Health Canada Accepts Santhera's Submission for Marketing Authorization of SNT-MC17 in Friedreich's Ataxia

 

The dossier submitted to the Therapeutic Products Directorate (TPD) of Health Canada includes efficacy data generated in the collaborative study with the US National Institutes of Health analyzing a variety of neurological and cardiac outcome measures. The NDS is supported by data from earlier clinical trials in FRDA conducted by academic institutions that demonstrated efficacy primarily in the treatment of the cardiac symptoms of this devastating disease. The safety package consists of data generated by Santhera with SNT-MC17 and Takeda in its earlier preclinical and clinical development program with idebenone.

 

The Canadian application for marketing authorization recommends a starting dose of 450 mg/day for patients below 45 kg body weight and 900 mg/day for patients of 45 kg or above body weight, with the option for the treating physician to use higher doses if needed. The dosing recommendations as well as the data package of the Canadian filing are equivalent to the previous EMEA and Swissmedic filings.

 

Health Canada's standard review process for a submission for Notice of Compliance with Conditions (NOC/c) calls for a 200 day review target. A positive review is followed by agreement on certain conditions, as anticipated to be required by the Canadian regulator for a NOC/c. Based on these timelines, Santhera expects the approval under NOC/c to be issued in Canada in the second half of 2008.

 

Klaus Schollmeier, Santhera's CEO commenting on the validation of the NDS said: "Canada is our third filing after the EU and Switzerland. If everything goes according to plan, the marketing authorization for this first approved pharmaceutical product for the treatment of FRDA can be expected in late 2008."

 

Friedreich's Ataxia (FRDA) is a rare but severe genetic neuromuscular disorder that results in the degeneration of an individual's nerve and muscle tissue. This disorder causes loss of muscle control, uncoordinated movements, muscle wasting and thickening of heart walls which frequently leads to a shortened life span. FRDA affects both Caucasian males and females equally and it is estimated that about 20,000 patients suffer from the disease in both North America and Europe. Average life expectancy for FRDA patients is limited to approximately 35 to 50 years.

 

The disorder results from a genetic defect in the gene encoding for frataxin. Reduced levels of this protein ultimately result in impaired energy production in mitochondria, the cells' energy production centers, and elevated oxidative stress. Tissues that have the highest need for energy, in particular nerve and cardiac tissues, are primarily affected by frataxin deficiency resulting in pathological changes in heart muscle anatomy and function and loss of nerve cells. SNT-MC17 is believed to improve the balance and flow of electrons within the mitochondria, therefore increasing the energy production within nerve and muscle cells of FRDA patients, protecting these cells from cell death. A number of clinical trials have provided strong evidence that SNT-MC17 may offer an effective treatment option for FRDA associated heart wall thickening (cardiomyopathy). In addition, data from the collaborative NIH clinical trial suggest positive effects on neurological function.

 

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Santhera Pharmaceuticals (SWX: SANN) is a Swiss specialty pharmaceutical company focused on the discovery, development and marketing of small-molecule pharmaceutical products for the treatment of severe neuromuscular diseases. Santhera's vision is to become a leading specialty pharmaceutical company offering therapies for a number of indications in this area of high unmet medical need which includes many orphan indications with no current therapy.

 

Santhera currently has five clinical-stage development programs, three of which are investigating its lead compound, SNT-MC17 (INN: idebenone), for the treatment of Friedreich's Ataxia (FRDA), Duchenne Muscular Dystrophy (DMD) and Leber's Hereditary Optic Neuropathy (LHON). Another clinical program is investigating JP-1730 (INN: fipamezole) for the treatment of Dyskinesia in Parkinson's Disease (DPD) in cooperation with Juvantia, the compound's owner. The fifth program comprises SNT-317 (INN: omigapil) in Congenital Muscular Dystrophies (CMD), a compound in-licensed from Novartis. For the most advanced program, SNT-MC17 in FRDA, the Company has applied for marketing authorization in Europe and Canada. The compound is also in Phase III clinical development for FRDA in the US while the other clinical programs are in Phase II. For further information, please visit www.santhera.com.

 

Klaus Schollmeier, Chief Executive Officer

Phone: +41 (0)61 906 89 52

 

Barbara Heller, Chief Financial Officer

Phone: +41 (0)61 906 89 54

 

Thomas Staffelbach, VP Public & Investor Relations

Phone: +41 (0)61 906 89 47

 

 

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