Leber’s Hereditary Optic Neuropathy (LHON)

LHON is a genetic disease that results in the degeneration of nerve cells in the retina and optic nerve, leading to the rapid loss of central vision and blindness. The disease affects young adult males predominantly.

The effects of LHON are rapid and severe, with the damage to retinal and optic neurons cells leading to blindness within a few months after the onset of symptoms. The symptomatic phase of the disease begins with blurring of central vision. Both eyes are usually affected within several months of symptom onset in the first eye. Within 12 months over 97% of patients will experience vision loss in the second eye, most often leaving them severely visually impaired.

Santhera has been granted orphan drug designation in the US and Europe for SNT-MC17 in LHON.

Clinical development of SNT-MC17 (INN: idebenone) in LHON

Santhera is currently recruiting patients for a Phase IIa (proof-of-concept) at the University of Munich in Germany and at the University of Newcastle in the UK.

The study, which is a double-blind, randomized and placebo-controlled trial, is designed to assess the efficacy of SNT-MC17 in the treatment as well as the prevention of vision loss in LHON patients. 84 LHON patients will be recruited for the study and they will be treated for a period of six months. Patients experiencing vision loss for up to 5 years are eligible to enroll.

SNT-MC17 for treatment of Leber's Hereditary Optic Neuropathy / Leber hereditäre Optikusneuropathie / Neuropathie Optique Héréditaire de Leber