SFDA Approves Pemazyre for Treatment of Advanced Cholangiocarcinoma

RIYADH, November 10, 2025 (Saudi Arabia Breaking News) – The Saudi Food and Drug Authority (SFDA) has approved the registration of Pemazyre (Pemigatinib) as a monotherapy for adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement who have progressed after at least one prior line of systemic therapy.

Pemazyre functions by inhibiting FGFR2 receptors located on the surface of certain cancer cells. These receptors are key drivers in tumor growth, and by blocking them, the drug disrupts the signaling pathways that stimulate cancer cell proliferation and division, thereby slowing tumor progression and spread.

The SFDA stated that the registration followed a comprehensive evaluation of the total evidence regarding the drug’s efficacy, safety, and quality, in line with approved regulatory standards. Clinical results showed an objective response rate (ORR) of 37%, including a complete response in 2.8% of patients and a partial response in 34.3%.

Based on these findings, the SFDA considers Pemazyre a valuable treatment option for patients with limited therapeutic choices following previous treatment failure, contributing to better disease control and prolonged survival.

The authority clarified that the approval was granted under the accelerated approval pathway, relying on response rate and duration of response as surrogate indicators of long-term clinical benefit, with a requirement to complete confirmatory studies to verify long-term outcomes.

Clinical studies identified the most common side effects as hyperphosphatemia, alopecia, diarrhea, dry mouth, fatigue, and taste disturbances, with potential ocular effects that require regular monitoring during treatment to ensure safety.

The registration of Pemazyre reflects the SFDA’s ongoing efforts to promote innovation in the healthcare sector, expand access to advanced treatments, and enhance patients’ quality of life. It aligns with the objectives of the Health Sector Transformation Program, one of the key initiatives under Saudi Vision 2030.