EU Court Says Orphan Designation Possible For Same But Better Drugs
Executive Summary
The European General Court says that companies are entitled to an independent and new period of orphan market exclusivity for a product that is highly similar to a previously authorized orphan drug.
The European General Court has clarified that a new medicinal product can be granted orphan designation even if it has the same active substance as an existing orphan product being marketed by the same company.
The designation can be allowed if the new medicinal product offers a significant benefit over the previously authorized orphan product.
The court’s ruling, handed down last month in Shire Pharmaceuticals Ireland v European Medicines Agency, is an important ruling that could have significant implications for companies developing orphan medicinal products, according to law firm Covington & Burling, which represented Shire in the case.
The judgement confirms that drug companies are entitled to an independent and fresh 10-year period of orphan market exclusivity for a product that is highly similar to a previously authorized orphan medicinal product, added the Brussels-based law firm Van Bael & Bellis.
The General Court confirmed that "it is in the interest of patients suffering from a rare disease to have access to a similar medicinal product giving them a significant benefit compared to a previously authorised orphan product" – Covington & Burling
According to EU intellectual property firm Carpmaels & Ransford, the ruling is good news both for patients suffering from rare diseases and innovator companies that would like to improve on existing orphan products. It builds on recent EU court decisions, which have affirmed that the EU Orphan Regulation should be interpreted in a way "so as to achieve its aim of incentivising research into rare diseases," the IP firm added.
The Case
The court ruling was issued on March 22. It concerns case T-80/16, which Shire filed to challenge the EMA's refusal in 2015 to validate the company’s orphan designation application for Idursulfase-IT, intended for the treatment of Hunter Syndrome. The EMA's refusal was based on the fact that Shire had already obtained marketing authorization for its orphan drug, Elaprase, which contained the same active substance (idursulfase), also for the treatment of Hunter Syndrome. The European Commission also intervened in the case in support of the EMA's positon.
Shire claimed that Idursulfase-IT differed from Elaprase in its composition (smaller number of excipients), method of administration (intrathecal versus intravenous) and therapeutic effects (effective in respect of cognitive disorders versus effective in combating somatic disorders only). It claimed that Idursulfase-IT would offer significant benefit to patients over existing treatments, including Elaprase.
Shire also argued that the Orphan Regulation distinguishes between the terms "medicinal product" and "active substance," and claimed that the EMA and the commission "had confused the two concepts," said law firm Covington.
Covington said the court ruling had helped provide a number of "helpful clarifications" on the criteria for designating a medicinal product as an orphan, including that the EMA's verification of the validity of an application for designation of a medicinal product as an orphan medicinal product under Article 5(4) of the Orphan Regulation "is purely administrative in nature."
Importantly, the law firm added, the General Court confirmed that "it is in the interest of patients suffering from a rare disease to have access to a similar medicinal product giving them a significant benefit compared to a previously authorised orphan product."
The judgment suggests that if Shire were to re-submit its application for orphan designation of Idursulfase-IT, then the EMA would be obliged to validate the application, Covington said. The EMA's orphan medicines committee, the COMP, would then have to assess whether Idursulfase-IT offers significant benefit to patients over existing treatments, taking into account the relevant scientific evidence.
From the editors of Scrip Regulatory Affairs.