Santhera Announces Intent To File Marketing Authorization Application for Leber's Hereditary Optic Neuropathy

LHON is a rare genetic eye disease that leads to a rapid loss of central vision and ultimately to blindness as a result of nerve cell degeneration in the retina and optic nerve. Catena® is the first drug that has ever been clinically investigated in a controlled clinical study in this neuro-ophthalmological disorder. An estimated 20,000 LHON patients live in Europe and in the United States. The global market potential is currently estimated to exceed USD 700 million annually excluding preventive treatment (Source: Datamonitor 2010). Santhera holds global rights on the use of Catena® as a treatment of LHON and has been granted orphan drug designations in the European Union and the United States.

"We are pleased with the prospect of bringing Catena® to market as the first treatment for LHON. Our position is that the consistent efficacy data, which are robust despite the small sample size, in combination with the excellent safety profile, results in a positive benefit/risk balance for our drug," said Klaus Schollmeier, Chief Executive Officer of Santhera. "We believe this innovative product, if approved, could meet a significant and unmet demand."

Data from the RHODOS study support the use of Catena® in LHON. Specifically, the data show that the drug can protect patients who are at highest risk of vision loss and enhance the recovery of visual acuity in severely affected eyes. These data are supported by secondary efficacy variables such as clinical benefit, color contrast sensitivity and changes in retinal nerve fiber layer thickness. The study conduct and analyses were in compliance with recommendations received from regulatory authorities.


Contact:
Klaus Schollmeier Chief Executive Officer T.: 061 906 89 52 E.: klaus.schollmeier@santhera.com