FDA Advisory Committee recommends new orphan anti-infective drug

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Yesterday’s FDA Anti-Infective Drug Advisory Committee meeting considered evidence about the safety and effectiveness of ISA, a new drug to treat a rare fungal infection threatening the lives of people with severe illnesses. The condition affects a few thousand Americans each year suffering from weakened immune systems due to conditions such as HIV, stem cell transplants, or cancer. Without treatment virtually all these patients would die. We heard very compelling public testimony from an affected patient, a doctor calling for more treatment options, and the father of a young boy who died from a fungal infection, not from his cancer. There are few other drug options to fight these infections and they are very imperfect. Because these conditions are rare, there is not a lot of clinical data about the effectiveness of the drug but it is promising. The committee voted to recommend that the FDA approve the medication for these uses, but with warnings about use in children, pregnant and nursing mothers. The committee emphasized the need for more studies including more information on an interesting ethnic variation in the drug’s physiology.