Roche Enters Tight Hemophilia Market

 Swiss drug manufacturer Roche is attempting to break into hemophilia A treatment, a $10 billion global market that is dominated by rivals who have cultivated close ties to sufferers of the genetic breeding disorder.

Roche’s Hemlibra, which has been on sale since 2017 for patients with resistance to existing treatments, is generally expected to win the US Food and Drug Administration’s approval for use in almost all patients this week. The FDA declined to issue a comment.

First-half sales were just 57 million Swiss francs, or $58 million.

To reach $5 billion global annual sales that some analysts predict, Hemlibra must wrench business away from giant rivals such as Shire, Bayer, Novo Nordisk, and Octapharma, whose clotting factors are now standard for people who lack proteins that stop bleeding.

These companies have long been sponsoring patient groups, summer camps, even kayak trips on the Pacific Ocean, as parts of the efforts to build the loyalty they hope will slow down an exodus from their products once new drugs like Roche’s arrive.

Roche is convinced that word of mouth in the hemophilia community, supported by its studies that show that Hemlibra shots may be more effective and must be administered less frequently than infusions of clotting factors, will overcome any prevailing allegiances.

“I’m not familiar with what the other companies are telling the patients,” said Dr. Gallia Levy, who leads Roche’s Hemlibra program. “What I do know is that I’ve seen a lot of patients telling other patients what is happening in their lives. Those sorts of stories mean the most to the patients.”

As part of Roche’s initial marketing campaign for Hemlibra, it has produced videos of hemophilia patients taking part in daily activities, including riding bikes, despite of their serious illness.

One video shows the consequences of failing to get appropriate treatment, featuring a Serbian man who has suffered joint bleedings as a child and now must use crutches.

Hemophilia A has become one of medicine’s most lucrative diseases, in spite of just 20,000 patients in the United States and several hundred thousand worldwide. It almost always affects men, passed down by a mutation on the X chromosome from their mother.

In 2016, the US government alone whipped out some $2 billion on hemophilia A therapies, data from its Medicaid and Medicare insurance programs show.

Shire’s Adynovate clotting factor runs at $537,000 annually, although the expenses for severe patients can reach several million dollars.

Roche has priced Hemlibra at $482,000, a lofty amount but a level that the independent US pricing group ICER has said could slash overall hemophilia treatment costs significantly.

Largely a cancer drugs maker, Roche is turning to rare diseases like hemophilia to replace revenue from its older medicine whose expiring patents have exposed the company to unprecedented generic competition.

Hemophilia represents the largest market for rare diseases and is expected to grow above 7 percent each year through 2022, as forecasted by Evaluate Pharma.