GSK ADC Gains Orphan Drug Status in Europe

GSK plc (LON: GSK) announced its B7-H3-targeted antibody-drug conjugate (ADC), GSK’227, has been granted Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for pulmonary neuroendocrine carcinoma (NEC), a category including small-cell lung cancer (SCLC).

The ODD is based on encouraging early clinical data from the Phase I ARTEMIS-001 trial, which demonstrated durable responses in patients with extensive stage SCLC (ES-SCLC) treated with GSK’227.

The designation provides GSK with potential benefits, including market exclusivity and reduced regulatory fees, further incentivizing the development of this promising therapy.

An estimated 250,000 individuals globally are diagnosed with SCLC annually, resulting in approximately 200,000 deaths.

This marks the fourth regulatory designation for GSK’227, affirming its potential across various solid tumor types, including lung, prostate, and colorectal cancers.

Previously, GSK’227 received Priority Medicines (PRIME) designation from the EMA for relapsed or refractory ES-SCLC and Breakthrough Therapy Designations from the US FDA for relapsed or refractory ES-SCLC and relapsed or refractory osteosarcoma.

These designations expedite the drug development process, allowing for closer collaboration with regulatory agencies and potentially faster access to market.