FDA sees early promise in new gene therapy for BAG3-associated DCM
Affinia Therapeutics, a Massachusetts-based biotechnology company, received some good news from the U.S. Food and Drug Administration (FDA) about one of its investigational gene therapies. Could a full FDA approval be in the cards?
The company has received the FDA’s orphan drug designation for AFTX-201, a new one-time infusion for patients with BAG3-associated dilated cardiomyopathy (DCM). This means Affinia now has access to certain benefits—including tax credits and fee exemptions—designed to help with the drug’s development. In addition, if the FDA does go on to approve AFTX-201, this status creates the potential for seven years of market exclusivity before competitors can start selling their own version of the therapy in the United States.
A clinical trial, UPBEAT, is already underway to explore the treatment’s potential to treat patients who present with heart failure-related challenges impacting their quality of life.
BAG3 DCM is caused by a mutation in the patient’s BAG3 gene. It is associated with an early onset of heart failure symptoms and an impaired quality of life. An estimated 2-3% of patients with DCM actually have BAG3-DCM.
“AFTX-201 is designed to address the underlying root cause of BAG3 DCM,” Hideo Makimura, MD, PhD, Affinia’s chief medical officer, said in a statement. “The UPBEAT clinical trial is actively recruiting at multiple institutions and we look forward to continue progressing the AFTX-201 clinical program so that we can bring this potentially transformative treatment to people living with BAG3 DCM.”
“BAG3 DCM is a devastating heart disease with a known genetic cause,” added Greg Ruf, founder and executive director of the DCM Foundation. “It is a condition that leads to early onset, progressive heart failure and premature death, with no approved treatment that addresses the underlying mechanism. The DCM Foundation and Genetic Cardiomyopathy Awareness Consortium commend the FDA for awarding orphan drug designation to AFTX-201 for the treatment of BAG3-associated DCM, acknowledging the pressing demand for innovative treatment alternatives for rare diseases like BAG3 DCM for which there remains significant unmet medical need.”
Michael has more than 19 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.