It was one of the first new drugs approved for acute myeloid leukemia in years. But more than a year after the Food and Drug Administration gave it the green light, and despite impressive clinical data, the manufacturer expects to see lower-than-expected sales this year.
Dublin, Ireland-based Jazz Pharmaceuticals said in its third-quarter 2018 earnings call last month that sales of Vyxeos (cytarabine:daunorubicin) were below expectations, reaching $21 million for the quarter and $75 million for the year to date. Consequently, it lowered its 2018 sales guidance from a range of $115-135 million to $95-110 million, citing several uptake issues like restrictions on usage by healthcare institutions. But an oncologist specializing in AML who also participated in clinical trials of the drug said some of the reasons likely boil down to its cost and impressions from the physician community.
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Following news of the third-quarter results, shares of Jazz, which had closed at $164.41 on Nov. 6, closed down more than 14 percent the next day.
Vyxeos received FDA approval in August 2017 for newly diagnosed AML in adults that arises secondarily to other drug therapies or myelodysplastic syndrome. It was one of the first drugs approved for AML in more than a decade, with the FDA decision coming four months after the approval of Novartis’s Rydapt (midostaurin), for newly diagnosed AML patients whose disease carries a mutation called FLT3, and two days after the approval of Celgene and Agios’s Idhifa (enasidenib) for IDH2-mutant AML.
Pfizer’s Mylotarg (gemcitabine ozogamicin) had been approved for AML in 2000, but was pulled from the market due to toxicity concerns in 2010. However, subsequent clinical testing has resolved the concerns, and it was approved again last year.
But rather than having a novel mechanism of action, Vyxeos is a new take on an old treatment for AML, encapsulating two chemotherapy drugs – daunorubicin and cytarabine – into a single liposome. The two-drug regimen, which has been in use for decades, is often called “7+3” because cytarabine is administered continuously via 24-hour infusion over seven days, while daunorubicin is administered for three days. By contrast, Vyxeos is given over 90 minutes on days 1, 3 and 5 for induction and then days 1 and 3 for consolidation, thus being significantly more convenient for patients and clinical staff.
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And as data presented at the American Society of Hematology’s 2018 annual meeting show, Vyxeos is also more effective than 7+3. Data from a Phase III study among older adults with high-risk and secondary AML showed that overall survival – the gold standard for efficacy in AML – was significantly longer in patients receiving Vyxeos than for those receiving 7+3. Another study indicated that Vyxeos can even be given on an outpatient basis.
So why, despite the drug’s convenience and efficacy advantages, have sales fallen short?
Dr. Gary Schiller, an oncologist and professor of medicine at the University of California Los Angeles who has participated in several Vyxeos trials, said the reasons boiled down to the drug’s high cost and impressions based on its mechanism of action and the clinical trials used for its approval. “The mechanism of action was not sufficiently ‘sexy’ for the audience,” he said in a phone interview. “Some of the big hitters in the leukemia community were not on the trial, and that’s always politically an issue.”
Schiller also cited the drug’s cost as a problem. A spokesperson for the company said the wholesale acquisition cost for Vyxeos is in the range of $47,895 and $79,825 for the entire course of therapy. “We strongly believe that Vyxeos represents a cost-effective treatment option based on currently accepted pharmacoeconomic standards for patients, compared to the 7+3 treatment regimen,” she wrote in an email.
Still, the spokesperson acknowledged the company has encountered barriers to wider adoption, particularly for secondary AML. Consequently, she said, it is implementing a number of initiatives to address the issue, including educating those treating the disease about its survival benefits. Additionally, it is working to increase awareness about the New Technology Add-on Payment that became available for the drug on Oct. 1.
Yet, Schiller said reimbursement was another issue, especially early on. When the drug was released, he said, some centers found they could get it reimbursed as an outpatient drug, but not as an inpatient drug. That, he said may have an an initial numbing effect on doctors.
“I think sometimes politics and impressions affect the marketplace,” Schiller said. “But we have randomized Phase III data that show it is statistically significantly better than conventional chemotherapy.”
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