Roche’s fast-moving Hemlibra breaks into wider hemophilia market with blockbuster approval

Roche Hemlibra
Roche's Hemlibra is now approved to treat all hemophilia A patients. (Roche)

Roche’s Hemlibra officially has the hemophilia indication it’s been waiting for—and that its competitors have been dreading.

On Thursday, the FDA approved the drug to treat hemophilia A patients without factor VIII inhibitors, putting a significantly larger group of patients in line for Hemlibra therapy. Previously, the drug was cleared only in patients with inhibitors, which develop as patients use factor VIII replacement drugs to prevent bleeding.

RELATED: Roche's Hemlibra, eyeing a big new market, puts up standard-busting bleed data

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Now that the drug has a green light in all U.S. hemophilia A patients—who number about 18,300, according to estimates provided by Roche—analysts expect its sales to soar. Industry watchers predict Hemlibra could rack up $2 billion by 2025, though some analysts have said that number could climb far higher. Jefferies analysts, for their part, have pegged 2025 sales at $5 billion.

One reason? Efficacy. In a phase 3 study in hemophilia A patients without factor VIII inhibitors, Hemlibra reduced bleeding incidents that needed treatment by 96% compared with no preventive therapy, Roche said in May. Hemlibra also boasts a convenience advantage: It can be administered subcutaneously every week, two weeks or four weeks.

RELATED: New Roche threat to Shire's hemophilia franchise? CEO Ornskov says he's not worried

But Hemlibra’s prospects don’t bode well for Shire, which entered the hemophilia sphere with its $32 billion Baxalta deal back in 2016. The company’s shares have been taking a beating throughout Hemlibra’s rise, and Thursday’s new approval sent them south once again.

Investors concerned about Shire’s ability to keep up with Roche in the hemophilia field have also hounded Takeda, which inked a $62 billion buyout agreement for Shire back in May. But the Japanese drugmaker’s executives have worked to reassure investors that the company has already factored that next-generation competition into its financial forecasts.