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Another win for Biogen as FDA approves hemophilia A therapy Eloctate

By Marie Powers, Staff Writer

Late Friday, the FDA approved Biogen Idec’s hemophilia A product Eloctate (antihemophilic factor [recombinant], Fc fusion protein) to control and prevent bleeding episodes, perioperative management and routine prophylaxis in adults and children. Eloctate becomes the first recombinant hemophilia A therapy with prolonged circulation in the body and the only treatment indicated to reduce the frequency of bleeding episodes for hemophilia A with prophylactic infusions every three to five days.

Approval came just months after the FDA green-lighted Biogen’s Alprolix (coagulation factor IX [recombinant], Fc fusion protein) as the first recombinant, DNA-derived hemophilia B therapy with prolonged circulation, adding heft to the Cambridge, Mass.-based company’s product portfolio for blood disorders. (See BioWorld Today, April 3, 2014.)

In a statement, George A. Scangos, the company’s CEO, called Eloctate “the first significant hemophilia A treatment advance in more than 20 years.”

The drug, like Alprolix, was developed with partner Swedish Orphan Biovitrum (Sobi), of Stockholm. The companies reported findings from the phase III A-LONG trial in July 2013. In that study, hemostasis assays showed that, despite differences between patients in thrombin generation, Eloctate and Advate – the standard of care from Deerfield, Ill.-based Baxter Healthcare Corp. – showed equivalent thrombin generation potential post-infusion. (See BioWorld Today, July 9, 2013.)

The open-label, multicenter A-LONG study examined the efficacy, safety and pharmacokinetics of Eloctate in 165 previously treated males 12 years and older with severe hemophilia A. Patients achieved statistically significant reduction in bleeding episodes in both of the study’s prophylaxis arms, relative to the on-demand treatment arm. In addition, 98 percent of bleeding episodes were controlled with one or two Eloctate infusions.

In a first glance, RBC Capital Markets LLC analyst Michael Yee called the approval “another incremental driver,” representing another product launch with upside potential for Biogen. Although Biogen’s hemophilia program is “not really a huge part of the thesis for most to own the stock,” sales could exceed expectations, with “lots of open space and upside to models” in the global market, he wrote.

Yee also speculated that, if the launch goes well – “and considering BIIB and other market observers think that long-acting products could be at least half the market in the future” – Biogen could move to acquire Sobi. For now, Biogen owns roughly half the franchise, he added.

The recommended starting prophylactic regimen for Eloctate is 50 IU/kg every four days. Based on clinical response, the regimen may be adjusted in the range of 25 to 65 IU/kg and every three to five days.

Hemophilia A affects approximately 16,000 adults and children in the U.S. The $5.2 billion market for hemophilia A therapies could reach $7 billion by 2016, representing a 6 percent average growth rate, according to an analysis of the hemophilia market conducted last year by Morningstar.

Biogen’s stock (NASDAQ:BIIB) closed Friday at $317.55 per share, but in after-hours trading it edged up $1.85.

See the next edition of BioWorld Today for more on this story.