Advanz Pharma Succeeds in Obtaining Temporary Reprieve of Revocation of Conditional Marketing Authorisation for Ocaliva®
- 17/09/2024
- News
On 4 September 2024, the President of the General Court of the European Union (the General Court) temporarily suspended the Implementing Decision of 30 August 2024 (the ID) of the European Commission (the Commission) that had revoked the conditional marketing authorisation (the CMA) for Ocaliva®, a prescription medicine used to treat adults with primary biliary cholangitis (PBC), an autoimmune condition that is responsible for the gradual destruction of the bile ducts in the liver and may result in liver failure while increasing the risk of liver cancer.
The President’s order causes the CMA to remain valid while interim proceedings and an action on the merits seeking the annulment of the ID run their course (Case T-455/24, Advanz Pharma v. Commission).
On 27 June 2024, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had recommended that the CMA should be revoked because the benefits of Ocaliva® were no longer considered to outweigh its risks. The CHMP based its recommendation on the available evidence, including a randomised comparative clinical trial designed to confirm the clinical benefits and safety of Ocaliva® in patients who do not qualify for an alternative treatment, real-world data, data from supportive studies, and information submitted by healthcare professionals and patient associations.
Details regarding the case in the public domain emerged only slowly and are still scarce. Advanz Pharma, the marketing authorisation holder for Ocaliva®, issued two press releases discussing the ID (here) and the order of the President of the General Court temporarily suspending the ID (here). By contrast, official sources are almost non-existent, despite what must be the strong interest of patients and healthcare professionals in the status of Ocaliva®. The website of the General Court only contains a reference to the court proceedings, but does not feature the President’s order. Additionally, it took the EMA more than a week before it published a terse statement indicating that the CMA of Ocaliva® would remain valid for the time being.
Given the dearth of information, it is at present impossible to judge the ID and the reasons of the President of the General Court to second-guess, albeit temporarily, the scientific judgment of the CHMP, as embraced by the Commission. The President generally has the power to order the suspension of a Commission decision if the “circumstances so require” (Article 278, Treaty on the Functioning of the European Union). This is an exception to the rule that a Commission decision takes immediate effect. It was for Advanz Pharma to (i) show urgency in that the ID risked causing serious and irreparable harm; (ii) put forward grounds for annulment of the ID that would seem meritorious at first sight; and (iii) demonstrate that the balance of interests leans in favour of the CMA continuing to apply.
In the meantime, the future of Ocaliva® is also in doubt in the United States. In a meeting held on 13 September 2024, the Gastrointestinal Drugs Advisory Committee of the Food and Drug Administration (FDA) not only voted against a full approval of Ocaliva® because the US marketing authorisation holder, Intercept Pharmaceuticals, failed to prove the medicine’s clinical benefit (Ocaliva® is on the US market following a 2016 accelerated approval), it also found that the medication’s overall risk-benefit profile is not favourable. The FDA is expected to make its final decision by 15 October 2024.
