A treatment that one Hall County family said had been helping their daughter’s rare illness has now been granted approval from the U.S. Food and Drug Administration.
The FDA issued a statement on Friday, September 19, saying that accelerated approval has been granted to Forzinity, which is an injection of the drug elamipretide, as the first-ever approved treatment of Barth syndrome. The illness is a rare and life-threatening disease of the energy-producing mitochondria in a person’s cells.
According to the statement from the FDA, Forzinity treats the illness by “binding to the inner part of the mitochondria, improving mitochondrial structure and function.” FDA Center for Drug Evaluation and Research George Tidmarsh said the approval is part of the administration’s work to find new ways to treat rare illnesses.
“The FDA remains committed to facilitating the development of effective and safe therapies for rare diseases and will continue to work diligently to help ensure patients with rare diseases have access to innovative treatments,” Tidmarsh said in a release…