Orphan Drug Access Still Eludes Some Cystic Fibrosis Patients

More than a year after a breakthrough drug for cystic fibrosis became available, fewer than half of patients who meet the Food and Drug Administration’s criteria can actually access the medication.

A study released at October’s 30th Annual North American Cystic Fibrosis Conference reported that, in 2015, only 42 percent of eligible patients received the new combination therapy, lumacaftor/ivacaftor. The combination drug treats patients who have not just one but two copies of a gene mutation that leads to the rare disease.

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