European EMEA acceptance of Marketing Authorization Application filing for SNT-MC17 in Friedreich's Ataxia by Santhera
Santhera Pharmaceuticals and Takeda Pharmaceutical Company Limited (TSE:4502, "Takeda"), jointly announced that the European Medicines Agency (EMEA) has accepted the filing of the Marketing Authorization Application (MAA) for Santhera's lead compound SNT-MC17 (INN: idebenone, originally developed by Takeda) for the treatment of Friedreich's ataxia (FRDA). SNT-MC17, which has been granted Orphan Drug Designation in the EU, could become the first approved product for the treatment of FRDA and will be marketed in Europe by Santhera's partner Takeda. SNT-MC17 has shown clinical efficacy in FRDA patients on neurological as well as cardiac endpoints in several clinical studies and proved to be well tolerated in all studies so far.
In a recently completed clinical trial conducted in collaboration with the US National Institutes of Health (NIH), Santhera tested the efficacy of three doses of SNT-MC17 in patients with FRDA. Study results were announced in fall 2006. The MAA file includes data generated in this collaborative study with the NIH analyzing a variety of neurological and cardiac outcome measures, 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 MAA 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 MAA file includes safety data generated by Santhera with SNT-MC17 as well as safety data from Takeda generated in its earlier preclinical and clinical development program with idebenone for the treatment of Alzheimer's disease. Santhera believes that the compound has the potential to be granted European marketing approval for the treatment of FRDA in the second half of 2008.
A milestone payment of EUR 3 million to Santhera from its European marketing partner Takeda is triggered by the EMEA's acceptance of the SNT-MC17 MAA filing.
Santhera has decided, despite the MAA filing, to continue its ongoing Phase III clinical trial with SNT-MC17 in Europe to collect additional safety and efficacy data in a wider population of FRDA patients, particularly for doses up to 1350 mg/day and 2250 mg/day in the two body weight groups. Santhera amended the study protocol based on the findings of the NIH study to primarily evaluate the benefits of SNT-MC17 on the neurological aspects of FRDA. Santhera also offers all FRDA patients that participate and complete the EU Phase III trial the opportunity to enroll in an open label extension study here patients will receive high dose SNT-MC17.
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