Setback for LHON Treatment in the US: IMP Supports Urgent Patient Need
International Mito Patients (IMP), the global federation representing patient organisations for mitochondrial diseases, expresses disappointment following the decision by the U.S. Food and Drug Administration (FDA) to issue a Complete Response Letter regarding the New Drug Application for Idebenone for the treatment of Leber’s Hereditary Optic Neuropathy (LHON).
According to the announcement, the FDA determined that the application could not be approved in its current form and requested additional clinical data from adequate and well-controlled studies, as well as clarification on certain manufacturing and nonclinical information. Importantly, the agency did not identify new clinical safety concerns related to idebenone.
LHON is a rare inherited mitochondrial disease that leads to rapid and severe vision loss, often affecting young adults and profoundly impacting independence, education, employment, and quality of life.
“We are deeply disappointed by the impact this decision may have on people living with LHON in the United States, where there are currently no approved therapies specifically for this condition,” said Paula Morandi, Chair of International Mito Patients. “For individuals facing the devastating and often sudden loss of vision caused by LHON, access to effective treatment options is urgently needed.”
While the FDA decision applies only to the regulatory process in the United States, Idebenone continues to be available in several other countries where it has been approved for the treatment of LHON.
“Although this outcome is discouraging for the U.S. LHON community, it does not diminish the progress made through years of research and collaboration among patients, clinicians, researchers, and industry partners,” Morandi added. “We remain grateful to everyone who has contributed to advancing the understanding of this disease and to developing potential therapies.”
International Mito Patients reaffirmed its commitment to working with the global mitochondrial disease community, regulators, researchers, and industry partners to accelerate the development of safe and effective treatments.
“We will continue advocating for the needs of people living with LHON and for stronger international collaboration to ensure that innovative therapies can reach patients who urgently need them,” Morandi said.
Click here to read the full press release from those behind the drug Idebenone, Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases.