HOW DOES A DRUG GET DEVELOPED?

DISCONTINUED: Stages of Development for Idebenone

The drug development process can be thought of as a series of stages, and drugs must successfully pass through each stage to become available to patients. This treatment has been evaluated, and the program has been discontinued. Thus, it is not in the pipeline.

Link to previous study

May 2009: IONIA study and 12-month open label extension study (IONIA-E). After the Phase III IONIA study 68 patients were enrolled into an open-label extension study where patients received 1350/2250 mg/day idebenone for 12 months (IONIA-E). Changes in ICARS and FARS were recorded during the total of 18 months combined study period.

Santhera announced that the phase III trial of Idebenone (Catena®) in the United States did not demonstrate benefit at the level of statistical significance.

The study did demonstrate that the drug was safe and well-tolerated and that the individuals who received Idebenone improved their scores more than those on placebo. However, the benefit did not reach statistical significance.

May 2010: Phase III – MICONOS (Mitochondrial Protection with Idebenone In Cardiac Or Neurological Outcome Study. Santhera announced that the phase III trial of Idebenone in Europe (MICONOS) did not reach primary or secondary endpoints which were neurological rating scales (ICARS and FARS). This was a 12-month placebo controlled trial in adults with FA.

2011: IONIA-E Results were reported:

  • May 2011 at the 4th International FA Scientific Conference in Strasbourg FR.
  • Published in Meier et al, J Neurol, 2011.
  • A third publication was also released in 2011 from the IONIA study looking at cardiac changes via echocardiogram during the 6-month study. Left ventricular mass index, posterior wall thickness, EF, and ECG parameters were not significantly improved by treatment with idebenone. Therefore, this study did not provide evidence of benefit in this cohort over a 6-month treatment period.

While data from several studies show modest improvements in FA patients, these improvements have not been reproducible or statistically significant.

July 2008: Catena® (idebenone) received conditional market approval in Canada for the treatment of Friedreich’s ataxia (FA).

April 2013: However, based on additional data that fails to confirm that treatment with Catena® is beneficial in FA, Santhera Pharmaceuticals announced it will discontinue sales of the drug on April 30, 2013. The company reported that no specific safety issues were identified in connection with removal of Catena® from the market.

The drug was not approved for use in the United States.

Idebenone is approved in Europe for the treatment of Leber’s hereditary optic neuropathy (LHON), a rare mitochondrial disorder.