Everolimus gets FDA approval for another indication

Everolimus gets FDA approval for another indication

26 Feb 2016

Everolimus (Afinitor) is now approved for treating inoperable, locally advanced or metastatic neuroendocrine tumors of gastrointestinal or lung origin, the FDA announced. The agency further specified that the tumors should be "progressive, well-differentiated [and] non-functional."

Clinical results and adverse events of the drug

Approval was based primarily on a 302-patient trial comparing everolimus with placebo, both in combination with best supportive care. Median progression-free survival was 11 months in the active-drug arm compared with 3.9 months for placebo. However, in an interim analysis, there was no difference in overall survival, and response rates (i.e., achieving significant tumor shrinkage) were 2% with everolimus and 1% with placebo.


Significant toxicities were seen with the drug as well:

  • It was discontinued in 29% of patients
  • Dose reduction or delay was needed in 70%
  • Three patients died of severe adverse reactions, according to the FDA
  • Serious adverse reactions were seen in 42% of those receiving everolimus.

Everolimus was previously approved for pancreatic neuroendocrine tumors, advanced breast and kidney malignancies, and nonmalignant tumors of the kidney and brain.

FDA’s decision into question

A recent investigation called the FDA's decision-making on the drug into question, noting the relatively modest benefits and serious toxicities seen in the drug's trials and in post-marketing surveillance.

Additional applications are reportedly pending for diffuse large B-cell lymphoma and seizures associated with tuberous sclerosis complex.


Source: MedPage Today

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