Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company focused on neuromuscular diseases, has received approval from Health Canada with conditions SNT-MC17/idebenone for the treatment of Friedreich's Ataxia. Health Canada's decision is the first marketing authorization worldwide for any Friedreich's Ataxia therapy.
The new drug will be marketed in Canada under the brand name Catena®. Santhera has established a wholly owned Canadian subsidiary, Santhera Pharmaceuticals (Canada), Inc. recently incorporated in Montréal, Quebec, to address the needs of Canada's several hundred patients with this rare and severely progressive muscle disease. Launch of Catena is anticipated for the end of October 2008.
In clinical studies submitted to Health Canada as part of the approval process, Catena showed statistically and clinically relevant improvements in Friedreich's Ataxia patients, as measured by Activities of Daily Living scores as well as cardiac and neurological functions. The approved product labeling allows for the treatment of symptoms of Friedreich's Ataxia.
Two doses are approved: a starting dose of 450 mg/day for patients below 45 kg body weight and 900 mg/day for patients above 45 kg body weight whereby treating physicians have an option to dose up to 1,350 mg/day for patients below 45 kg body weight and up to 2,250 mg/day for patients of more than 45 kg body weight if needed. Under the conditions of the Notice of Compliance with Conditions, Santhera has agreed to submit additional data from its ongoing phase III clinical trial in the United States to confirm the efficacy of the therapy.
In the United States, the IONIA (Idebenone effects On Neurological ICARS Assessments) phase III trial has currently enrolled 41 patients. It was agreed with the US Food and Drug Administration under a Special Protocol Assessment process to recruit a minimum of 51 patients but to include more patients if available. Given the current prospects for patient availability, Santhera and its US clinical investigators believe that the final study will include about 60 to 65 patients.