Roche's pricey new hemophilia drug Hemlibra gets a rare blessing from U.S. cost watchdogs

As the market for hemophilia treatments gets more and more crowded, Roche could use all the help it can get generating enthusiasm among doctors and patients for its new entry, Hemlibra. It just scored a big thumbs-up that will no doubt give the company a lift as it takes on that marketing challenge—and as it faces analysts' questions during its fourth-quarter earnings release on Thursday.

The Institute for Clinical and Economic Review (ICER) issued a report concluding that Hemlibra is cost effective, despite its price of $482,000 for the first year and $448,000 after that. The agency determined that the drug, a weekly injection for reducing bleeding episodes in patients with hemophilia A, would reduce the healthcare budget in the U.S. by $720,000 per patient annually for children under 12, and by $1.85 million a year for people over 12.

To complete its analysis, ICER considered a variety of factors reported during clinical trials comparing Hemlibra to other treatments, including rates of bleeding events, pain and quality of life as reported by patients. In talking with patients and patient groups, ICER learned that the disease restricts education and career choices, recreational activities and more, and that over time, joint injuries from repeated bleeding episodes can require surgeries, according to the report. Hemlibra’s savings come in its potential to relieve all of those complications.

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The ICER review comes just days after Roche won a positive opinion on Hemlibra from the Committee for Medicinal Products for Human Use (CHMP) in Europe, which is recommending the product to prevent bleeding episodes in patients who have developed antibodies against factor VIII. The company got FDA approval for the same indication last November. So now it’s in a full-on fight with Shire for market share in hemophilia A. Shire nabbed EU approval for its factor VIII drug, Adynovi (or Adynovate in the U.S.), earlier this month and a final decision on Roche's product is expected in the near future.

Whether or not the ICER verdict will help Roche meet the sky-high sales estimates for Hemlibra is an open question, however. Jefferies analysts have pegged the drug as a potential $5-billion-a-year blockbuster, on the condition that Roche can get the label expanded to include patients who don’t have antibodies to factor VIII. In November, the company released positive results from a phase 3 trial in patients without factor VIII inhibitors, announcing a significant reduction in treated bleeds over time.

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Roche’s marketing challenge could be exacerbated down the road by next-generation treatments like gene therapy. Spark Therapeutics and Biomarin Pharmaceutical are both moving ahead with their gene therapy treatments for the disease. And hopes for a permanent cure were made clear earlier this week, when Sanofi announced its $11.6 billion purchase of Bioverativ, that Biogen spinoff that’s working on gene therapy treatments for hemophilia A and B.

If Roche can live up to the high expectations for Hemlibra, the drug will certainly help ease the minds of investors, who have been nervous about the company’s patent cliff. Roche’s cancer drugs Avastin, Herceptin and Rituxan are all facing biosimilar threats—a significant challenge, considering the triad once brought in $20 billion a year in sales.

Hemlibra is one of four new drugs that could help Roche fill in the sales hole left by its cancer all-stars, analysts say, but the short-term hit on earnings will be significant. One analyst predicted flat profit margins for the company this year because of the high costs associated with launching Hemlibra and the other new products. Roche CEO Severin Schwan commented recently that the need to pour resources into marketing “puts a lot of pressure on the profitability.” No doubt that will be a big topic of discussion when Roche announces its fourth-quarter earnings later this week.