Zai Lab has received orphan drug designation (ODD) from the European Medicines Agency (EMA) for zocilurtatug pelitecan (Zoci), its investigational delta-like ligand 3 (DLL3)-targeting antibody-drug conjugate (ADC) to treat pulmonary neuroendocrine carcinomas (NECs).
The EMA’s Committee for Orphan Medicinal Products (COMP) recommended the designation, recognising the critical need for novel treatment approaches in NECs.
The COMP noted that early clinical data in patients with relapsed or refractory extensive-stage small cell lung cancer (SCLC) indicate zoci may demonstrate a more favourable effect than existing authorised therapies.
The committee highlighted durable responses and described this as a “clinically relevant advantage".
The US Food and Drug Administration (FDA) previously granted zoci fast track and orphan drug designations for SCLC. More recently, it also extended fast track designation to cover extrapulmonary NECs (epNECs).
Zai Lab president and global research and development head Rafael Amado said: “This important designation from the EMA supports Zoci’s potential to become a first-in-class therapy for pulmonary neuroendocrine carcinomas. This milestone is another demonstration of Zai Lab’s commitment to address critical unmet needs for patients with limited treatment options.”
The EMA’s ODD programme offers regulatory and development benefits, including the possibility of market exclusivity following approval, reduced development fees, and support to enhance clinical development efficiency for orphan drugs.
Zoci targets DLL3, extensively expressed in cancers such as SCLC and epNECs, and generally linked to poor patient outcomes.
Zai Lab plans three registration-enabling studies for Zoci, across second-line and third-line SCLC, first-line SCLC, and epNECs, aiming for completion by the end of 2026.
In April 2026, Boehringer Ingelheim and Zai Lab entered a clinical collaboration to investigate a dual DLL3-targeting approach for patients with extensive-stage small cell lung cancer (ES-SCLC) and other neuroendocrine carcinomas (NECs).
