FDA approves three new drugs for the market, first monoclonal antibody tested for efficacy solely in animal models

December 18, 2012 | By Márcio Barra

FDA just gave the green light on three new drugs last Friday, December 15th, all of them of orphan status.
The first is the new leukemia drug Ponatinib, from Aria Pharmaceuticals. Iclusig™ (ponatinib) is a BCR-ABL inhibitor, an abnormal tyrosine kinase expressed in chronic myeloid leukemia and Philadelphia-chromosome positive acute lymphoblastic leukemia. This drug was specifically designed, through computational methods, to inhibit this enzyme activity.
The second is Signifor, from Novartis. It is the first approved therapy in the U.S. for Cushing’s disease, a rare condition where an individual produces and overproduction of Cortisol. Signifor, a multireceptor-targeted somatostatin analogue, binds and activates 4 of the 5 somatostain, inhibiting secretion of adrenocorticotropic hormone. It was approved for use in Europe back in April, by EMA.
The third is Raxicumab, from GlaxoSmithKline, a monoclonal antibody developed by Human Genome Sciences, who was bought by GSK back in July 16, 2012, for $3.6 billion. Raxicumab is intended to treat patients poisoned with anthrax, a lethal disease caused by the bacterium Bacillus anthracis. It acts through a novel mechanism which blocks the bacterium toxin.
Raxicumab approval is the first time that the FDA approves a monoclonal antibody for the market without any efficacy studies conducted in humans. Due to ethical concerns (as Bacillus anthracis is a lethal bacteria), only safety studies were conducted in healthy volunteers, with efficacy studiesconducted in animal models.



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