The European Medicines Agency (EMA) has suggested conceding a provisional marketing approval in the European Union (EU) to Ocaliva (obeticholic acid) for the treatment of patients with major biliary cholangitis. Conditional approval is one of EMA’s main actions to make easy earlier access by patients to medicines that fulfill unmet medical needs. It permits the Agency to suggest a medicine for marketing authorization before the availability of confirmatory clinical trial data.
This is to be used in combination with another medicine, ursodeoxycholic acid (UDCA), in patients who have not responded sufficiently to UDCA, or on its own in adults who are not able to bear treatment with UDCA.
Primary biliary cholangitis is a uncommon and life-threatening disease that causes the steady devastation of the small bile ducts in the liver. These ducts transport fluid called bile from the liver towards the intestines where it is used to help digest fats. Due to the destruction of the ducts, bile builds up in the liver causing damage that may lead to liver cancer.
There are few treatments available for patients with primary biliary cholangitis such as:
- Liver transplantation can considerably get better a patient’s chance of survival; however this is a long and intricate operation only suitable for patients who have advanced liver disease.
- UDCA is the only medicine currently approved to treat primary biliary cholangitis, but up to half of all patients treated with UDCA either fail to respond to the medicine or experience partial benefits. There is therefore a clear unmet medical need for these patients, as well as for patients who are unable to tolerate treatment with UDCA.
The protection and effectiveness of Ocaliva were verified in a phase III study with 216 participants. After 12 months, the amount of patients achieving reductions in levels of their alkaline phosphates was higher in patients treated with Ocaliva.
The most frequent side effects observed with Ocaliva were itching of the skin and weakness.
As part of the conditional marketing authorization, the applicant for Ocaliva has to provide results from two studies. Until availability of full data, the CHMP will review the benefits and risks of Ocaliva yearly to determine whether the conditional marketing authorization can be maintained.
The opinion by the CHMP at its October 2016 meeting is an intermediary step on Ocaliva’s path to patient access. The CHMP opinion will now be sent to the European Commission for the acceptance of a decision on an EU-wide marketing authorization. Once a marketing authorization has been settled, a decision on price and reimbursement will then take place at the level of each Member State considering the possible role in the circumstance of the national health system of that country.
Date: 14/10/2016