GSK PLC (LSE:GSK, NYSE:GSK) has received a major boost for its experimental drug GSK5764227 (GSK’227), which targets a rare and aggressive bone cancer called osteosarcoma.
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to the treatment, which is being tested for patients whose cancer has returned after at least two prior treatments.
This status is reserved for discoveries that show early evidence of significant improvement over existing options for serious conditions, the major kicker being it helps fast-track development and review processes to bring promising treatments to patients sooner.
Relapsed or refractory osteosarcoma, which mostly affects children and young adults, has no approved therapies after initial treatments fail.
“For patients with relapsed or refractory osteosarcoma, there are no approved treatment options once the cancer returns a second time,” said Hesham Abdullah, GSK’s Senior Vice President, Oncology R&D.
The FDA’s decision was based on data from the ARTEMIS-002 trial, which tested GSK’227 in 42 osteosarcoma patients and showed promising results.
The drug is also being explored for other cancers, including lung cancer and sarcoma, as part of GSK’s ongoing global trials.
Osteosarcoma is rare, with fewer than 1% of new US cancer cases annually, but it has a high recurrence rate, leaving patients with limited options after chemotherapy.
GSK’227 combines a targeted antibody with a cancer-fighting payload, representing a new approach to treating this challenging disease.