CHMP Recommends Jazz Pharmaceuticals Therapy for Biliary Tract Cancer

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Jazz Pharmaceuticals, headquartered in Dublin, Ireland, announced on April 25, 2025 that the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion to recommend conditional marketing authorization for zanidatamab, a dual human epidermal growth factor receptor 2 (HER2)-targeted bispecific antibody (1). The investigational treatment is being recommended as a monotherapy for treatment of adults who have unresectable locally advanced or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC) and have previously received one line of systemic therapy.

The positive opinion was based on data from a Phase IIb trial (HERIZON-BTC-01), which evaluated the performance of zanidatamab in previously treated, inoperable, and advanced or metastatic HER2-positive BTC (1).

Jazz Pharmaceuticals had announced, on Nov. 7, 2023, a five-year collaboration with MD Anderson Cancer Center to evaluate zanidatamab in HER2-expressing cancers (2,3).

“This positive CHMP opinion is a welcome step for physicians and patients in Europe who face a critical unmet need in HER2-positive biliary tract cancers, a rare and aggressive group of cancers with poor prognosis and limited treatment options,” said Robert Iannone, MD, executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals, in a company press release (1). “If approved, zanidatamab would be the first HER2-targeted therapy licensed for this difficult-to-treat cancer in the EU [European Union], marking an important milestone in addressing this unmet need. We look forward to the European Commission’s decision and the opportunity to provide a new treatment option for patients.”

Biliary tract cancers include gallbladder cancer and cholangiocarcinoma (CCA), the latter of which is the second-most common primary liver cancer following hepatocellular carcinoma (1). The global mortality rate of CCA has risen in recent decades, according to Jazz, although BTCs account for less than 1% of cancers in humans.

However, curative surgery is an option only for a minority of patients, as early BTC disease symptoms are vague or nonspecific in nature, meaning most cases are diagnosed at an advanced stage (1). Second-line treatments have been largely limited to chemotherapy, Jazz said, in the absence of molecular analysis.

CHMP’s April 2025 recommendation will be reviewed with an expectation of a final decision by the European Commission, which has authority to approve medicines in all EU member states, Iceland, Liechtenstein, and Norway (1).

In addition to its development of treatment for BTCs, Jazz Pharmaceuticals is also the maker of the first and currently only commercially approved cannabidiol (CBD) drug product on the market (Epidiolex), relative to which clinical study data showed, in February 2025, that a CBD drug product intermediate from DSM-Firmenich, CBtru, is absorbed into the bloodstream at a similar level (4).

In November 2023, Autifony Therapeutics announced that it had entered into an exclusive global licensing agreement with Jazz to discover and develop drug candidates targeting two ion channels linked to neurological disorders, under which Autifony would lead drug discovery and preclinical development, and Jazz would lead all subsequent clinical development, manufacturing, regulation, and commercialization (5).

References

1. Jazz Pharmaceuticals. Jazz Pharmaceuticals Receives CHMP Positive Opinion for Zanidatamab for the Treatment of Advanced HER2-Positive Biliary Tract Cancer. Press Release. April 25, 2025.
2. Barton, C. Frontrunners in Bispecific Antibodies. Pharmaceutical Technology 2024, 48 (3) 13–15.
3. Jazz Pharmaceuticals. Jazz Pharmaceuticals and MD Anderson Announce Five-Year Collaboration to Evaluate Zanidatamab in HER2-Expressing Cancers. News Release. 7 Nov. 2023.
4. DSM-Firmenich. New Clinical Study Validates dsm-firmenich’s CBtru as a Promising Pathway for Patient-Friendly CBD-Based Therapies. Press Release. Feb. 20, 2025.
5. Autifony Therapeutics. Autifony Announces Exclusive Global License and Collaboration Agreement with Jazz Pharmaceuticals on Two Ion Channel Targets for Neurological Disorders. Press Release. Nov. 14, 2023.