US FDA rejects Eisai/Astex drug for AML indication
This article was originally published in Scrip
Investors obviously had been preparing themselves for a rejection from the US FDA for Eisai's and Astex Pharmaceuticals' nucleoside metabolic inhibitor Dacogen (decitabine) as a treatment for acute myeloid leukaemia (AML) in older adults, with shares of the latter company first falling 3.8% in morning trading on 7 March, before the stock regained its composure, even getting a bump of 2.7%, or 5 cents.
Dacogen already is approved as a treatment for myelodysplastic syndromes (MDS) in the US, an indication it won in May 2006.
The drug's safety profile in the AML population was similar to that of the MDS patients in the Phase III study.
But the firms said the FDA declined to approve the expanded indication in the AML population because the prespecified analysis of the primary endpoint in the DACO-016 study did not demonstrate statistically significant superiority of Dacogen over the control arm of low-dose cytarabine or best supportive care, although the drug showed a 2.7-month improvement in median overall survival, 7.7 months versus 5 months (scripintelligence, 8 February 2012).
The rejection from US regulators for Dacogen in the AML indication hardly came as a surprise, given that Dr Richard Pazdur, director of the FDA's Office of Hematology and Oncology Products, said at an advisory committee meeting last month that for a drug to be approved in the US, "one has to demonstrate clinical benefit."
"It is not just a demonstration for activity. The drug approval process is not a screening process for drug activity," Dr Pazdur told the FDA's Oncologic Drugs Advisory Committee (ODAC) at a 9 February 2012 meeting (scripintelligence, 10 February 2012).
Although the ODAC voted 10-3, with one abstention, against approval, some of the oncologists on the panel said they would nonetheless use Dacogen to treat newly diagnosed AML in patients 65 years or older who are not candidates for induction chemotherapy.
Treatment options for elderly patient with AML, a rapidly progressive disease, with a median survival ranging from 3.9 months for patients 65 to 74 years of age to 1.4 months for patients 85 or older, are limited.
Elderly patients with AML are often either not candidates for or choose not to receive standard induction chemotherapy.
There currently are no approved therapies for AML patients who do not receive standard induction chemotherapy.
Astex noted that a separate marketing authorization application (MAA) for Dacogen as a treatment for AML was submitted to the European Medicines Agency in May 2011 by the Dublin, California-based biotech's partner Janssen, a Johnson & Johnson subsidiary.
The EMA is expected to issue a decision on the MAA later this year, Astex said.