The first medicine that uses RNA interference, a method cells employ to mute a gene before it can make a harmful protein, has made its way to Europe.
Three weeks after the FDA approved patisiran (Onpattro), from Alnylam Pharmaceuticeuticals (NASDAQ: [[ticker:ALNY]]), for a rare and deadly disease called hereditary transthyretin amyloidosis (hATTR), the European Medicines Agency has followed suit. The EMA specifically approved patisiran for adults with hATTR and stage 1 or stage 2 polyneuropathy, the nerve damage often suffered by hATTR patients. Alnylam officials have estimated that 20,000 to 30,000 patients worldwide have hATTR and the nerve damage.
Patisiran is the first marketed RNAi medicine, the culmination of two decades of scientific work. But its commercial prospects are unclear. The market battle for hATTR—a disease that, up until recently had no approved treatments, just a generic medicine used off-label—is about to heat up. European regulators approved inotersen, a rival hATTR drug from Akcea Therapeutics (NASDAQ: [[ticker:AKCA]]), in July and the FDA could follow by early October. And Pfizer’s (NYSE: [[ticker:PFE]]) hATTR drug tafamidis (Vyndaqel) has been approved in Europe since 2011. The company plans to file for FDA approval of tafamidis—specifically for hATTR patients with heart damage—based on data it released at a conference earlier this week.
Nonetheless, Alnlyam’s stock has jumped more than 20 percent since Pfizer released the tafamidis data. In a research note, Jefferies analyst Maury Raycroft wrote that there were “some weaknesses” with Pfizer’s results that “ultimately should enhance [patisiran’s] commercial opportunity.” Namely, the benefit of tafamidis on hATTR patients isn’t clear, so tafamidis’ commercial impact on patisiran and inotersen could be limited.
Still, it remains to be seen whether insurers will cover the cost of patisiran. The EMA ruling comes just a day after the Institute for Clinical and Economic Review, a nonprofit drug pricing watchdog, issued an updated report on patisiran and inotersen and criticized the price Alnylam has set. Patisiran’s average net price, taking rebates from insurers into account, is $345,000 per patient, per year. Alnylam has agreed in principle to the structure and key terms of “value-based agreements” with certain insurers, including Harvard Pilgrim Health Care, that tie its drug’s price to the benefit it provides patients, but hasn’t provided many details on the discounts. ICER, whose influence has grown in recent years, believes patisiran’s price should be cut 90 to 95 percent. “The announced price of patisiran, even taking into account expected discounts, far exceeds commonly cited cost-effectiveness thresholds,” ICER chief scientific officer Dan Ollendorf said in a statement.
As Xconomy reported earlier this month, ICER has questioned how the data Alnylam has accrued so far translate to a higher-quality life for patients. Alnylam has countered that ICER’s review is “premature” and “must be viewed in balance with the urgent need and the potential benefits that new treatments for hATTR amyloidosis may offer to patients and their families.”
Here’s more on patisiran, the treatment landscape for hATTR, and RNAi.
