STAT+: 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.
Called Wainua, the treatment is the fourth approved medicine for ATTR-PN. Two, sold by Alnylam Pharmaceuticals, must be administered by a health care professional. A third, invented by Ionis, can be injected at home but requires weekly dosing. Wainua is comparably effective but less burdensome, said Mina Makar, who leads AstraZeneca’s cardiovascular, renal, and metabolic disease division. The companies did not disclose Wainua’s list price. Alnylam’s medicines cost upward of $400,000 a year.
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