Cambridge, Mass. — Zai Lab Limited said the European Medicines Agency has granted Orphan Drug Designation to zocilurtatug pelitecan, or zoci, for the treatment of pulmonary neuroendocrine carcinomas.
Zoci, formerly known as ZL-1310, is Zai Lab’s potential first-in-class Delta-like ligand 3-targeting antibody-drug conjugate.
The designation follows a positive opinion from the EMA’s Committee for Orphan Medicinal Products, which recognized the seriousness of neuroendocrine carcinomas and the need for new treatment options.
Small cell lung cancer is the most significant pulmonary neuroendocrine carcinoma, accounting for about 15% of the roughly 2.5 million lung cancer cases diagnosed worldwide each year, or an estimated 375,000 new cases annually. Zai Lab said SCLC is one of the most aggressive and lethal solid tumors and has limited treatment options.
In granting the designation, the committee noted that preliminary clinical data in patients with relapsed or refractory extensive-stage SCLC suggest zoci may offer a more favorable effect than currently authorized therapies, including durable responses. The committee characterized the finding as a clinically relevant advantage.
The U.S. Food and Drug Administration previously granted Fast Track designation and Orphan Drug Designation to zoci for SCLC. The FDA also recently granted Fast Track designation to zoci for extrapulmonary neuroendocrine carcinomas.
“This important designation from the EMA supports zoci’s potential to become a first-in-class therapy for pulmonary neuroendocrine carcinomas,” said Rafael G. Amado, M.D., President, Head of Global Research and Development at Zai Lab. “This milestone is another demonstration of Zai Lab’s commitment to address critical unmet needs for patients with limited treatment options.”
EMA Orphan Drug Designation provides regulatory and development incentives, including potential market exclusivity following approval, reduced development fees and enhanced development efficiency.
Zoci targets Delta-like ligand 3, a validated therapeutic target that is overexpressed in many neuroendocrine carcinomas, including small cell lung cancer and extrapulmonary neuroendocrine carcinomas, and is generally associated with poor clinical outcomes.
Zai Lab said zoci is on track to potentially become its first global oncology launch. The company plans three registration-enabling studies across second- and third-line SCLC, first-line SCLC and extrapulmonary neuroendocrine carcinomas by the end of 2026.
The company said zoci’s safety profile and systemic and intracranial efficacy support its potential role as a new standard of care in previously treated extensive-stage SCLC, as well as a backbone DLL3-targeting antibody-drug conjugate in first-line combination regimens.
