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Endpoints article on Givosiran approvalUPDATED: Make that 2 approved RNAi drugs at Alnylam after the FDA offers a speedy OK on ultra-rare disease drugSeventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed. The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier. AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari. advertisementadvertisement John Maraganore Alnylam “We’re a little bit not as prepared as if February 4 was going to be the date,” Alnylam CEO John Maraganore tells me. “But that’s OK, we’re ready to process the start form as we speak and ship drugs shortly after.” The OK was hardly a surprise, but the early timing helped burnish Alnylam’s prospects, sending shares up 10.5% and leaving Alnylam with a market cap right at $13 billion. For a company and a technology that was being shunned by several Big Pharmas 8 years ago, a rising market cap on new approvals is sweet. For a company still fairly new to FDA approvals, the federal sanction was a textbook case on gaining a timely approval, charting a course with fast-path designations, getting a pass on a panel review and marching straight to the approval announcement. The only hiccup came as some analysts questioned the safety data toward the end, which highlighted some key concerns. But as Maraganore said, the benefits here — with a 74% reduction in attack rates — clearly outweighed the real risk involved, making this an easy one for regulators. In addition, Maraganore says Alnylam got a broad label without a black box warning. The next step at Alnylam is to build up the commercial wing a bit more after getting started with Onpattro (patisiran). They have a deal with Ironwood to cover the GI specialists involved, but Maraganore says that Alnylam is in the countries it needs to be as it gets started with drug number 2. The price? $575,000 per patient for the wholesale rate. Maraganore expects the final amount will net out at $442,000. That’s more expensive than Onpattro, says the CEO, but “that’s very much in line with ultra-rare” diseases. Alnylam is already working on deals with payers on value-based agreements, offering rebates when the drug doesn’t work as expected. And Maraganore says he’s also working on what he believes is a first in the industry: A prevalence-based rebate deal, so if the prevalence of the disease in their populations rises with better diagnoses, they’ll build more rebates in. That’s not likely to immunize the company from the growing chorus of critics who have focused in on high drug prices — most notably Democratic presidential candidate Elizabeth Warren, who just days ago offered a plan aimed at using compulsory licensing to break control of companies’ IP when they charge large amounts for drugs to treat rare disease. “I think compulsory licensing is the biggest political gimmick in the world,” Maraganore commented. Say you take the IP from the developer and give it to a generic drug company. They still have to make the drug, test it and get an FDA approval. But like drug importation, he adds, “it’s just a gimmick.” If all works out according to plan, they may not have long to wait for drug number 3. The company is on track to report pivotal data on lumasiran in a matter of weeks, setting up a new drug application in early 2020. And then there’s inclisiran, the RNAi drug for PCSK9 they licensed out to The Medicines Company, which is also being tapped as a likely near term approval. In many ways, Alnylam represents a new breed of biotech. They kept most of their global rights for the main drugs in the pipeline, intending all along to make the journey into commercialization. And now that they’ve begun recording revenue, the pressure is starting to mount on Maraganore on when he expects to turn the corner to profitability. The CEO wasn’t ready to offer a year to me, but he is making a promise on one thing: “2019 is peak loss year,” he says. “Now every year, barring an upset, is meant to be a better and better year toward profitability.” AUTHOR |
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