Jazz and Lilly cancer drugs cleared for use by NHS

Two therapies for bile duct cancer and leukaemia – Jazz Pharma's Ziihera and Eli Lilly's Jaypirca, respectively – have been recommended by reimbursement agency NICE for NHS use.

HER2-directed bispecific antibody Ziihera (zanidatamab) is backed in final guidance for the treatment of adults with HER2-positive advanced biliary tract cancer (BTC) who have received at least one prior line of systemic therapy, making it an option for around 65 patients a year in England.

It is the first anti-HER2 drug to be recommended for use by the NHS in this type of cancer, and the cost of treatment will be covered by the Cancer Drugs Fund (CDF) while additional data on its efficacy and safety is collected. BTC – an umbrella term that includes gallbladder cancer (GBC) and cholangiocarcinoma (CCA) – is an aggressive disease that is often detected late, with fewer than one in three people in England surviving for a year after diagnosis.

NICE's decision stems from the HERIZON-BTC-01 trial, which showed that people treated with Ziihera lived for an average of 18 months, which was significantly longer than the six months observed in a group treated with a standard chemotherapy regimen known as FOLFOX.

In addition, unlike FOLFOX, Ziihera does not have to be given through a surgically implanted device that can cause side effects, including pain, exhaustion, and nerve damage, said NICE.

Jazz has previously suggested that peak sales of Ziihera could reach $2 billion, if it can also get approval for the bispecific in more common indications like HER2-positive breast and gastric cancers. That would be a good return on the $325 million upfront payment and $1.76 billion in potential milestones laid out in its licensing deal for the drug with Zymeworks at the end of 2022.

New at-home treatment option for CLL

Meanwhile, around 200 people in England with chronic lymphocytic leukaemia (CLL) whose disease has returned after treatment could soon be treated at home with Lilly's oral reversible BTK inhibitor Jaypirca (pirtobrutinib), reducing the need for frequent hospital visits.

NICE's draft recommendation is that Jaypirca should be an option for people who have previously been treated with an irreversible, covalent BTK inhibitor – such as AbbVie/Johnson & Johnson's Imbruvica (ibrutinib) or AstraZeneca's Calquence (acalabrutinib) – and for whom further BTK inhibitor treatment is not suitable. The new recommendation comes on the back of data from the BRUIN CLL-321 study.

At the moment, these patients are usually treated with AbbVie/Roche's BCL-2 inhibitor Venclexta (venetoclax), which is also dosed orally, sometimes with the intravenously-administered anti-CD20 antibody rituximab. Venclexta is also dosed orally, but the need for frequent monitoring in the early, titration stages of treatment requires frequent visits to a clinic or hospital for blood tests to check for a side effect called tumour lysis syndrome.

Jaypirca has been approved in the UK as a second-line treatment for CLL since last September, and the new indication has been driving sales uptake, with Lilly reporting 2025 sales rose around 50% to $506 million. The company also recently reported positive results for the drug as a frontline therapy, which analysts view as being the key to driving sales into $1 billion-plus blockbuster territory.