Genzyme priced Kynamro at just $176,000 annually, still a steep price but much cheaper than Juxtapid’s cost. With hospital budgets under fire across the country, don’t think that such a move won’t matter to physicians (along with cost-wary patients), even with Kynamro’s significant side effect problem. Genzyme and Isis’s drug will have to carry a warning on its box regarding a serious risk of liver toxicity, and unlike Juxtapid’s pill form, Kynamro is a less-preferable injection.
It should be an entertaining battle between the two drugs in the near future. I’d give the lead ever so slightly to Kynamro in an era where cost and dollars matter considerably, especially with Sanofi’s massive apparatus backing it up. While Aegerion will have to spend more pushing its drug and competing with Kynamro for market share, Juxtapid’s effectiveness in clinical trials and lack of Kynamro’s dubious safety profile should give it some momentum.
Investors would be wise to temper their expectations in the short term with competition afoot, however.
BioMarin on the hunt for approval
Unlike Aegerion and NPS, biotech firm BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) already boasts of drugs on the market. The company’s orphan drug BMN-110 — formerly known as GALNS — is making things interesting after a successful phase 3 clinical trial, however.
BioMarin designed BMN-110 to treat Morquio-A syndrome, also known as MPSIVA, which affects around 1 out of every 200,000 people in the U.S., for a total domestic patient population of around 1,500. BMN-110 hit its primary endpoint in its phase 3 trial by improving results of patients in a six-minute walk test over a placebo. It may not sound glamorous, but the success was enough to convince many that BMN-110 is on its way to success.
BMN-110’s safety record is strong, and BioMarin expects to submit the drug for FDA approval in the first quarter of 2013. Keep an eye on the approval process, which some experts expect to deliver the thumbs-up in the U.S. by the last quarter of the year. BioMarin expects to price BMN-110 at more than 250,000 per year, with analysts projecting $500 million or more in potential peak sales.
Who’s ready to step up?
Which drug among these four will soar the furthest in 2013? BMN-110 isn’t out yet and still awaits regulatory approval, so hold off on that one until the FDA gives the go-ahead. With the HoFH market a competitive one, however, look for NPS’s Gattex to get things going this year. The drug will take time to warm up — and don’t expect massive sales so soon after approval — but Gattex looks like a long-term winner.
And long-term investing, as smart investors know, is the best therapy around.
The article 4 Orphan Drugs Making Waves in 2013 originally appeared on Fool.com and is written by Dan Carroll.
Fool contributor Dan Carroll has no position in any stocks mentioned. The Motley Fool recommends BioMarin Pharmaceutical (NASDAQ:BMRN).
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