General Court annuls decision of the Commission to refuse marketing authorisation for orphan product

09/07/13

Laboratoires CTRS had developed Orphacol, an orphan product which treats rare but very serious liver disorders in newborns. CTRS had applied for a marketing authorisation ("MA") under Article 10a of Directive 2001/83 on the basis of well-established medicinal use ("WEU") of the active ingredient, cholic acid. That application was refused by the Commission on the grounds that the conditions for granting an MA under Article 10a were not met.

In a judgment handed down last Thursday, the General Court annulled the Commission's decision, finding in CTRS' favour on all three substantive grounds for annulment.

First, the Court found that the long-established use of cholic acid as a hospital preparation in France was sufficiently systematic and well-documented to prove WEU, rejecting the Commission's argument that hospital preparation use could not demonstrate WEU under Article 10a. Secondly, the Court considered that it was possible to invoke exceptional circumstances justifying lack of certain data in a WEU application under Article 10a, rejecting the Commission's argument that the concepts of WEU and exceptional circumstances could not be applied simultaneously. Thirdly, the Court found that the Commission was wrong to conclude, in the decision, that the grant of an MA for Orphacol would undermine the objectives of the Paediatric Regulation and the protection of innovation.

In light of those findings, the Court decided that it was not necessary to rule on a procedural complaint also raised by CTRS, concerning the Commission's breach of the rules of the Comitology procedure in the adoption of the contested decision.

The judgment is here.