Subject matter of the dispute was the decision of the European Medicines Agency (“EMA”) not to validate an application by Shire Pharmaceuticals Ireland (“Shire”) for designation of a medicinal product as orphan medicinal product containing the same active substance (idursulfase) as an already authorised orphan product by Shire and whose orphan status was applied for in the same indication.
Shire claimed that the product in question was a different medicinal product compared to the one already authorised since it would differ in composition, route of administration and therapeutic effect. If this medicinal product were then also authorised as an orphan product, on the basis of this position the new medicinal product would be protected by an independent market exclusivity right under Article 8(1) of Regulation 141/2000 (“Orphan Regulation”), which prohibits the responsible authorities from accepting marketing authorisation (“MA”) applications or granting MAs for similar medicinal products in the same indication for a period of 10 years from the date on which the MA was granted (so-called “Orphan Market Exclusivity Right”).
The EMA has always been of the opinion that a company can only obtain an independent orphan status for the same active substance for different diseases, but that this would not be possible for the same disease. The consequence of this position is that the 10-year Orphan Market Exclusivity Right for both products begins with the first approval of the active substance so that the subsequent approval of the second drug would not enjoy its own 10-year Orphan Market Exclusivity right due to the lack of an independent orphan status.
In this case, the European Court of Justice has agreed with the European Court of First Instance that new medicinal products, even if they contain the same active substance as existing orphan medicinal products and the orphan status is applied for the same therapeutic indication, should be able to be independently designated as orphan medicinal products and thus be subject to an independent 10-year orphan market exclusivity right if at the time of MA grant they fulfil the criteria for designation as orphan medicinal products pursuant to Article 3(1) of Regulation 141/2000, i.e. if they have a significant benefit compared to the already authorised medicinal product.
Article 5 (1) of the Orphan Regulation requires the EMA to examine whether an application for designation as an orphan medicinal product has already been the subject of an earlier MA application. Article 5(2) of the Orphan Regulation contains a list of documents to be submitted with the application for designation as an orphan medicinal product. If these documents referred to in Article 5 (2) were submitted, the CJEU requires the EMA to validate the application for designation as an orphan medicinal product if this medicinal product is not identical to the medicinal product already designated as an orphan medicinal product. It is subsequently the responsibility of the Committee for Orphan Medicinal Products (COMP) to assess whether the new (second) product actually meets the criteria for designation as an orphan medicinal product set out in Article 3(1) of the Orphan Regulation, in particular with respect to the significant benefit.
With regard to the criteria by which the (non-)identity of the products is assessed, the CJEU did not set any general criteria, but simply stated in recital 40 of its judgment that on the basis of the alleged differences in composition, route of administration and therapeutic effects, the second product is not the same as the first. It can therefore be assumed that this will remain a matter of facts on a case by case basis.
Overall, this ruling of the CJEU is of great importance for the originator industry, since it confirms the possibility of obtaining an independent orphan status with a new orphan market exclusivity right even if the new product represents a further development of an already approved product with the same active substance for the same disease, provided that this has a significant benefit.
