On 22 March 2018 the EU General Court handed down its judgment in Case T-80/16 Shire Pharmaceuticals Ireland Ltd v European Medicines Agency (EMA). The case centred around whether a new medicinal product can obtain orphan designation when it has the same active substance as an existing orphan product held by the same marketing authorisation holder (“MAH”).

The proceedings were brought by Shire after the EMA refused to validate Shire’s application for orphan designation for its Idursulfase-IT product in 2015. The basis for the EMA’s decision was that the active substance idursulfase had already been granted orphan designation in 2001, and was authorised in 2007 as orphan medicinal product Elaprase. Both products were intended for the treatment of Hunter Syndrome. Accordingly, the EMA concluded that Idursulfase-IT was covered by the 2001 designation and Shire was not entitled to a new period of market exclusivity.

Shire brought proceedings in 2016 challenging the EMA’s decision.

In its judgment, the Court found in favour of Shire and annulled the EMA’s 2015 decision refusing to validate Shire’s application for orphan designation for Idursulfase-IT.

The Court considered that the EMA was not entitled to refuse to validate Shire’s 2015 application on the ground that Shire had already obtained a MA for its orphan product Elaprase. The Court highlighted that the fact that the two products contained the same active substance did not necessarily mean that they are the same medicinal product. It was apparent from the data submitted with the application that there were differences between Elaprase and Idursulfase-IT (including composition, administration and therapeutic effects), and it was not appropriate to question whether these differences were “insignificant” at the stage of application for designation as an orphan product, which is a purely administrative stage.

The Court held that there is nothing in Regulation (EC) No. 141/2000 (the “Orphan Regulation”) which prevents a company applying for orphan designation for a product containing the same active substance as another product authorised in its name for the same indication, provided that the second product can satisfy the “significant benefit” test (Article 3(1)(b)). To consider “significant benefit”, one inevitably has to compare against products which have already been authorised, and there is nothing in the legislation or guidance to suggest that significant benefit cannot be achieved where the two products are owned by the same entity. This is entirely in line with previous EU General Court case law, e.g. Teva Pharma and Teva Pharmaceuticals Europe v EMA.

The Court dismissed the EMA’s arguments that allowing Shire’s claim would lead to “duplication” of market exclusivity and a “misuse” of the Orphan Regulation. The Court considered that it was in the interests of patients suffering from a rare disease to have access to an alternative medicine giving them a significant benefit compared to a previously authorised product.

Overall, the Court held that where a product meets the criteria for orphan designation set out in Art.3(1) of the Regulation, it must itself also be considered an orphan product, including where that product contains the same active substance as another orphan product. The fact that the first product has been awarded a period of market exclusivity (under Article 8(1)) does not preclude a second, similar product also being granted its own market exclusivity, as long as the Article 3(1) criteria are fulfilled. It was “irrelevant” that the MAH for the original orphan product and the sponsor of the second product were the same pharmaceutical company.

This is an important ruling for innovator companies, confirming that investing in the continued development of orphan products to demonstrate further “significant benefit” can lead to additional orphan designations.

The full judgment can be accessed here.