GSK’s orphan drug for leukaemia approved
pharmafile | August 30, 2007 | News story | Sales and Marketing |Â Â Â
Atriance, a new treatment for two rare, but deadly, types of blood cancer has been approved in Europe as an orphan drug
GSK's Atriance (nelarabine) will treat patients with T-cell acute lymphoblastic leukaemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) whose disease has not responded to, or has relapsed following, treatment with at least two chemotherapy regimens.
There are only a few hundred patients diagnosed with relapsed/refractory T-ALL/T-LBL each year in Europe and patients with T-ALL and T-LBL tend to have a worse prognosis than patients with B-cell disease.
"The approval of nelarabine for this orphan indication, addresses the real patient need that exists in these rare types of leukaemia and lymphoma," said Andrew Witty, President, European Pharmaceuticals, GlaxoSmithKline.
Professor Dieter Hoelzer, from J.W.Goethe Universitt, Germany hailed the new drug.
"This is a significant and important approval for those patients affected and their specialist physicians across Europe. Nelarabine may offer a valuable chance for patients with few existing treatment options to go on to have potentially curative treatment, such as a stem cell transplant," said Professor Hoelzer.
Many therapies currently used are combinations of cytotoxic drugs which can have a high rate of toxicity. Nelarabine given as a single drug has shown activity and a predictable side-effect profile, and the way it is administered means some patients are able to return home between cycles.
A new approach to development and pricing
Atriance is of particular interest because of the collaboration which made its final development possible, as well as GSK's approach to its pricing.
Witty explained: "Full development for nelarabine began in the 1990s and it has taken over a decade of GSK development and participation in innovative collaborations with well-recognised organisations, such as the National Cancer Institute (NCI) in the US, to provide the data to bring this product to market in Europe."
Witty told the Financial Times earlier this year that GSK was taking a different approach to the cost of the new treatment.
Orphan drugs are often among the most expensive treatments in the world because of the difficulty in developing complex products for small numbers of patients, but Atriance will be more modestly priced than most, at around about $5,000 for a 21-day treatment cycle,
He said: "The economics [for Atriance] are very unlikely to work, but we have never seen this as a commercial opportunity. This is part of our commitment to ensure the right balance between the company and society."
He added that GSK has plans to earn further revenue for the drug by broadening its use into the treatment of other cancer types.
