42. Tegsedi

(Akcea Therapeutics)

Active ingredient: inotersen 
Disease: hereditary transthyretin-mediated amyloidosis
Peak sales estimate: N/A 
Approved: Oct. 5 
Companies: Ionis and Akcea 

The scoop: Soon after Alnylam Pharmaceuticals won FDA approval to market its breakthrough RNAi therapy Onpattro for polyneuropathy in hereditary transthyretin-mediated amyloidosis (hATTR) patients, Ionis’ Akcea followed with Tegsedi in that same indication and has priced the RNA drug at the exact same tag: $450,000. Patients can self-administer Tegsedi at the recommended once-weekly dosing and Onpattro must be administered intravenously by a trained medical professional, though at a lower frequency of once every three weeks. However, Tegsedi’s convenience is compromised by a boxed warning. The FDA is asking doctors to check Tegsedi patients’ platelet counts every week and test kidney function every two weeks. The hATTR arena could soon be complicated by Pfizer’s tafamidis, which recently posted positive data in ATTR cardiomyopathy. Though neuro and heart are two different indications within ATTR, analysts noted that both could likely be treated by these same products. — Angus Liu 

42. Tegsedi

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