BLINCYTO (blinatumomab)

Key points

Favourable opinion for reimbursement in monotherapy for the treatment of paediatric patients aged 1 year or older with Philadelphia chromosome negative CD19 positive B-cell precursor ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic hematopoietic stem cell transplantation.

What therapeutic improvement?

Slight therapeutic improvement compared to historical treatment.

Role in the care pathway?

In a situation of a Philadelphia chromosome negative ALL which is refractory or in second relapse, treatments are poorly standardised; allogeneic hematopoietic stem cell transplantation (HSCT) is recommended if not received after the first relapse and if complete remission can be obtained. Palliative supportive care can be proposed. A second transplant is sometimes considered.

The anti-CD 19 CAR-T tisagenlecleucel (KYMRIAH) has recently been granted an MA in the treatment of paediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse, and it is recommended by the Committee as a first-line treatment in paediatric and young adult patients up to 25 years of age with B-cell ALL that is refractory, in relapse post-transplant or in second or later relapse, while at the same time highlighting the uncertainties that remain concerning the efficacy and safety of KYMRIAH and the complexity of the treatment process.

Role of the medicinal product in the care pathway

BLINCYTO (blinatumomab) represents an alternative to chemotherapy-based salvage therapies in the treatment of paediatric patients aged 1 year or older with Philadelphia chromosome negative CD19 positive B-cell precursor ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic HSCT.

The Committee highlights the fact that BLINCYTO can be administered at patients’ homes in a home hospitalisation context, unlike chemotherapy-based salvage therapies, which require hospitalisation, partly due to the risk of infectious complications related to the haematotoxicity of chemotherapies.

Given the lack of comparative data to KYMRIAH (tisagenlecleucel), the role of BLINCYTO compared to KYMRIAH is not known. However, in patients with a general health status and life expectancy compatible with the administration of CAR-T treatment, the Committee considers that KYMRIAH should be favoured. The Committee reiterates that the efficacy and safety of KYMRIAH in patients previously treated with BLINCYTO has not been established (see Committee opinion for KYMRIAH dated 12 December 2018). In addition, KYMRIAH is not recommended if the patient has presented a relapse with CD19-negative leukaemia following previous treatment with an anti-CD19 therapy.

Finally, as in adult patients, on the basis of currently available data, the role of BLINCYTO compared to allogeneic HSCT, either as a “bridge” to allogeneic transplantation or to delayed allogeneic transplantation, or as a substitute for allogeneic transplantation, particularly in non-eligible patients, is not known.