UMDF is disappointed by the U.S. Food and Drug Administration’s (FDA) decision not to approve idebenone for Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that causes sudden and often irreversible vision loss.

“Far too many patients continue to face the devastating effects of vision loss from this condition,” said Kristen Clifford, UMDF President and CEO. “Idebenone has shown the ability to preserve and improve vision for LHON patients, which has led to therapeutic approvals in multiple countries. UMDF remains committed to advocating for timely access to this therapy for patients in the United States, where the need for treatment options remains urgent.”

Leber Hereditary Optic Neuropathy is a rare genetic mitochondrial disorder that primarily affects the optic nerve, leading to central vision loss. Though vision loss can onset at any age, LHON most commonly presents in adolescents and young adults and can result in permanent legal blindness. The condition is caused by one of several mutations in mitochondrial DNA that causes cell injury, particularly in the retinal ganglion cells responsible for vision. It’s estimated that about 4,000 people are legally blind due to LHON in the United States. 

Malinda Marsh, Co-Founder of LHON Collective, a global initiative dedicated to accelerating progress toward a cure for LHON while improving the lives of people living with LHON, said, “LHON Collective appreciates the FDA’s rigorous and thoughtful approach to reviewing treatments for rare diseases, but is disappointed by the FDA’s decision to not approve Idebenone for the LHON community. We are hopeful for continued dialogue and engagement as partners in the shared goal of improving outcomes for individuals affected by LHON and similar inherited optic neuropathies. Given the rapid vision loss that occurs with onset of LHON, patients do not have the luxury of time. Any vision retained leads to better quality of life and greater independence. This decision to not approve Idebenone hinders LHON medical providers’ ability to properly support their patients in achieving those goals.”

Idebenone has long been studied as a potential therapy for mitochondrial disease. It was authorized for LHON by the European Medicines Agency in 2015, where it is marketed as Raxone. In recent years, other countries including Chile, Bahrain, and Taiwan have also approved idebenone for LHON.