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Dear Health Care Professional Letter - CATENA^®

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Contact: Bureau of Cardiology Allergy and Neurological Sciences

January 20, 2010

Dear Health Professional(s):

Santhera Pharmaceuticals has previously announced that Health Canada issued a marketing authorization with conditions under the Notice of Compliance with Conditions (NOC/c) Policy for the following indication for CATENA^® tablets: "CATENA^® may be useful in the symptomatic management of patients with Friedreich's Ataxia (FRDA)". This authorization reflected the promising nature of the clinical efficacy and safety data, and the need for a confirmatory study to verify its clinical benefit.

The NOC/c was based on the promising results from a 6 month double-blind, randomized, placebo-controlled, Phase 2 study (the NICOSIA study) conducted in 48 patients aged 9 - 18 years with a genetically confirmed diagnosis of FRDA.

A second study (the IONIA study) has recently been completed. This was a double-blind, randomized, placebo-controlled Phase 3 study, also of 6 months duration, investigating the efficacy, safety and tolerability of two doses of idebenone (450 or 900 mg/day in patients ≤ 45 kg and 1350 or 2250 mg/day in patients > 45 kg body weight), compared to placebo in patients with FRDA. Seventy (70) ambulatory FRDA patients between the ages of 8 and 18 years were recruited into this study at two clinical centres in the USA. Neither the primary nor secondary neurological endpoints reached statistical significance for either active dose compared to placebo. While on average both active treatment arms improved over baseline and over placebo in neurological function scales applied in this study, a lower than expected effect size on active treatment and an unexpectedly large improvement in the placebo arm combined to prevent statistical significance from being reached.

The safety results of the IONIA study were consistent with previous experience from the NICOSIA study and from the literature, and suggest that idebenone is safe and well tolerated at doses of up to 2250 mg/day.

The IONIA study was one of the commitments made to Health Canada as part of the conditional authorization of CATENA^® under the NOC/c Policy, and the results of the IONIA study have been communicated to Health Canada.

Health Canada has agreed that CATENA^® should continue to be available under the NOC/c Policy until the results of a third study of idebenone in FRDA (the MICONOS study) are available. It is anticipated that the results of MICONOS will be available in mid-2010.

The MICONOS study, a Phase 3 double-blind, randomized, placebo-controlled study of the efficacy, safety and tolerability of idebenone in the treatment of FRDA patients, is currently ongoing in Europe. A total of 232 patients have been enrolled and the efficacy of three idebenone doses is being compared to placebo during a 12 month treatment period. The MICONOS study is expected to address the shortcomings of the IONIA study: the sample size is larger (232 in MICONOS versus 70 in IONIA); the study duration is longer (12 months for MICONOS versus 6 months for IONIA); and the study is powered to detect a smaller, but still clinically meaningful treatment effect.

Santhera has committed to provide the results of the MICONOS study to Health Canada as soon as they become available, in anticipation that they will provide confirmatory evidence of the efficacy, safety and tolerability of idebenone in FRDA patients.

It should be noted that there have been no changes to the CATENA^® Product Monograph based on the information provided above. A copy of the package insert is enclosed with this letter, and a copy of the full Product Monograph is available on request from the Canadian Importer, GMD Distributing Inc. (1-866-270-1733), or on the Health Canada Web site at: www.hc-sc.gc.ca. More information is available on the CATENA^® Web site at www.catenainfo.ca. For any medical/ scientific inquiries, you may call Santhera Medical Information at 1-877-687-8177.

Original Signed by:

MJ Roach
General Manager, North America

William T. Andrews, MD,
Vice President, Medical Affairs