care, there are no disease-modifying treatments available and nearly 25% of patients require a heart transplant. A one-time therapy that addresses the genetic root cause of BAG3 DCM could be transformative and make a real difference for those living with BAG3 DCM.”
AFTX-201 delivers a full-length, fully human BAG3 transgene using Affinia’s proprietary adeno-associated virus capsid engineered for efficient cardiac transduction. The company said the therapy is designed to achieve therapeutic effects at doses five to 10 times lower than gene therapies that use conventional capsids such as AAV9 or AAVrh74. The treatment is intended to be administered as a one-time intravenous infusion.
Preclinical studies in animal models demonstrated that AFTX-201 increased BAG3 protein levels in the heart and fully restored cardiac function, according to the company.
“The EMA Orphan Drug designation indicates the importance of developing new treatment options for rare diseases such as BAG3 DCM,” said Hideo Makimura, M.D., Ph.D., Chief Medical Officer at Affinia. “This designation, along with the recent FDA acceptance of our IND application for AFTX-201, reinforces our belief that our investigational medicine has the potential to be the best-in-class treatment option for those affected by this devastating disease.”
The EMA grants Orphan Drug designation to therapies intended to treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union. The designation provides incentives that include regulatory support, reduced fees, and up to 10 years of market exclusivity following approval.
Affinia said BAG3-associated dilated cardiomyopathy affects more than 70,000 patients across the United States, Canada, the European Union, and the United Kingdom, and remains an area of significant unmet medical need.
