EMA grants Orphan Drug Designation to Zai Lab’s DLL3-targeting ADC zocilurtatug pelitecan for pulmonary neuroendocrine carcinomas, supporting development in SCLC and other neuroendocrine cancers.
Written By: Chikkula Pavan Kumar, PharmD
Reviewed By: Pharmacally Editorial Team
Zai Lab has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for zocilurtatug pelitecan (zoci; formerly ZL-1310), its investigational Delta-like ligand 3 (DLL3)-targeting antibody-drug conjugate (ADC), for the treatment of pulmonary neuroendocrine carcinomas (NECs). The designation follows a positive opinion from the EMA’s Committee for Orphan Medicinal Products (COMP), which recognized the severity of pulmonary NECs and the significant unmet need for new therapeutic options.
Regulatory Recognition for High-Unmet-Need Cancer
In granting the designation, the COMP highlighted preliminary clinical evidence suggesting that zoci may provide a clinically relevant advantage over currently available therapies for patients with relapsed or refractory extensive-stage small cell lung cancer (SCLC), including the potential for durable antitumor responses.
“Receiving Orphan Drug Designation from the EMA is an important milestone for zocilurtatug pelitecan,” said Rafael G. Amado, M.D., President and Head of Global Research and Development at Zai Lab. “This recognition supports our efforts to advance innovative treatment options for patients with pulmonary neuroendocrine carcinomas and other difficult-to-treat cancers.”
Addressing an Aggressive Disease
Pulmonary neuroendocrine carcinomas comprise a spectrum of aggressive lung cancers characterized by rapid disease progression and poor long-term outcomes. Small cell lung cancer represents the most significant subtype, accounting for approximately 15% of the estimated 2.5 million lung cancer cases diagnosed globally each year.
Affecting an estimated 375,000 patients worldwide annually, SCLC remains one of the most aggressive and lethal solid tumors. Despite advances in first-line treatment, outcomes following disease progression remain poor, underscoring the need for novel therapeutic approaches capable of delivering durable clinical benefit.
Promising Early Clinical Findings
Zocilurtatug pelitecan is an antibody-drug conjugate designed to selectively bind DLL3-expressing tumor cells and deliver a cytotoxic payload directly to cancer cells. DLL3 is a validated therapeutic target that is highly expressed across several neuroendocrine malignancies and is generally associated with unfavorable clinical outcomes.
Preliminary clinical data have demonstrated encouraging antitumor activity and durable responses in patients with relapsed or refractory extensive-stage SCLC. Clinical studies to date have also demonstrated a generally manageable safety profile, supporting continued evaluation of the investigational therapy across multiple neuroendocrine cancers.
Zoci is currently being evaluated in an ongoing global Phase 1 clinical study (NCT06179069) in patients with extensive-stage SCLC.
Regulatory Incentives and Development Path
The EMA’s Orphan Drug Designation provides several regulatory incentives intended to support the development of therapies for rare diseases, including protocol assistance, reduced regulatory fees, and enhanced interactions with European regulators. Following potential approval, the designation may provide up to ten years of market exclusivity within the European Union.
The European designation further strengthens the program’s regulatory momentum. In the United States, the Food and Drug Administration (FDA) has previously granted both Fast Track Designation and Orphan Drug Designation to zoci for the treatment of SCLC. More recently, the agency granted Fast Track Designation for the treatment of extrapulmonary neuroendocrine carcinomas (epNECs).
Expanding Development Across Neuroendocrine Tumors
Building on the encouraging clinical and regulatory progress, Zai Lab plans to initiate three registration-enabling studies by the end of 2026. The planned development program will evaluate zoci in second- and third-line SCLC, first-line SCLC, and extrapulmonary neuroendocrine carcinomas.
The latest EMA designation reinforces growing confidence in DLL3-targeted therapies and positions zocilurtatug pelitecan as a potentially important new treatment option for patients with aggressive neuroendocrine cancers, where effective therapies remain limited and outcomes continue to be poor.
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About the Writer
Chikkula Pavan Kumar (LinkedIn), PharmD is a Doctor of Pharmacy with a keen interest in clinical pharmacy, pharmacovigilance, and evidence-based practice. In his words, he is passionate about patient safety and translating complex medical information into clear, research-driven communication.