FDA approves AstraZeneca, Ionis treatment for rare nerve disease
The drug is a monthly injection for a genetic disorder called ATTR-PN, which affects about 30,000 people around the world and can be fatal if left untreated.
The Food and Drug Administration approved a novel medicine for a rare and devastating nerve disease Thursday, clearing a treatment that promises to be more convenient than available therapies.
The drug, from partners AstraZeneca and Ionis Pharmaceuticals, is a monthly injection for a genetic disorder called ATTR-PN, in which a buildup of toxic bodily proteins progressively damages patients’ peripheral nerves. The disease affects about 30,000 people around the world and can be fatal if left untreated.
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