On 22 October 2015, the US Food and Drug Administration (FDA) approved Onivyde (irinotecan liposome injection), in combination with fluorouracil and leucovorin, to treat patients with advanced (metastatic) pancreatic cancer who have been previously treated with gemcitabine-based chemotherapy.
Pancreatic cancer can be difficult to diagnose early and treatment options are limited, especially when the disease has spread to other parts of the body (metastatic disease) and surgery to remove the tumour is not possible.
The FDA granted Priority Review and orphan drug designations for Onivyde. Priority review status is granted to applications for drugs that, if approved, would be a significant improvement in safety or effectiveness in the treatment of a serious condition. Orphan drug designation provides incentives such as tax credits, user fee waivers, and eligibility for orphan drug exclusivity to assist and encourage the development of drugs for rare diseases.
The effectiveness of Onivyde was demonstrated in a three-arm, randomised, open label study of 417 patients with metastatic pancreatic adenocarcinoma whose cancer had grown after receiving the chemotherapeutic drug gemcitabine or a gemcitabine-based therapy.
The study was designed to determine whether patients receiving Onivyde plus fluorouracil/leucovorin or Onivyde alone lived longer than those receiving fluorouracil/leucovorin. Patients treated with Onivyde plus fluorouracil/leucovorin lived an average of 6.1 months, compared to 4.2 months for those treated with only fluorouracil/leucovorin. There was no survival improvement for those who received only Onivyde compared to those who received fluorouracil/leucovorin.
In addition, patients receiving Onivyde plus fluorouracil/leucovorin had a delay in the amount of time to tumour growth compared to those who received fluorouracil/leucovorin. The average time for those receiving Onivyde plus fluorouracil/leucovorin was 3.1 months compared to 1.5 months for those receiving fluorouracil/leucovorin.
The safety of Onivyde was evaluated in 398 patients who received either Onivyde with fluorouracil/leucovorin, Onivyde alone or fluorouracil/leucovorin. The most common side effects of treatment with Onivyde included diarrhoea, fatigue, vomiting, nausea, decreased appetite, stomatitis and pyrexia. Onivyde was also found to result in lymphopaenia and neutropaenia. Death due to sepsis following neutropaenia has been reported in patients treated with Onivyde.
The labeling for Onivyde includes a boxed warning to alert health care professionals about the risks of severe neutropaenia and diarrhoea.
Onivyde is not approved for use as a single agent for the treatment of patients with metastatic pancreatic cancer.
Onivyde is marketed by Merrimack Pharmaceuticals Inc. of Cambridge, Massachusetts.