Advertisement Santhera receives EMA acceptance for idebenone MAA review - Pharmaceutical Business review
Pharmaceutical Business review is using cookies

ContinueLearn More

Santhera receives EMA acceptance for idebenone MAA review

Santhera Pharmaceuticals has received the European Medicines Agencys (EMA) acceptance for reviewing its marketing authorization application (MAA) of idebenone for the treatment of Leber's Hereditary Optic Neuropathy (LHON).

The MAA submission was based on the RHODOS study results which was, a placebo-controlled, randomized trial and has demonstrated efficacy of idebenone in LHON.

The MAA also includes nonclinical data from a disease-relevant model of LHON which demonstrated that retinal ganglion cell death and other retinal pathologies induced by mitochondrial complex 1 inhibition (the mechanism by which LHON causes the blindness in humans), could be prevented with idebenone.

Santhera chief scientific officer Thomas Meier said the MAA emphasizes the consistency of the RHODOS results across all visual acuity endpoints and analyses favoring Santhera’s idebenone over placebo, and the clinically meaningful benefit afforded to LHON patients.