New therapies to stimulate growth of AML market
By Mia Burns
As a boost of new therapies enters the acute myeloid leukemia market along with an increasing number of cases among the elderly, revenue for the market across the United States, France, Germany, Italy, Spain, and the United Kingdom will increase from $151 million in 2012 to $430.7 million in 2017. This will represent a compound annual growth rate of 23.3 percent, according to a new report from GlobalData. Although the market will grow substantially during this five-year period, lackluster clinical trial results and cost-consciousness may continue to plague drug development.
In 2012, the majority of the acute myeloid leukemia market therapies sales were generated in the United States, which enjoyed a market share of $108.8 million, compared to sales in the five European Union countries, which were estimated to be about $42 million. By 2017, the acute myeloid leukemia market in the United States and the five European Union countries is expected to reach $324.4 million and $106.3 million in sales, respectively.
“The primary driver of the increase of the acute myeloid leukemia market size is the increased use of branded therapies more so than an increasing incidence,” says Cheryl S. Gradziel, Ph.D., GlobalData’s analyst covering oncology. “Since most patients are currently treated with generics, even minimal uptake of premium priced new agents will have a major impact on market size.” Such therapies include Sunesis’ vosaroxin for relapsed and refractory acute myeloid leukemia, Novartis’ midostaurin for patients with FLT3 mutations, and Cyclacel’s sapacitabine for newly-diagnosed elderly patients.
According to the report’s key findings, the uptake of new drugs will be limited despite high levels of unmet need as key opinion leaders are relatively unimpressed by these agents. In addition, key opinion leaders are unconvinced that FLT3 inhibitors will be as successful as monotherapies in acute myeloid leukemia market.
“The biology of AML is incredibly complex, and is still poorly understood,” Dr. Gradziel told Med Ad News Daily. “FLT3 is one of the few molecular targets that has been implicated in disease progression. There is great hope that FLT3 inhibitors will be the first small molecule targeted therapies to improve the outlook of the subset of acute myeloid leukemia patients with FLT3 mutations. However, these mutations are not believed to cause acute myeloid leukemia, but instead occur late in pathogenesis. This means that FLT3 inhibitors will not be effective in the relapse-causing leukemic stem cells. So while some key opinion leaders think that FLT3 inhibitors will be useful tools for patients with FLT3 mutations, most feel strongly that these targeted therapies must be prescribed in combination with standard chemotherapy rather than as monotherapies if they are to be truly effective.”
A lack of breakthrough efficacy of pipeline drugs has failed to impress clinicians. “Most of the drugs in late-stage development have shown promise in early studies, usually at achieving complete remission (CR),” Dr. Gradziel says. “However, this does not always translate to overall survival benefits, so key opinion leaders are hesitant to assume that promising results from Phase II studies will be reproducible in Phase III and, ultimately, be clinically relevant. Acute myeloid leukemia clinicians are looking for therapies that are game-changers, not ones that provide only small improvements over the current standard of care. They want to see drugs that can achieve higher CR rates and CRs of longer duration, which indicates the quality of remission. Concomitant improvements in duration of overall survival compared with the current standard of care are critical as well, especially in order to gain regulatory approval. Ideally, these therapies will be well tolerated, especially by the elderly, and improve patients’ overall quality of life. Without dramatic improvements in one or more of these areas compared with the current standard of care, it will be difficult for key opinion leaders to justify the high cost of premium-priced new therapies.”
In the past, there was a severe shortage of cytarabine, the backbone of intensive chemotherapy for acute myeloid leukemia, but this issue has largely been resolved, according to Dr. Gradziel. “Cytarabine is no longer listed under the FDA’s Current Drug Shortages,” she told Med Ad News Daily. “Although two manufacturers’ generic formulation of daunorubicin is on the FDA’s Current Drug Shortage list, daunorubicin is currently still available from other manufacturers. Additionally, daunorubicin can be replaced by other anthracyclines in the treatment of acute myeloid leukemia given a shortage. None of the key opinion leaders interviewed in the United States or the five European Union countries reported difficulties with obtaining any of the chemotherapy drugs for acute myeloid leukemia. Consequently, we don’t emphasize drug shortages as a barrier to growth in the market.”
Posted: August 2013