REG - GSK PLC - GSK'227 US FDA Orphan Drug Designation in SCLC

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RNS Number : 9003K  GSK PLC  10 December 2025

Issued: 10 December 2025, London UK

 

GSK'227, a B7-H3-targeted antibody-drug conjugate, granted Orphan Drug
Designation in small-cell lung cancer by the US FDA

 

·   Designation supported by early clinical data showing durable responses
in certain types of small-cell lung cancer (SCLC)

·   Extensive stage SCLC is associated with high rates of relapse, few
treatment options and poor prognosis

·   Fifth regulatory designation further supports plan to accelerate
development of GSK'227 in solid tumours with transformational potential

 

 

GSK plc (LSE/NYSE: GSK) today announced that its B7-H3-targeted antibody-drug
conjugate GSK'227, now referred to by its International Nonproprietary Name,
risvutatug rezetecan, has received Orphan Drug Designation (ODD) from the US
Food and Drug Administration (FDA) for the treatment of small-cell lung cancer
(SCLC). The ODD was supported by preliminary clinical data showing durable
responses in patients with extensive stage SCLC (ES-SCLC) who were treated
with risvutatug rezetecan in the phase I ARTEMIS-001 clinical trial.(( 1 
(#_edn1) ))

 

In the US, SCLC constitutes about 13% of all lung cancers. In 2025, an
estimated 29,500 people in the US will be diagnosed with SCLC. 2  (#_edn2) Of
patients with SCLC, 70% have extensive-stage disease, meaning the cancer has
spread throughout one or both lungs and/or to other parts of the body.(( 3 
(#_edn3) )) ES-SCLC is an aggressive and difficult-to-treat cancer with
limited treatment options. The 5-year survival rate is approximately 3%.(3)
Most patients with ES-SCLC relapse after initial treatment and the median
overall survival with standard-of-care treatments for relapsed ES-SCLC is
approximately 8 months. 4  (#_edn4)

 

This designation follows the recent announcement that risvutatug rezetecan was
granted ODD from the European Medicines Agency (EMA) for the treatment of
pulmonary neuroendocrine carcinoma, a category of cancer that includes SCLC.
It is the fifth regulatory designation for risvutatug rezetecan, exemplifying
the potential of this B7-H3-targeted ADC, which is being developed in a range
of solid tumours, including lung, prostate and colorectal cancers. Previously,
risvutatug rezetecan was granted Priority Medicines (PRIME) Designation by the
EMA for relapsed or refractory ES-SCLC and Breakthrough Therapy Designations
for relapsed or refractory ES-SCLC and relapsed or refractory osteosarcoma
granted by the US FDA.(( 5  (#_edn5) ))(,( 6  (#_edn6) )),( 7  (#_edn7) )

 

About risvutatug rezetecan

Risvutatug rezetecan (GSK5764227) 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 risvutatug rezetecan. GSK's global phase III trial
(NCT07099898) for risvutatug rezetecan 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.

 

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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 Q3 Results for 2025.

 

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WC1A 1DG

 1  (#_ednref1) Wang J, et al. Presented at IASLC WCLC 2024.

 2  (#_ednref2) Cancer Facts and Figures 2025, American Cancer Society,
accessed 6 November 2025

 3  (#_ednref3) SEER Explorer Surveillance Research Program, National Cancer
Institute, accessed 27 October 2025.

 4  (#_ednref4) G. Mountzios, et al. Tarlatamab in small-cell lung cancer
after platinum-based chemotherapy. N Engl J Med, 393 (2025), pp. 349-361. DOI:
10.1056/NEJMoa2502099

 5  (#_ednref5) 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/.

 6  (#_ednref6) 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/.

 7  (#_ednref7) 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/
(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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