atients with rare types of blood cancer were given a new ray of hope this week after Celgene’s novel drug Vidaza – which it acquired via its $2.9 billion purchase of Pharmion in 2007 - was approved in the UK, five years after it was launched across the Atlantic.

Vidaza (azacitidine) is the first in a new class of epigenetic therapies – which target gene errors causing disease - to be given the green light in Europe, after being cleared by UK regulators to treat certain forms of myelodysplastic syndromes, including the subgroups chronic myelomonocytic leukaemia (CML) and acute myeloid leukaemia (AML).

MDS encompasses a group of incurable blood cancers that occur in immature blood cells, for which treatment to date has largely been focused on managing related symptoms, such as fatigue. Nearly 2,000 patients a year are diagnosed with MDS and, according to the MDS Foundation, in 30% of cases the disease will progress to AML, which can be fatal in just six to 12 months.

Until now, the only curative option for MDS was a blood stem cell transfusion but, according to the company, this is not suitable for many patients, and therefore Vidaza fills an urgent unmet medical need as it offers a means by which to fight the underlying cause of the condition and extend life expectancy.

In patients with MDS, the genes responsible for bone marrow cell multiplication and their subsequent development into blood cells are blocked by methylation. Vidaza works by preventing this process so that the genes can become active again, and so “represents a major step forward in prolonging and improving the lives of patients with this incurable cancer,” according to Celgene.

Clinical studies have clearly demonstrated Vidaza’s benefits to MDS patients, and its regulatory approval was based on a Phase III trial showing that it significantly extended median survival of higher-risk patients from 15 months to over two years compared to conventional care regimens.

In addition, data published in the Lancet Oncology just this week shows that the drug also reduces the burden of hospital stays by 25% and cuts infections by a third compared to CCR, as well as the lowering the need for patients to undergo blood transfusions, the company said.

NICE delay?
David Hall, Chairman of the MDS UK Patient Support Group, said: “The granting of full marketing authorisation for Celgene’s Vidaza will bring new hope to MDS patients, particularly those with higher-risk forms of these diseases that historically have been associated with a bleak outlook”.

However, he stressed that, in UK, approval is “no guarantee” that patients will get access to “such a new and potentially highly effective therapy”, because until the UK’s cost effectiveness watchdog the National Institute for Health and Clinical Excellence has “exercised its lengthy approval procedure”, the prescription of new drugs is largely at the discretion of local primary care trusts.

According to Hall, “a combination of procedural inertia, ignorance of the degree of patient suffering and misplaced costing assumptions, make it extremely unlikely, in the majority of the UK regions, that the required levels of funding will be authorised,” and he concluded that this, “coupled with the long approval cycle from NICE, represent a frustrating, unnecessarily long and painful wait for medication”.