NICE to reassess Pfizer’s leukaemia drug Besponsa

The National Institute for Health and Care Excellence will reassess Pfizer’s Besponsa (inotuzumab ozogamicin) as a treatment for adults with a certain form of leukaemia.

The move follows a successful appeal by Pfizer against the Institute’s decision to reject Besponsa (inotuzumab ozogamicin) as a treatment for adults with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukaemia (ALL).

NICE’s appraisal committee will now take another look at the evidence for the drug before issuing a second Final Appraisal Determination (FAD) regarding funding for its use on the NHS.

Besponsa is an antibody drug conjugate comprised of a monoclonal antibody targeting CD22, a cell surface antigen expressed on around 90 percent of B-cell malignancies, linked to a cytotoxic agent.

When the drug binds to CD22 on malignant B-cells it is taken into the cell, where the cytotoxic agent calicheamicin is released to destroy it.

Data from the INO-VATE trial, which compared the drug to standard of care chemotherapy in 326 adult patients with relapsed or refractory CD22-positive ALL, and showed that the therapy more than doubled complete remission rates.

However, in draft guidelines released in June last year, the Institute argued that evidence from clinical trials showed no survival benefit from Besponsa compared to current treatment, but did note that more people who were treated with the drug were able to subsequently have a stem cell transplant and go into remission than those on standard care.

ALL is an aggressive type of leukaemia with high unmet need and a poor prognosis. The current standard treatment is intensive, long-term chemotherapy, but currently only 10 percent of adults with ALL who relapse after first-line therapy survive five years or more.

It is estimated that 117 patients would be eligible for treatment with the drug in its first year if it were recommended for routine use.

"NICE’s decision to uphold the appeal and re-assess the evidence for inotuzumab ozogamicin is good news for some of the most vulnerable leukaemia patients in the UK. Pfizer is committed to ongoing collaboration with NICE to ensure that eligible patients are granted access to inotuzumab ozogamicin," said Craig Eagle, Head of Oncology at Pfizer UK.