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November 19, 2008: CHMP Confirms Original Opinion on Santhera's SNT-MC17/Idebenone for Treatment of Friedreich's Ataxia
News release CHMP
Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty pharmaceutical company focused on neuromuscular diseases, announced today that the European Medicines Agency (EMEA) has informally advised that it would maintain its negative opinion on the Company's Marketing Authorization Application (MAA) for SNT-MC17/idebenone in Friedreich's Ataxia. According to the information received, the Committee for Medicinal Products for Human Use (CHMP) of the EMEA in its reexamination concluded that it cannot support an early approval at this point in time but rather prefers to wait until additional data from at least one of Santhera's two pivotal trials become available for review. The Company has two Phase III studies running and both these trials have achieved their recruitment target. Santhera intends to file for marketing authorization in the United States and in the European Union next year.
Klaus Schollmeier, Chief Executive Officer of Santhera, commented: "Our success in making additional data available in the near future has been the major obstacle throughout the regular review process as well as the reexamination. The confirmation of the original CHMP opinion is obviously a disappointment but not a surprise. As a result of today's decision, Friedreich's Ataxia patients in the European Union must continue to wait for the first controlled pharmaceutical product to treat their devastating disease. We confirm our commitment to make this important drug available to patients in Europe and in the United States as we are already able to do in Canada."
Meanwhile, Santhera's two Phase III trials are both recruited. In Europe, the twelve-month MICONOS (Mitochondrial Protection With Idebenone In Cardiac Or Neurological Outcome Study) trial has achieved the enrollment of 204 Friedreich's Ataxia patients and will be closed to recruitment shortly. In the United States, the last patient was randomized into the IONIA (Idebenone effects On Neurological ICARS Assessments) trial on October 31, 2008. A total of 70 Friedreich's Ataxia patients have been enrolled into this six-month study. Subject to positive outcome of the IONIA trial, Santhera expects to file a New Drug Application with the US Food and Drug Administration (FDA) before the end of 2009. In the United States, the program has been granted fast track status by the FDA in 2007. A new MAA is planned to be submitted to the EMEA within the same timeframe.
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Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the discovery, development and commercialization of small-molecule pharmaceutical products for the treatment of severe neuromuscular diseases, an area of high unmet medical need which includes many orphan indications with no current therapy. Santhera currently investigates three compounds in five clinical-stage development programs. The Company's first product, SNT-MC17 (INN: idebenone), has received a marketing approval with conditions from Health Canada to treat Friedreich's Ataxia and is marketed under its brand name CATENA®. The product is also under review in Switzerland, while two pivotal clinical trials in the United States and in Europe have recently achieved their enrollment targets. The compound has also shown efficacy in a phase II clinical trial as a potential treatment for Duchenne Muscular Dystrophy. For further information, please visit the Company's website www.santhera.com
CATENA® is a trademark of Santhera Pharmaceuticals, registered in Canada and the United States.
For further information, contact
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, Head Public & Investor Relations
Phone: +41 (0)61 906 89 47
This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.
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The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.
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Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:
1) Gene Therapy for Friedreich's Ataxia research project:
The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.
2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.
The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.