Zolbetuximab for pancreatic cancer: Is approval expected soon?

In addition to its EMA approval and FDA approval as the first targeted therapy for some gastric cancers, zolbetuximab is also being studied as a potential treatment for pancreatic cancer. What are the latest clinical trial results in this area, and how close is a potential approval?

Here's what you need to know.

What types of pancreatic cancer is zolbetuximab relevant for?

As all targeted therapies, zolbetuximab is only relevant for a specific subset of pancreatic cancers. More specifically, those expressing the claudin 18.2 (CLDN 18.2) protein. CLDN 18.2 is most commonly expressed in pancreatic ductal adenocarcinoma (PDAC). According to some authors, up to 95% of PDAC tumors express this protein. This makes the targeted therapy with zolbetuximab a potentially promising treatment option for a large group of patients ¹.

How is zolbetuximab expected to work in pancreatic cancer?

Zolbetuximab works by targeting and binding to the CLDN 18.2 protein. This protein is located on the surface of tumor cells, making it an accessible target for monoclonal antibody therapy. When zolbetuximab binds to CLDN 18.2, it helps immune cells like natural killer (NK) cells to attack and kill the cancer cells.

Through these mechanisms, zolbetuximab aims to reduce tumors and potentially improve outcomes for patients with Claudin 18.2-positive pancreatic cancers.

Latest clinical trial results for zolbetuximab in pancreatic cancer

While there are no clinical trial results available yet, there are several trials ongoing. They're investigating the efficacy and safety of zolbetuximab as a potential pancreatic cancer treatment. Some of these are:

NCT03816163 (Phase 2 trial)

This study aims to find the right dose of zolbetuximab to use with chemotherapy for treating metastatic pancreatic cancer. It is currently ongoing globally. Participants are receiving either zolbetuximab and chemotherapy or chemotherapy alone.

The main goals are to determine the optimal dose, assess if combining zolbetuximab with chemotherapy can improve survival, and evaluate the safety of zolbetuximab in combination with chemotherapy and how patients manage side effects during the study.

The results of the NCT03816163 study are expected towards the end of 2026 ².

NCT06396091 (Phase 1 trial)

This study is currently recruiting participants. It will test zolbetuximab combined with chemotherapy in adults with metastatic pancreatic cancer who have not received chemotherapy before.

The study has two main goals: to assess the safety of zolbetuximab with chemotherapy and to see how well participants tolerate any side effects. It is an open-label study. This means that both the participants and doctors know who is receiving the treatment, with different groups receiving varying doses of zolbetuximab.

The study is expected to be completed in the second half of 2026 ³.

Is zolbetuximab approved for treating pancreatic cancer?

As of December 2024, no. The medicine is only approved for the treatment of HER2-negative, CLDN 18.2-expressing gastric and gastroesophageal cancers.

As clinical trials progress and more data are available about the medicine's efficacy and safety, it will be easier to comment on a potential regulatory approval application. For the time being, it seems that zolbetuximab is unlikely to be approved for pancreatic cancer until some of the ongoing clinical trials are complete. This is expected to be in 2026.

Can my doctor already prescribe zolbetuximab for pancreatic cancer?

The best answer is, maybe.

From a purely legal point of view, your doctor has the authority to prescribe a medicine for use other than indicated. This is known as "off-label use".

However, since there is insufficient clinical trial data available about the specific use of Vyloy (zolbetuximab) in pancreatic cancer, your doctor is unlikely to prescribe the medicine yet.

There may be a moment in the coming months or years when preliminary clinical trial results are shared. On the basis of such results, if positive, your doctor may decide to prescribe the medicine. Even though it will not be approved anywhere for this indication yet. If that happens, you'll be able to access the medicine as an individual patient.

Buying zolbetuximab as an individual patient

If your doctor prescribes zolbetuximab for pancreatic cancer, but it's unavailable in your country, you can buy and import it under the so-called Named Patient Import regulation.

The regulation applies when a medicine:

• is for a life-threatening or debilitating condition;
• is approved elsewhere;
• has no local alternatives, and
• is for personal use.

This process requires a prescription from your treating doctor. Other documentation requirements may apply, depending on your country.

Do you want to use the Named Patient Import regulation to get zolbetuximab before it's approved for pancreatic cancer or available in your country? You will first need to consult your treating doctor and get a suitable prescription.

Already have a prescription? Share it with our team at Everyone.org, so we can support you with buying Vyloy immediately.