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OCALIVA

Biliary cholangitis - Reevaluation
Opinions on drugs - Posted on May 29 2020

Reason for request

Reevaluation

Reevaluation.

Favourable opinion for reimbursement in the treatment of primary biliary cholangitis (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.

What therapeutic improvement?

No clinical added value in the management of the disease.

Role in the care pathway?

The first-line treatment of primary biliary cholangitis is based on the prescription of ursodeoxycholic acid (UDCA), irrespective of disease stage. The earlier the treatment is initiated in the disease, the more effective UDCA is. Bezafibrate is used off-label in patients not responding to or unable to tolerate UDCA.

At a very advanced stage of the disease, which has become rare under treatment with UDCA, a liver transplant is often necessary.

Role of the medicinal product in the care pathway

OCALIVA (obeticholic acid) is a second-line treatment that may be prescribed either in combination with UDCA in the event of an inadequate response to UDCA or as monotherapy in the event of intolerance to UDCA.

Special recommendation

The initial dose and adaptation of the dose in patients with primary biliary cholangitis depend on the patient’s liver function.


Clinical Benefit
Substantial

The clinical benefit of OCALIVA (obeticholic acid) is high in the MA indication.

Clinical Added Value
no clinical added value

Considering,

  • the results of the 3-year open-label extension phase of the phase III clinical study (POISE) suggesting that efficacy is maintained with respect to the biological endpoints,
  • the characteristics of the patients included in this study, who were predominantly at an early stage of the disease, and the absence of robust data on the efficacy of obeticholic acid for more advanced and more severe stages of the disease,
  • pending robust data on the regression of hepatic fibrosis (substitution endpoint), but especially clinical data (transplant-free survival, disease decompensation), particularly in the advanced stages of the disease,
  • the weakness of the available data concerning the use of OCALIVA (obeticholic acid) as monotherapy, a situation that is nonetheless rare,
  • the safety profile demonstrating:
    • pruritus (which is a symptom of the disease) as a common adverse event, leading to treatment discontinuations,
    • hepatic lesions identified as an important potential risk in the risk management plan,

the Transparency Committee considers that OCALIVA (obeticholic acid) provides no clinical added value (CAV V) in the care pathway for the treatment of primary biliary cholangitis in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.

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