By its judgement of 11 June 2015, the General Court annulled the Commission's decision granting a marketing authorization for Kolbam thus protecting the 10 year market exclusivity of Orphacol (Case T-452/14).
Orphacol was authorized in 2013 for the treatment of inborn errors of primary bile acid synthesis caused by 2 types of enzyme defects. Such authorization was granted only after the General Court's annulment of the Commission's 2012 decision to refuse the marketing authorization despite a positive opinion of the European Medicines Agency. Because of its orphan drug status Orphacol enjoys a 10-year market exclusivity. This incentive was instituted by article 8 of the orphan drug regulation (EC) No 141/2000 in order to promote the development of treatments for patients with rare diseases.
Like Orphacol, Kolbam's active ingredient is a cholic acid and is indicated to treat inborn errors of primary bile acid synthesis. An application for authorisation was submitted in 2012 for 5 indications but the drug was authorized in 2014 for only 3 indications as the other 2 indications related to enzyme defects already covered by Orphacol and no clinical superiority was established. While the drugs are formally authorized in different indications, Laboratoires CTRS, the marketing authorization holder of Orphacol sought the annulment of the marketing authorization for Kolbam because of references in the summary of product characteristics (other than in section 4.1 of the SmPC) and assessment report to the efficacy of Kolbam in the Orphacol approved indications. Such references, the Court agreed, encourage off-label use and thus circumvent Orphacol's 10-year market exclusivity.
The Commission's argumentation used in the court case raises doubt whether a new decision for marketing authorization for Kolbam can be issued on the basis of the same dossier removing the references to the efficacy of Kolbam in the Orphacol approved indications. Given the fact that the majority of the data concerned patients with the Orphacol therapeutic indications, the Commission maintained it was essential to draw conclusions in respect of those indications in order to reach a conclusion on the Kolbam indications.
The judgment of the General Court is still subject to appeal (within 2 months) but limited to points of law only.