The European Commission revokes the approval of the drug against CBP
Revocation of the authorisation of obetycholic acid for the treatment of primary biliary cholangitis (CBP)
The recent decision by the European Commission to withdraw marketing authorisation for obeticlic acid for the treatment of primary bile cholangitis (PBC) is a blow to thousands of patients with this rare liver disease. The news has raised major concerns among the scientific community, patient organisations and the pharmaceutical industry.
Primary biliary cholangitis: a chronic and progressive disease
CBP is a chronic autoimmune disease that affects the bile ducts of the liver, causing inflammation and damage to the liver tissue. There is no definitive cure for this disease, and the available therapies focus mainly on managing symptoms and slowing the progression of the disease.
Obetiklic acid: the ok revoked
Obetiklic acid, marketed as Ocaliva, was considered one of the most promising treatment options for PBC patients. This drug, acting as an X-pharnesoid receptor agonist, helps to reduce inflammation and protect liver cells. The European Commission’s decision to withdraw the marketing authorisation for the drug is based on a recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The CHMP considered that the clinical benefits of the drug were not sufficiently demonstrated, particularly in light of the results of the COBALT clinical study, which did not reach the primary endpoint.
The Advanz Pharma Replica
Advanz Pharma, the company producing the drug, has challenged this decision, pointing out the limitations of the COBALT study and the positive results from other clinical trials and real data. The company also highlighted the importance of obetycholic acid for patients with PBC, who had few effective therapeutic alternatives.
Implications for patients
The withdrawal of the marketing authorisation for obetycholic acid has profound implications for patients with CBP. Many of them have benefited from this drug, which has improved their quality of life and slowed the progression of the disease. Discontinuing obetycholic acid treatment may lead to worsening of symptoms, progression of liver fibrosis and the need for liver transplantation. Patients may also face difficulties in finding effective and safe therapeutic alternatives.
The next steps
Advanz Pharma has announced its intention to appeal the European Commission’s decision. Meanwhile, patient organisations and the scientific community are mobilizing to demand a reassessment of the situation and to find solutions that protect the rights of patients with PBC.