Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Swiss specialty pharmaceutical company Santhera has reached an agreement with the FDA under the special protocol assessment procedure relating to the Phase III clinical trial to evaluate idebenone for the treatment of Friedreich's ataxia.
Subscribe to our email newsletter
The trial with SNT-MC17, named IONIA (idebenone effects on neurological ICARS assessments), is a double-blind, randomized, placebo-controlled study of six months duration investigating the efficacy of two doses of SNT-MC17 compared to placebo. The primary endpoint of IONIA will be a neurological endpoint, measured by the international cooperative ataxia rating scale (ICARS), comparing the change in the ICARS for each of the treatment groups with placebo over the 24 week study period. The study will also investigate additional neurological endpoints as well as activities of daily living parameters and cardiac measures.
The IONIA study will recruit a minimum of 51 ambulatory Friedreich's ataxia (FRDA) patients between the ages of 8 and 17 years and will be conducted at two clinical centers in the US – the Children's Hospital of Philadelphia and the School of Medicine of the University of California, Los Angeles. Patient recruitment is expected to start soon.
Klaus Schollmeier, CEO, said: “With the offering of an extension study and FDA's fast-track designation granted, we now believe that we can successfully complete the development of SNT-MC17 for FRDA in the US during the course of 2009. SNT-MC17 has a good chance to become the first pharmaceutical product approved in the US for this devastating disease.”