FDA approves Akcea rare disease drug with a black box warning

FDA has approved inotersen (Tegsedi—Akcea Therapeutics) with a black box warning. The drug is indicated to treat the nerve pain, not heart damage, associated with the rare and fatal condition hereditary transthyretin amyloidosis (hATTR).

FDA has approved inotersen (Tegsedi—Akcea Therapeutics) with a black box warning. The drug is indicated to treat the nerve pain, not heart damage, associated with the rare and fatal condition hereditary transthyretin amyloidosis (hATTR). The approval is a win for Akcea after FDA declined to approve another one of the company’s drugs based on the same technology. Inotersen is the second drug on the U.S. market for hATTR. In August, patisiran sodium (Onpattro—Alnylam) received FDA approval for hATTR. Patisiran sodium requires an I.V. infusion every 3 weeks, while patients can inject themselves with inotersen once a week. However, FDA requires patients using inotersen to have platelet monitoring at least weekly, as well as biweekly kidney tests and liver tests to be done 3 times a year. The drug has been available in Europe since July and was approved in Canada just last week. Akcea is ready to launch in the U.S. immediately, according to company CEO Paula Soteropoulos. "We have a full sales team that’s been on the ground already connecting with the physicians, helping to find the patients, nursing case management team is on the ground, and market access team is working with payers," she added.