Zanidatamab’s Approval for Biliary Tract Cancer: How Long Will You Have to Wait?

For patients with HER2-positive biliary tract cancer (BTC) who have progressed after initial treatment, prognosis remains poor. Currently, there are no HER2-targeted therapies approved for BTC, as there are for other types of cancer.

Zanidatamab, an investigational HER2-targeted therapy, aims to address this gap. Recent clinical trial data suggest it may improve patient outcomes by delivering durable antitumor responses and manageable safety ¹. If approved, zanidatamab could significantly alter the BTC treatment landscape. It could offer an option for patients who have run out of options.

However, zanidatamab is still awaiting regulatory approval from agencies like the FDA and EMA. So, when might patients worldwide gain access to this new targeted therapy for BTC?

What is zanidatamab used for?

Zanidatamab is developed to treat multiple HER2-positive solid tumors. Including advanced or metastatic gastroesophageal adenocarcinoma (GEA), biliary tract cancer (BTC), and breast cancer ².

As a dual HER2-tageted bispecific antibody, zanidatamab binds to two different sites on the HER2 protein. In addition, it enhances the immune system's ability to attack cancer cells. This binding mechanism may help it overcome resistance seen with other HER2-targeted therapies.

Clinical trials are ongoing for zanidatamab in breast cancer and GEA. However, it is already under priority review for FDA approval for treating previously treated, unresectable, HER2-positive, locally advanced, or metastatic biliary tract cancer ³.

What are the clinical trial results of zanidatamab?

Accoding to the published results of the long-term Phase 2b HERIZON-BTC-01 trial, these are the key outcomes shown by zanidatamab:

• 41.3% of patients treated with zanidatamab had a partial or complete response;
• The median duration of response was 14.9 months for patients with high HER2 expression (IHC 3+). For patients with lower HER2 expression (IHC 2+), it was 7.5 months;
• The median overall survival was 15.5 months (18.1 months for IHC 3+ patients and 5.2 months for IHC 2+ patients);
• The median progression-free survival was 5.5 months ⁴.

These results highlight the potential of zanidatamab to address a significant unmet need for targeted therapies that can improve BTC overall survival. Current standard-of-care therapies achieve overall survival of 6 to 9 months. According to its clinical trial results, zanidatamab could significantly improve this result ⁴.

When will zanidatamab get FDA approval?

Zanidatamab is currently being reviewed by the FDA. The target decision date is 29 November 2024 ³. If positive, this will be the first approved targeted therapy for HER2-expressing biliary tract cancer.

When will zanidatamab get EMA approval?

Earlier in 2024, the EMA accepted the application for zanidatamab's marketing authorization in Europe. This application is curently still in review.

Typically, EMA approval decisions take up to 210 days. If the agency sticks to this timeline, we could expect zanidatamab's EMA approval to become a fact towards mid-2025. Unless, of course, the decision is negative or additional information is requested from the manufacturer.

When will zanidatamab be available in the UK?

This is a more difficult question to answer, as there is no active application for the marketing authorization of zanidatamab in the UK.

However, such an application is not a prerequisite for approval. After Brexit, the MHRA could decide to approve a medicine on the basis of another trusted agency's approval (e.g. the FDA or the EMA). If the MHRA goes down this road, it could theoretically approve it as soon as zanidatamab gets its FDA approval (hopefully in November 2024), or its EMA approval (hopefully by mid 2025).

How to get zanidatamab for BTC before it's available in your country

Medicine approval processes around the world take time. If you're in urgent need of treatment for your biliary tract cancer, that can be discouraging. However, the good news is you don't have to wait until zanidatamab is available in your country. You just need to wait until it gets an approval somewhere in the world. As soon as that happens, you can access it via the Named Patient Import regulation.

Here's how it works.

Buy zanidatamab as a Named Patient

Patients worldwide are legally allowed to buy and import medicines for personal use, if they could improve their lives or address life-threatening conditions. Specifically if these medicines are not approved or available locally yet.

If you want to start your treatment with zanidatamab before it's approved where you are located, this might be an option for you and your doctor. The regulation that makes this possible is known as Named Patient Import. Under this regulation, patients can import a medication not yet approved or available in their country if:

• it is approved elsewhere;
• it has no local alternatives, and
• it is for personal use.

This process requires a prescription from your treating doctor. And you can only make use of it after zanidatamab has approval somewhere in the world. Other documentation requirements may apply, depending on your country.

Do you want to use the Named Patient Import regulation to get zanidatamab before its FDA, MHRA or EMA approval? 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 zanidatamab as soon as it gets its first global approval. Wherever that may be.

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References:

1. Zanidatamab in previously-treated HER2-positive (HER2+) biliary tract cancer (BTC): Overall survival (OS) and longer follow-up from the phase 2b HERIZON-BTC-01 study. Journal of Clinical Oncology, 299 May 2024. 
2. Jazz Pharmaceuticals Presents Updated Phase 2 Data for Zanidatamab Demonstrating Increased mPFS in HER2-Positive Metastatic Gastroesophageal Adenocarcinoma at ESMO 2024. Jazz Pharmaceuticals plc. Investors, 16 September 2024.
3. Wahner, Ashling. FDA Grants Priority Review to Zanidatamab for HER2+ Metastatic Biliary Tract Cancer. OncLive, 29 May 2024.
4. Jazz Pharmaceuticals Presents Overall Survival and Longer Follow-Up Data from HERIZON-BTC-01 Trial Evaluating Zanidatamab in Previously Treated HER2-Positive Biliary Tract Cancer at ASCO 2024. Jazz Pharmaceuticals plc. Investors, 1 June 2024.