FDA approval of Biogen Idec's long-acting hemophilia A product upped the ante in the space, as Eloctate (antihemophilic factor [recombinant], Fc fusion protein) became the first recombinant hemophilia A therapy with prolonged circulation in the body and the only treatment indicated to reduce the frequency of bleeding episodes in the indication by offering prophylactic infusions every three to five days.

Approval represented the first major advance in the treatment of hemophilia A in more than two decades, according to Joe Ciaffoni, Biogen's senior vice president of U.S. commercial.

"Historically, these patients have been confronted with a choice either not to treat prophylactically or, if they did choose to treat prophylactically, to treat three or more times per week," Ciaffoni said, calling the extended interval of three to five days "a meaningful improvement" with the potential to transform the standard of care.

Approval adds heft to the Cambridge, Mass.-based company's product portfolio in blood disorders. Earlier this spring, the FDA green-lighted Alprolix (coagulation factor IX [recombinant], Fc fusion protein) as the first recombinant, DNA-derived hemophilia B therapy with prolonged circulation.

In describing the real-world impact of hemophilia agents with prolonged circulation, Ciaffoni related the case study of a 4-year-old boy with only one productive vein who participated in the Alprolix trial and now uses the therapy. For previous prophylactic treatments, the boy's mother commuted more than two hours each way three times a week to the hemophilia treatment center, spending more than $100 per trip out-of-pocket, so her son could be infused. Introduction of Alprolix reduced that burden significantly, and Biogen hopes Eloctate will produce similar improvements in quality of life for patients, he said.

"When you hear about the difference these therapies can make, it's moving," Ciaffoni told BioWorld Today.

The FDA's nod for the therapy, designed to control and prevent bleeding episodes, perioperative management and routine prophylaxis in adults and children, came late Friday, following the market's close. On Monday, Biogen's shares (NASDAQ:BIIB) inched higher early before sliding late in the day to close at $318.16, up 61 cents.

Biogen plans to disclose pricing information prior to Eloctate's launch next month, but Ciaffoni said pricing will be consistent with the annual treatment cost for current hemophilia A therapies taken prophylactically. The company also plans to offer personalized assistance programs.

UPSIDE ALSO COULD DRIVE SHARES OF PARTNER SOBI

Eloctate, 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 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.

No participants in A-LONG developed inhibitors to Eloctate, and there were no reports of serious vascular clots or serious allergic reactions.

The pediatric indication was supported by interim safety and pharmacokinetic data in 38 boys ages 2 to 11 from the phase III Kids A-LONG study, showing that Eloctate was generally well tolerated and no inhibitors were detected.

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.

Analysts said the Eloctate label was generally in line with the agent's previously published clinical data and profile.

In a first glance, RBC Capital Markets LLC analyst Michael Yee called the approval "another incremental driver" for Biogen, representing a product launch with upside potential for the company. 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, Yee wrote.

The $5.2 billion market for hemophilia A therapies could reach $7 billion by 2016, representing a 6 percent average growth rate, according to a market analysis conducted last year by Morningstar.

In a flash comment, Wells Fargo Securities LLC analyst Brian Abrahams pointed out that hemophilia A represents a significant market opportunity, affecting 16,000 patients in the U.S. and 142,000 globally. On that basis, Abrahams modeled 2020 combined sales of approximately $650 million for Eloctate in the U.S. and European Union (EU), representing a revenue contribution of approximately 4 percent of the company's total sales. Although Biogen's total hemophilia sales could reach $127 million this year, Abrahams noted that several competitors, including Bayer AG and Baxter International Inc., are looking to enter the market.

In April, Baxter snagged the assets of development partner Chatham Therapeutics LLC in a $70 million deal plus potential development, regulatory and commercial milestone payments. That acquisition gave Baxter full access to Chatham's gene therapy platform, including a hemophilia B program that was part of a 2012 collaboration between the companies to evaluate Baxter's BAX 335, a preclinical hemophilia A program and potential future application to additional hemophilia treatments. (See BioWorld Today, April 3, 2014.)

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.

Jefferies Group LLC analyst Eun Yang wrote in a Sobi note that the Eloctate launch solidified the drug's first-to-market advantage and likely monopoly in the long-acting hemophilia A category for up to two years. Though Yang did not comment on takeover prospects, she observed that "successful U.S. launch would be a positive indicator of EU sales opportunity, likely driving SOBI shares higher."

On Monday, Sobi (STOCKHOLM:SOBI) gained SEK 1.90 (U.S. 28 cents) to close at SEK 84.90 (US$12.69).

FDA approval is the first for Eloctate, which remains under review by regulators in several other countries, including Canada, Australia and Japan.