GSK Receives EMA Orphan Drug Designation for GSK’227 in Small-Cell Lung Cancer, Strengthening Its ADC Oncology Pipeline

GSK Receives EMA Orphan Drug Designation for GSK’227 in Small-Cell Lung Cancer, Strengthening Its ADC Oncology Pipeline

(IN BRIEF) GSK announced that its B7-H3-targeted antibody-drug conjugate GSK5764227 (GSK’227) has been granted Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for the treatment of pulmonary neuroendocrine carcinoma, including small-cell lung cancer (SCLC). The designation is supported by early Phase I data from the ARTEMIS-001 trial, which showed durable responses in relapsed or refractory ES-SCLC patients. GSK’227 is now the recipient of four regulatory designations, including PRIME status from the EMA and two Breakthrough Therapy Designations from the FDA. The development underscores GSK’s commitment to advancing its ADC portfolio and addressing unmet needs in aggressive cancers like SCLC.

(PRESS RELEASE) LONDON, 28-Oct-2025 — /EuropaWire/ — GSK plc (LSE/NYSE: GSK) announced that its investigational antibody-drug conjugate (ADC) GSK5764227 (GSK’227) has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for the treatment of pulmonary neuroendocrine carcinoma (NEC) — a category that includes small-cell lung cancer (SCLC). The designation is based on encouraging early clinical data from the Phase I ARTEMIS-001 trial, which demonstrated durable responses in patients with extensive stage small-cell lung cancer (ES-SCLC) who had relapsed or were refractory to prior therapies.

The ODD highlights the potential of GSK’227 to meet a critical unmet medical need in ES-SCLC, one of the most aggressive and difficult-to-treat forms of lung cancer. Despite advances in immunotherapy and chemotherapy, patients with relapsed or refractory disease continue to face poor survival outcomes, limited treatment options, and significant quality-of-life challenges. Globally, approximately 250,000 patients are diagnosed with SCLC every year, leading to an estimated 200,000 deaths annually.

GSK’227, a B7-H3-targeted ADC, is designed to deliver a potent cytotoxic payload directly to cancer cells expressing the B7-H3 antigen, which is overexpressed in several solid tumours. The therapy aims to minimize systemic toxicity while enhancing antitumour activity, providing a targeted approach for patients with few existing options.

“This latest regulatory milestone reinforces our confidence in GSK’227’s potential as a transformative therapy for patients with small-cell lung cancer and other solid tumours,” said a GSK spokesperson. “We are committed to accelerating the development of our ADC portfolio and bringing innovative, precision-based cancer treatments to patients who need them most.”

This marks the fourth regulatory designation for GSK’227, underscoring its growing clinical promise. The therapy has previously received Priority Medicines (PRIME) designation from the EMA for relapsed or refractory ES-SCLC and Breakthrough Therapy Designations from the U.S. Food and Drug Administration (FDA) for both relapsed or refractory ES-SCLC and osteosarcoma.

As GSK continues to expand its oncology pipeline, the company is advancing multiple ADC programs across lung, colorectal, prostate, and head and neck cancers, reinforcing its strategic focus on antibody-drug conjugates as a cornerstone of next-generation cancer treatment.

About GSK’227

GSK’227 is a novel investigational B7-H3-targeted antibody-drug conjugate composed of a fully human anti-B7-H3 monoclonal antibody covalently linked to a topoisomerase inhibitor payload. GSK acquired exclusive worldwide rights (excluding China’s mainland, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to progress clinical development and commercialisation of GSK’227. GSK’s global phase III trial for GSK’227 in relapsed ES-SCLC began in August 2025.

About GSK

GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at gsk.com.

Cautionary statement regarding forward-looking statements

GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described in the “Risk Factors” section in GSK’s Annual Report on Form 20-F for 2024, and GSK’s Q2 Results for 2025.

References

1. Wang J, et al. Presented at IASLC WCLC 2024
2. Qian Wang, Zeynep H. Gümüş, Cristina Colarossi, Lorenzo Memeo, Xintong Wang, Chung Yin Kong, Paolo Boffetta, SCLC: Epidemiology, Risk Factors, Genetic Susceptibility, Molecular Pathology, Screening, and Early Detection, Journal of Thoracic Oncology, Volume 18, Issue 1, 2023, Pages 31-46, ISSN 1556-0864, https://doi.org/10.1016/j.jtho.2022.10.002.
3. GSK. GSK receives US FDA Breakthrough Therapy Designation for its B7-H3-targeted antibody-drug conjugate in relapsed or refractory extensive-stage small-cell lung cancer. Available at: https://www.gsk.com/en-gb/media/press-releases/gsk-receives-us-fda-breakthrough-therapy-designation/.
4. GSK. GSK’s B7-H3-targeted antibody-drug conjugate, GSK’227, receives EMA Priority Medicines (PRIME) Designation in relapsed extensive-stage small-cell lung cancer. Available at: https://www.gsk.com/en-gb/media/press-releases/b7-h3-targeted-antibody-drug-conjugate-receives-ema-priority-medicines-designation-in-relapsed-extensive-stage-small-cell-lung-cancer/.
5. GSK. GSK’s B7-H3-targeted antibody-drug conjugate, GSK’227, receives US FDA Breakthrough Therapy Designation in late-line relapsed or refractory osteosarcoma. Available at: https://www.gsk.com/en-gb/media/press-releases/gsk-b7-h3-targeted-antibody-drug-conjugate-gsk227-receives-us-fda-breakthrough-therapy-designation-in-late-line-relapsed-or-refractory-osteosarcoma/.

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SOURCE: GlaxoSmithKline plc