Canadian Biotech Zymeworks Achieves FDA Approval for Rare GI Cancer Drug

The United States Food and Drug Administration (FDA) has officially approved Zihera, a groundbreaking cancer-treating antibody originally developed by Vancouver-based biotech company Zymeworks. The approval represents a significant milestone in the fight against metastatic HER2-positive biliary tract cancer, offering new hope for patients battling this rare and aggressive disease.

Zihera (zanidatamab) was brought to market by Jazz Pharmaceuticals, a global biopharmaceutical company headquartered in Dublin, Ireland. The drug targets adults suffering from metastatic HER2-positive biliary tract cancer, a form of cancer that originates in the bile ducts, gallbladder, or nearby structures and has spread to other parts of the body. Jazz Pharmaceuticals secured the commercialization rights to Zihera in 2022 in a deal with Zymeworks valued at $375 million USD upfront, with the potential for additional royalties based on net sales across North America, Europe, and Japan.

Metastatic HER2-positive biliary tract cancer is exceptionally rare and deadly, with an incidence rate of roughly two per 100,000 individuals in Western countries, including Canada. The disease has a grim prognosis, with a five-year survival rate of under five percent. In this context, Zihera represents a transformative development, as it is a non-chemotherapy option designed to improve outcomes for patients with advanced stages of the disease.

The FDA’s approval was granted following the promising results of a Phase 2b clinical trial. In this study, 52 percent of participants experienced significant tumor shrinkage, with the benefits lasting an average of 15 months. Rob Iannone, executive vice president, global head of research and development, and chief medical officer at Jazz Pharmaceuticals, emphasized the importance of this development, stating that it represents a major advance in addressing an area of high unmet medical need.

The announcement also sparked celebrations within the Canadian biotech community, as it highlights the success of Zymeworks, a company co-founded in Vancouver in 2003. Ali Tehrani, Zymeworks’ co-founder and former president and CEO, expressed his excitement in a LinkedIn post, reflecting on the journey from dream to reality. “Back in 2003, when I co-founded the company, this was the dream that many said was not possible,” Tehrani wrote. “Today it became reality—most importantly for patients who will benefit from it.”

The FDA approval of Zihera signifies a transformative milestone for both Jazz Pharmaceuticals and Zymeworks, underscoring their leadership in the development of groundbreaking cancer treatments. This achievement not only validates their commitment to addressing some of the most challenging medical conditions but also represents a critical step forward in the fight against metastatic HER2-positive biliary tract cancer—a disease with historically limited treatment options and dismal survival rates.

As Zihera becomes accessible in the United States, it offers a beacon of hope for patients and their families, with the potential to significantly enhance survival outcomes and improve quality of life. The non-chemotherapy nature of the drug further distinguishes it as a patient-friendly alternative, capable of reducing the burden of treatment while delivering impactful results. With half of the trial participants experiencing tumor shrinkage and the benefits extending over 15 months on average, Zihera is set to redefine the standard of care for this aggressive form of cancer.

Jazz Pharmaceuticals’ global vision extends beyond the U.S. market. By targeting approvals in Europe, Japan, and other regions, the company is poised to reach a broader patient population, amplifying its impact on global healthcare. This worldwide expansion reflects a steadfast dedication to ensuring that innovative therapies like Zihera are available to those in need, regardless of geography.

For Zymeworks, this milestone also celebrates its long-standing efforts in antibody research and development. Co-founder Ali Tehrani’s remarks capture the spirit of perseverance and innovation that brought this dream to fruition. The approval is a testament to the collaborative spirit of the biotech sector and serves as inspiration for other emerging players in the field.

With Zihera now paving the way, both Jazz Pharmaceuticals and Zymeworks are positioned to continue shaping the future of cancer treatment, offering patients not just new options, but also renewed hope. This breakthrough reaffirms that meaningful progress in healthcare is not just about tackling diseases but also about improving lives on a global scale.