A PATIENT PRESCRIBED the drug eculizumab could expect to pay around €300,000 a year for treatment. In 2010 Forbes revealed it to be the world’s single most expensive drug. However, the company which makes it, Alexion, is now waiving the fee for the chance to try it out on some of the victims of the deadly E.coli outbreak in Germany, which so far has claimed ten lives.
The drug, also known as Soliris, is already used around the world as the only treatment for the rare, debilitating and life-threatening blood disorder PNH. However, its manufacturer wants to market it for the treatment of another disorder called atypical Hemolytic Uremic Syndrome (aHUS) – which can cause kidney failure, neurological complications and death – in the US and in Europe, and submitted an application to do so in April.
Some of those affected by the E.coli outbreak in Germany have developed aHUS, reports Nature.com, 300 of them in fact. Now a hospital in Hamburg is administering eculizumab, to patients presenting with uHUS. Nature.com says that Alexion is providing the hugely expensive drug free of charge, in order to prove that it can work, and to speed up gaining approval to sell it to treat aHUS. Research published recently by doctors in Germany has indicated a dramatic improvement in three young patients treated with the drug, after an initally “devastating” prognosis. Nephrologist Franz Shaefer told Science Magazine:
HUS cases with severe neurological symptoms are very rare, but now they are everywhere. A lot of [colleagues in other hospitals] have asked about the new treatment, and a few patients are already being treated with it.
The drug’s massive cost hit the headlines in Scotland recently, after a 68-year-old man died from PNH after being refused access to eculizamub. A PNH suffered in Canada meanwhile says Alexion is currently providing him with the medicine free of charge “on a compassionate basis”, but that he fears their generosity may run out, leaving him to foot the $500,000 bill.
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