A US Food and Drug Administration advisory committee has voted to recommend approval of Swiss pharmaceutical major, Novartis' Exjade (deferasirox), a once-daily oral treatment for chronic iron overload associated with multiple blood transfusions.
At present, the only FDA-approved method of removing this excess iron is through subcutaneous infusion of the iron chelator, Desferal (deferoxamine), also sold by Novartis. Using this drug, patients require eight to 12-hour infusions almost every day, so an orally-active alternative is expected to rapidly gain market share. The new drug is administered as a once-daily drink made by dispersing the Exjade tablets in a glass of water or orange juice.
A number of diseases cause patients to require routine blood transfusions, including thalassaemia, sickle cell disease and various other forms of anaemia and myelodysplastic syndromes.
Desferal sales have stayed fairly flat over the last several years, bringing in upwards of $100 million dollars a year for the Swiss firm. But Exjade has much greater potential and should expand the market for iron chelators by extending treatment to patients unwilling to submit to the Desferal regimen, according to Novartis, which said earlier this year that it was expecting peak sales of $650-$750 million for the new drug [[23/06/05d]] [[03/05/05h]].
Analyst notes published after the panel meeting forecast peak sales of the drug between $500 million and $1 billion a year.
The Exjade filings were based on the results of a pivotal clinical trials programme, including a Phase III head-to-head evaluation versus Desferal, which showed that Exjade significantly reduced liver iron concentration at doses of 20-30 mg/kg/day.
Exjade is under priority review with the FDA and has been designated an orphan drug in both the European Union and the USA. Also, it is currently under priority review in Canada, Switzerland, Australia and New Zealand.