ANAGRELIDE AOP (anagrélide)

• Key points

Unfavourable opinion for reimbursement for the reduction of elevated platelet counts and the associated clinical symptoms in high-risk essential thrombocythaemia (ET) patients.

• Role in the care pathway?

An adequate response to the first-line treatment (hydroxyurea or, potentially, pegylated interferon alpha used off-label) is obtained in the majority of patients. XAGRID, a proprietary medicinal product containing anagrelide, may only be envisaged as second-line treatment in patients who are intolerant or are not responding to their current therapy, in accordance with its MA. Busulfan may also be used off-label, but its leukaemogenic potential restricts its role to last-line treatment or in very elderly subjects. The choice of cytoreductive agent is based on the profile of each patient and, in particular, the expected safety.

Role of the medicinal product in the care pathway

ANAGRELIDE AOP is a new medicinal product, containing anagrelide, a generic of the proprietary medicinal product THROMBOREDUCTIN, which has no MA in France. It is not a generic of XAGRID.

Considering:

• the efficacy data available for THROMBOREDUCTIN as first-line treatment, derived from a single-blind, non-inferiority study versus hydroxyurea, having included patients considered to be high-risk on the basis of diagnostic criteria and treatment regimens different from those now accepted by consensus, and for which, in addition, the demonstration of non-inferiority is debatable,
• the existence of several proprietary medicinal products containing anagrelide reimbursed in France in second-line therapy (XAGRID and its generics),
• the descriptive, non-comparative clinical data, which are insufficient to assess ANAGRELIDE AOP as a second-line treatment, in a context in which ANAGRELIDE AOP is not bioequivalent to XAGRID,

the Committee deems that ANAGRELIDE AOP has no role in the care pathway for the treatment of high-risk essential thrombocythaemia (ET) patients, as either first or second-line therapy.