Unless you were watching the new streaming releases of Season 6 of Breaking Bad on Netflix (for which I might excuse you) or were abducted by aliens (for which I will not) you must have heard the news….. the first extended half-life factor has been approved for commercial sale. It’s the hemophilia B drug APROLIX. The news was announced at the HFA meeting in Tampa last Saturday, and it truly is the Shot Heard Round the World (an appropriate reference to the American Revolution). This ain’t just a new product, folks; it could shake up the industry. Physicians are watching; patients are watching; Wall Street is watching. What will the “uptake” be?
Uptake is how many patients will switch to using a new product. The hemophilia community tends to be a “wait and see” creature, perhaps due to our devastating experiences of the 1980s. When Advate was launched in 2002 (yes, drugs are launched, like ships), investors predicted a rosy 20% uptake, but initially it was more like 12%. As the risk-takers who switched early reported good results, more and more patients switched. But Aprolix is truly a different kind of drug. Not just a new generation, it is a new type of hemophilia drug: an extended half-life, meaning fewer infusions for the same results.
And Hemophilia B has not had a buffet of drugs to try like hemophilia A. There was only one recombinant (you all know what that means, right?) FIX for years, until October 2013, when Rixubis was approved. With extended half-life factor, prepare for some major shifts in our landscape. Much may depend on the price, which no one is talking about. If it’s too high, will insurance cover it?
And what about individual results? Will each patient respond well? Advate had major hiccups when there appeared to be an increase in inhibitor formation in users. Was this due to higher scrutiny of inhibitors with a new product? The initial scare seemed to be due to increased scrutiny. And these are among the most scrutinized things on the planet.
And lest we get too carried away, don’t forget there are other products in the pipeline by other companies, some extended half-life (the products, not the companies, though we hope they are long lasting too). Basically, the news about Aprolix marks the beginning of a new era in hemophilia. Maybe a Revolution, maybe the Renaissance. A Hemophilia Renaissance.
Below, an article in its entirety about the news….
U.S. FDA approves Biogen’s hemophilia B drug
Inc has won U.S.
approval for its long-acting hemophilia B treatment Alprolix, the U.S. Food and
Drug Administration said on Friday.
Hemophilia B is a rare, inherited disorder in
which a person’s blood does not clot properly, which can lead to prolonged
bleeding and bruising. It affects about 4,000 people in the United States and
Alprolix is a bioengineered version of the blood
coagulation factor IX, a protein needed for normal blood clotting. Biogen is
developing the drug in partnership with Swedish Orphan Biovitrum AB. It
received approval in Canada a week ago.
Hemophilia drugs in general must be infused two
to three times a week to prevent bleeding episodes. Biogen’s drug needs to be
administered once a week or once every 10 to 14 days.
The company is also developing a long-lasting
drug, Eloctate, for Hemophilia A, a more common form of the disease, that
affects about 20,000 people in the United States. Patients with hemophilia A
lack or have reduced levels of coagulation factor VIII.
Current treatments for hemophilia B generate
about $1 billion a year, according to Biogen. The market for hemophilia A
therapies is worth about $6 billion.
Eloctate would compete with Baxter International
Inc’s drug Advate. Baxter is seeking to develop its own long-acting version but
Biogen is expected to be first to the market.
Alprolix is expected to generate sales of $286
million by 2019, according to Thomson Reuters data. Eloctate is expected to
generate sales of $1.1 billion.
(Reporting by Toni Clarke in Washington; Editing
by David Gregorio
) Fri Mar 28,
2014 5:11pm EDT
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