Protestors want the European Commission to ignore official advice and keep marketing authorisation for drugs used to treat a rare liver disease.
ADVERTISEMENTThe fear that a key drug could be taken off the market led a group of patients to take to the streets and protest in front of the EU Council in Brussels this week.
Obeticholic acid, marketed as Ocaliva, is used to treat adult patients with a rare autoimmune disease, primary biliary cholangitis (PBC).
Ocaliva was designated in 2010 as an “orphan medicine”, a term used for drugs that treat rare diseases, but a recent review by a committee at the European Medicines Agency (EMA) recommended revoking its marketing authorisation.
The opinion from the Committee for Medicinal Products for Human Use (CHMP) will now be sent to the European Commission, which should then issue a final decision that’s legally binding in all EU member states.
“We are calling on the European Commission to ask the EMA to reconsider this recommendation,” Mo Christie from the PBC Foundation told Euronews.
A Commission spokesperson told Euronews that a decision on this file would be provided within the deadline set out in the relevant legislation but could not comment further at this stage.
The legal deadline is 67 days from the CHMP opinion, provided that the Member States in the Standing Committee support the Commission draft decision. In general, the Commission always follow the scientific assessments performed by EMA due to its scientific expertise to conduct the assessment.
PBC gradually destroys bile ducts in the liver, potentially leading to liver failure and an increased risk of liver cancer, and Ocaliva is used as an alternative when initial treatments fail, stop working, or have intolerable side effects, she explained.
“There’s no other second-line treatment for PBC licensed in Europe,” she added.
Carine Verstraeten, a member of Belgian patient organisation Mijn Lever, said she had used another medication for over 25 years. “But then, all of a sudden, that drug got much weaker,” she said, adding that Ocaliva brought results back to normal.
Conditional marketing authorisation was first granted in 2016, but the EMA requires clinical benefits to be confirmed through further studies.
In its latest review, the CHMP concluded Ocaliva was no more effective than a placebo in preventing disease progression or death, in early-stage PBC patients or others.
But for Gillian Kaye, a protestor diagnosed with PBC about 30 years ago, it’s vital to her wellbeing.
“Being a person who is quite active, with grandchildren, I'm very concerned that I'll no longer be able to get the drug if the EU doesn't give it a licence,” she said.
Updated to add a comment from a European Commission spokesperson.
