FDA Halts Approval of Infigratinib for Rare Liver Cancer Treatment
Key Takeaways
- FDA withdraws approval of infigratinib for cholangiocarcinoma.
- Challenges in patient enrollment led to the decision.
- Further research is ongoing to find effective treatments.
Did You Know?
Introduction
The U.S. Food and Drug Administration (FDA) has withdrawn its accelerated approval of infigratinib, marketed as Truseltiq, for a rare type of liver cancer called cholangiocarcinoma. This decision impacts patients with advanced stages of this disease who have a specific genetic mutation known as FGFR2 fusion.
Background on Infigratinib
Infigratinib had initially earned FDA approval in May 2021 for treating adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma. This approval came through an expedited process aimed at quickly bringing treatments for serious conditions to the market.
However, this expedited approval required the drug's sponsor, QED Therapeutics, to conduct a confirmatory study to validate infigratinib's effectiveness and safety. The study in question, a Phase 3 trial named PROOF, was aimed at comparing infigratinib to the standard treatment of gemcitabine and cisplatin.
Challenges in Conducting the Study
The PROOF trial faced difficulties in enrolling enough patients, leading QED Therapeutics to ask the FDA to withdraw infigratinib's approval. The trial was not stopped due to safety issues but because it was considered commercially unfeasible to continue. This resulted in the decision to cease distributing infigratinib for this specific use.
Previous Trial Results
The initial approval of infigratinib was based on data from a Phase 2 trial involving 108 participants. The trial showed that 23% of patients experienced an overall response, with a small number achieving partial or complete responses. However, the duration of these responses was relatively short, with a median duration of five months.
Common side effects reported included high phosphate levels in the blood, increased creatinine, and various other symptoms affecting the eyes, skin, and digestive system, among others.
Eligibility Criteria
For the Phase 3 PROOF trial, participants had to be at least 18 years old with confirmed cholangiocarcinoma. Patients with gallbladder cancer or other specific conditions were excluded. Participants must not have received prior treatments that could interfere with the study's outcomes.
Treatment Protocol
Participants in the trial were divided into two groups. One group received infigratinib orally, while the other received the standard chemotherapy combination of gemcitabine and cisplatin. Those in the latter group who experienced disease progression were allowed to switch to infigratinib.
Outcome Measures
The primary endpoint for evaluating the trial's success was progression-free survival, which measures how long patients live without the disease worsening. Secondary endpoints included overall survival, response rates, and safety profiles.
Impact on Patients
The withdrawal of infigratinib's approval may affect patients seeking second-line treatments for advanced cholangiocarcinoma. It emphasizes the complexities involved in drug development and the challenges of meeting regulatory requirements.
Future Directions
Despite this setback, the pursuit of effective treatments for cholangiocarcinoma continues. Researchers and pharmaceutical companies are exploring other potential therapies to offer hope to patients with this rare and challenging condition.
Conclusion
The FDA's decision to withdraw infigratinib's approval highlights the importance of ongoing research and clinical trials to ensure the safety and effectiveness of new cancer therapies. It serves as a reminder of the rigorous process involved in bringing innovative treatments to patients in need.
