When Biogen announced the price on its new rare disease drug Spinraza—$750,000 the first year and $375,000 after that—critics popped up immediately. Biogen and its partner Ionis saw their shares drop.
Biogen tried to explain. It had “carefully considered” the price, spokesman Matt Fearer told CBS News, and in the context of its clinical value—and Biogen’s need to fund other R&D—Spinraza’s price “is fairly in line with other therapies for rare orphan diseases,” Fearer said.
Since then, despite the complaints, analysts have raised their expectations for the drug, based on conversations with doctors and payers. But the pricing questions linger, if only in the context of the public debate—and political pushback—pharma’s been facing on its price-setting and price hikes for more than a year now.
And Fearer was right in the sense that other rare disease meds are as costly or even more so. Nowhere is this more apparent than in a recent accounting of the world’s most expensive drugs, compiled by Reinsurance Group of America.
The ranking is based on wholesale acquisition cost drawn from proprietary databases, scholarly publications and online research, RGA says, and uses wholesale acquisition cost as its benchmark. That’s not net price, of course; it’s without rebates and discounts. But Spinraza’s list price doesn’t include those incentives for payers either, and neither do the rest of the panoply of public U.S. price tags on rare disease meds.
To those who follow such rankings, this one includes some familiar faces farther down the list—Alexion’s Soliris, for instance, in sixth place at $542,640—and some less-familiar brands as well. Here goes:
Glybera, $1.21 million per year
This UniQure treatment for lipoprotein lipase deficiency is not yet approved in the U.S., but its price tag in Europe tops $1 million. It’s a gene therapy, the first to win a regulatory nod, and on the leading edge of a potential wave of therapies of that ilk, many costing upwards of $1 million or more.
Payers and other experts, worried about their advent, have been tossing around potential reimbursement schemes and pricing mechanisms that would extend payments over time, peg them to performance and even securitize them. Drugmakers have offered their own plans. Payment questions—and the small number of patients eligible—have had Glybera struggling so far to win customers.
This treatment for urea cycle disorders (UCDs) was the centerpiece of Horizon Pharma’s $1.1 billion buyout of Hyperion back in 2015. At the time, Hyperion boasted some promising numbers for Ravicti and another UCD therapy, Buphenyl, with Ravicti bringing in $95.4 million in net 2014 sales.
By 2016, the drug was bringing in $151 million, way up from its 2015 total of about $87 million. Horizon has paired up with Swedish Orphan Biovitrium (SOBI) to launch the med commercially in Europe this year, and it’s working on new indications for the med as well. Its FDA orphan drug exclusivity expires in 2020.
The therapy is used to treat Pompe disease, an enzyme deficiency that affects an estimated one in 40,000 live births each year, or 5,000 to 10,000 people worldwide. Sanofi acquired the med and its sister Pompe treatment, Myozyme, in its 2011 Genzyme buyout. Sanofi reports its Pompe disease sales as a franchise—they amounted to 725 million euros last year worldwide—but Myozyme isn’t available in the U.S. anymore. Stateside sales in Pompe disease were 240 million euros, or $257 million at today’s exchange rates.
An oral treatment for hyperammonemia in patients with a NAGS deficiency, Carbaglu (carglumic acid) boasts an indication that Horizon has tried, and so far failed, to win for Ravicti. Sold by the rare diseases unit at Recordati, the Italy-based pharma, Carbaglu was approved in 2010 in the U.S. and won an orphan drug designation in 2014 for treatment of organic acidemias. Recordati also is developing an IV formulation for patients who can’t use the oral version. EvaluatePharma estimates (reg. req.) its 2016 worldwide sales at $48 million.
This treatment for malignant osteoporosis and chronic granulomatosis, first approved in 2000, has changed hands several times since, and according to market-watchers, that’s one reason for its $500,000-plus price. Backed by private equity, the specialty drugmaker, Vidara Therapeutics, bought the drug from InterMune in 2012 for $55 million. After that deal, according to Bloomberg, its price climbed by 434% in two years.
Horizon Pharma then grabbed it for $660 million in 2014. Sales of the med grew to $104 million in 2016, down slightly from 2015’s $107 million. In December, the drug failed a trial in Friedreich’s ataxia—a new indication Horizon had hoped to snare. Actimmune has also had a storied history for other reasons:InterMune CEO Scott Harkonen touted in a misleading press release more than a decade ago, a move that earned him 6 months of house arrest and three years of probation.
Rounding out the top 10 are Soliris; Alprolix, a Biogen hemophilia B drug that runs $503,880 annually; Indelvion, another hemophilia B med, this one from CSL Behring, with a $500,000 tag; BioMarin’s Naglzayme, which treats mucopolysaccharidosis Type IV A, at $485,747; and Folotyn, for peripheral T-cell lymphoma, sold by Spectrum Pharmaceuticals for $450,540 per year.
By Tracy Staton
Source: Fierce Pharma
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