NICE says yes to BeiGene's Brukinsa after Scottish nay


BeiGene's BTK inhibitor Brukinsa (zanubrutinib) has become the first drug in the class to be recommended for routine NHS use in England and Wales to treat Waldenström macroglobulinaemia (WM) – a rare form of non-Hodgkin lymphoma (NHL).

The final appraisal document from health technology assessment (HTA) agency NICE says that Brukinsa can be used to treat WM in adults who have had at least one prior treatment, and only if  bendamustine plus rituximab therapy is also suitable.

It creates a disparity in access within the UK, however, as the drug was rejected by the Scottish Medicines Consortium (SMC) earlier this month. BeiGene has said it intends to resubmit the drug for use by NHS Scotland.

The recommendations come 10 months after Brukinsa was approved by the Medicines and Healthcare products Regulatory Agency (MHRA).

That approval also included first-line use of the drug for eligible patients unsuitable for chemo-immunotherapy, typically carried out with bendamustine plus rituximab, or dexamethasone plus rituximab and cyclophosphamide.

However, NICE's appraisal committee said, "for people who have not had previous treatment and if chemoimmunotherapy is unsuitable, the cost-effective estimates for zanubrutinib are above what NICE usually considers an acceptable use of NHS resources."

The document notes, however, that there is a high unmet need for an effective and well tolerated treatment for WM "where current chemoimmunotherapy options can cause severe adverse reactions and the need for frequent hospital visits."

It also notes that "clinical evidence from an indirect comparison suggests that people with Waldenström macroglobulinaemia may live longer and have a better quality of life with zanubrutinib than with standard care."

NICE's decision restores access to a BTK therapy for WM, after NICE concluded that Johnson & Johnson and AbbVie's market-leading BTK drug Imbruvica (ibrutinib) – previously available for WM patients in the UK via the Cancer Drugs Fund – was ineffective for this type of blood cancer.

WM is a rare type of B-cell lymphoma that affects fewer than 2% of patients with NHL, and there are around 4,000 people living with WM in the UK.

Second indication coming

Meanwhile, BeiGene is closing in on a new indication for Brukinsa in Europe, after the EMA's human medicines committee recommended approval of the drug for the treatment of relapsed or refractory marginal zone lymphoma (MZL), another B-cell NHL, around a year after it was cleared for the new use in the US.

If approved, the drug will be the first and only approved BTK inhibitor for MZL in Europe, said BeiGene. The European Commission will make a final decision on Brukinsa as a treatment for adult patients with MZL who have received at least one prior anti-CD20-based therapy within the next few weeks.