Landmark judgment on orphan market exclusivity and off-label prescribing

22/06/15

In its judgment on 11th June in Case T-452/14 CTRS v Commission the General Court, for the first time ever, annulled a marketing authorisation on the grounds that statements in the Summary of Product Characteristics (SmPC) and assessment report for the relevant product were liable to facilitate the off-label prescribing of that product, and thus circumvent the orphan market exclusivity attaching to a competing product.

The judgment is the latest in a series of cases concerning CTRS’ orphan product Orphacol, which is authorised for the treatment of two very rare and serious genetic liver disorders. Following the General Court’s judgment in Case T-301/12 CTRS v Commission, annulling the Commission’s prior refusal to authorise this product, CTRS obtained its marketing authorisation in September 2013, and benefited for a 10-year period of orphan market exclusivity from that date, for the indications for which it was authorised. The Commission subsequently granted a marketing authorisation for another product, Kolbam, for different indications. CTRS’ objection was that the SmPC and assessment report attaching to that authorisation contained repeated references to the safety and efficacy of Kolbam in the indications for which Orphacol benefited from market exclusivity.

The Court agreed with CTRS that the SmPC and the assessment report were integral parts of the statement of reasons for a marketing authorisation decision. An action for annulment of the marketing authorisation could therefore be based on the content of those documents. The Court then went on to deliver a trenchant ruling on the effectiveness of orphan market exclusivity, finding that this important incentive for the development of orphan products would be circumvented if a market authorisation for a competing product was formulated in such a way as to facilitate off-label prescribing in the indications covered by the orphan exclusivity of the first product.

On that basis the Court found that although the disputed statements in the Kolbam SmPC and assessment report did not formally alter the scope of Kolbam’s marketing authorisation, those statements were liable to facilitate off-label prescribing of Kolbam for the Orphacol indications, and thus undermine Orphacol’s market exclusivity. It therefore annulled the Kolbam marketing authorisation decision.

For the Judgment click here

For the Judgment in the related case T-301/12 click here

For Bristows' report click here