EU court overrules commission in Orphacol drug case
The General Court of the European Union has annulled the European Commission’s decision to refuse marketing authorisation for orphan drug Orphacol.
The ruling means that the commission must ensure that a decision granting marketing authorisation for Orphacol is issued immediately.
Orphan drugs are used in the treatment of diseases that affect small populations. As such, the cost of developing orphan drugs is unlikely to be recovered by the expected sales, and they are frequently not patent protected.
However, drugs awarded orphan status enjoy 10 years of market exclusivity from the date of authorisation.
Orphacol has been used to treat patients with a rare liver condition since 1993. It is produced by French pharmaceutical company Laboratoires CTRS but its active ingredient, cholic acid, has not been authorised for use in the EU.
The product had been supplied first as a hospital preparation in France, and then under a “patient-needs” scheme for particular named patients.
Laboratoires CTRS submitted its application for marketing authorisation under the “well-established use” procedure, where the results of pre-clinical tests and clinical trials are replaced with references to scientific literature.
The Committee for Medicinal Products for Human Use concluded that the drug had fulfilled the criteria for “well-established medicinal use”, as its safety and efficacy had been adequately demonstrated by its use in patients since the early 1990s.
However, the European Commission refused to grant marketing authorisation for Orphacol in 2011.
It claimed that as Laboratoires CTRS could not demonstrate that the drug had been used to treat the disease in the EU safely and consistently for at least 10 years, it did not meet the legal requirements for obtaining marketing authorisation.
Specifically, it considered that the French hospital preparation use was not sufficient for the purposes of demonstrating well-established medicinal use.
Despite strong opposition from EU member states, the commission persisted in refusing to grant marketing authorisation.
In January 2012, Laboratoires CTRS filed an action challenging the commission’s position, claiming it had no legal basis to refuse marketing authorisation.
The day before the hearing, the commission replaced its original proposal with a re-worded version which again denied marketing authorisation.
The court declined to adjudicate on the merits of the case, and in July 2012 Laboratoires CTRS brought a second action to the General Court, challenging the commission’s refusal to grant a marketing authorisation for Orphacol.
On July 4, the court determined the commission had not acted lawfully in refusing the marketing authorisation.
“The commission interpreted the legislation in a very narrow way,” said Marina Vickers, associate at Bristows LLP, the firm that represented Laboratoires CTRS in this case.
Marie Manley, partner at Bristows LLP, and leader of the legal team that represented the plaintiff, told LSIPR that the judgment has clarified how to interpret the well-established use provision, and its interaction with the ‘exceptional circumstances’ provision.
She added that the commission’s refusal to grant marketing authorisation may have left Laboratoires CTRS vulnerable to competitors looking to launch their own versions of the drug during the case.
