There was relief for Ionis and Akcea late on Friday with US approval for the groups’ amyloidosis therapy Tegsedi – particularly after the knockback for another of their antisense projects, Waylivra, in August. But a black box warning for thrombocytopenia and renal toxicity will not help Tegsedi compete against Alnylam’s Onpattro, which avoided these safety concerns when it got the FDA nod for the same indication, polyneuropathy of hereditary transthyretin-mediated amyloidosis. Tegsedi is given subcutaneously versus Onpattro’s intravenous delivery, but this advantage could be outweighed by the need for monitoring with Tegsedi. Ionis and Akcea might have missed a trick by setting Tegsedi’s list price at $450,000 per year, the same as Onpattro’s, especially after an Icer report concluded last week that both products were too expensive. Icer recommended annual prices of $25,000 for Tegsedi and $46,000 for Onpattro to hit $150,000 per quality-adjusted life year. Stifel believes that the Ionis/Akcea drug will capture 20-25% of the market, although EvaluatePharma sellside consensus is more optimistic. Forecasts for Onpattro have come off since its approval, which did not include cardiomyopathy because of safety concerns, leaving the way clear for Pfizer's tafamidis in this subset of amyloidosis patients.
|The amyloidosis landscape|
|Annual sales ($m)|
|Tafamidis*||Pfizer||Marketed in Europe; US filing due H2 2018||22||134||577||1,074|
|*Marketed in Europe for polyneuropathy but sales forecasts mostly reflect expected demand in separate cardiomyopathy indication. Source: EvaluatePharma.|