General Court Judgment on Orphan Designation
- 18/04/2018
- Articles
On 22 March 2018, the General Court (GC) has ruled that a medicine can obtain the designation of orphan medicinal product (OMP) even if it (i) contains the same active substance; and (ii) is intended to treat the same condition as an authorised OMP of the same sponsor, provided that the product represents a “significant benefit” compared to the previously authorised product (e.g., a more efficient formulation and means of administration). This finding is good news for pharmaceutical companies in that it confirms their entitlement to an independent and fresh 10-year period of orphan market exclusivity for a product that is highly similar to a previously authorised OMP.
The GC delivered its judgment in a dispute between Shire and the European Medicines Agency (EMA). The EMA had refused to validate Shire’s application for the designation of Idursulfase-IT (active substance: idursulfase) as an OMP for the treatment of Hunter Syndrome because Shire had previously obtained a marketing authorisation for an OMP for the treatment of the same disease that contains the same active substance (trade name: Elaprase®). Pursuant to Article 5(1) of Regulation (EC) No 141/2000 of 16 December 1999 on orphan medicinal products (Regulation 141/2000), an application for designation as an OMP should be submitted “at any stage of the development of the medicinal product before the application for marketing authorisation is made” (emphasis added).
Although containing the same active substance, Idursulfase-IT differs from Elaprase® in its composition (smaller number of excipients), method of administration (intrathecal vs intravenous) and therapeutic effects (effective in respect of cognitive disorders vs effective in combating somatic disorders only).
The GC stressed that, although the active substance is one of the components or the main constituent of a medicinal product, it must not be confused with the medicinal product itself. Hence, the EMA could not refuse to validate Shire’s application for designation of Idursulfase-IT as an OMP on the ground that it had already obtained a marketing authorisation for Elaprase®.
However, the GC also indicated that, in view of the already existing treatment for Hunter Syndrome (Elaprase®), it should be assessed whether Idursulfase-IT represents a “significant benefit” within the meaning of Article 3(1)(b) of Regulation 141/2000 for patients in comparison with the existing authorised treatment. Such a “significant benefit” may take the form of, for instance, a more efficient formulation and means of administration. The GC left it to EMA’s Committee on Orphan Medicinal Products to assess whether Idursulfase-IT meets this requirement.
GC, 22 March 2018, Case T-80/16, Shire Pharmaceuticals Ireland Ltd v. European Medicines Agency (EMA)
