EMA Grants Orphan Drug Designation to Zai Lab's Zoci for Lung-Derived Neuroendocrine Carcinoma

On June 16, Zai Lab announced that the European Medicines Agency (EMA) has granted orphan drug designation to zocilurtatug pelitecan (known as "zoci," formerly ZL-1310), a potentially first-in-class antibody-drug conjugate (ADC) targeting delta-like ligand 3 (DLL3), for the treatment of neuroendocrine carcinoma originating in the lung.

The designation follows a positive opinion from the EMA's Committee for Orphan Medicinal Products (COMP), which recognized the severity of neuroendocrine carcinoma and the urgent need for new treatment options.

Small cell lung cancer is the most prevalent form of lung-derived neuroendocrine carcinoma. Globally, approximately 2.5 million new lung cancer cases are diagnosed each year, with small cell lung cancer accounting for roughly 15%—an estimated 375,000 new cases annually. It remains one of the most aggressive and rapidly progressive solid tumors, carrying a particularly poor prognosis.

In granting the orphan drug designation, the COMP noted that preliminary clinical data in patients with relapsed or refractory extensive-stage small cell lung cancer suggest that zoci may offer superior efficacy compared with currently approved therapies, including durable responses. The committee concluded that this represents a clinically relevant advantage.

Prior to the EMA's decision, the U.S. Food and Drug Administration had already granted zoci Fast Track Designation and Orphan Drug Designation for the treatment of small cell lung cancer. More recently, the FDA also extended Fast Track Designation to zoci for the treatment of extrapulmonary neuroendocrine carcinoma.

The accumulating regulatory milestones across both the U.S. and Europe underscore the growing recognition of zoci's potential in addressing a group of highly lethal neuroendocrine malignancies with limited therapeutic options.