Riyadh: The Saudi Food and Drug Authority (SFDA) has granted approval for the registration of Pemazyre (Pemigatinib) as a standalone treatment for adults with locally advanced or metastatic cholangiocarcinoma. This approval is specifically for patients with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement who have already undergone at least one prior line of systemic therapy.
According to Saudi Press Agency, Pemazyre functions by inhibiting the FGFR2 receptor found on certain cancer cells. This receptor is crucial in promoting tumor growth. By targeting FGFR2, Pemazyre disrupts the signaling pathways responsible for cancer cell proliferation, thereby slowing down the progression and spread of the tumor.
The SFDA announced that the decision to register Pemazyre was made following a thorough assessment of its efficacy, safety, and quality, as per the approved regulatory standards. Clinical study results demonstrated an objective response rate (ORR) of 37%, with a complete response observed in 2.8% of patients and a partial response in 34.3%.
Based on these findings, the SFDA believes that Pemazyre offers a significant treatment option for patients with limited alternatives after previous treatments have failed. This contributes to better disease management and extends patient survival.
The SFDA further explained that the registration was conducted under the principle of accelerated approval, using response rate and duration of response as provisional indicators of long-term clinical benefit. There is an ongoing requirement to perform confirmatory studies to validate the long-term benefits.
Clinical trials have highlighted that the most common side effects include hyperphosphatemia, alopecia, diarrhea, dry mouth, fatigue, and taste disturbances. Eye changes may also occur, warranting regular monitoring during treatment to ensure patient safety.
This approval is part of the SFDA's continuous efforts to foster innovation in healthcare and expand treatment options for patients. It aligns with the Health Sector Transformation Program, a component of Saudi Arabia's Vision 2030, aimed at enhancing quality of life and healthcare system sustainability.