Roche gets double dose of good Hemlibra news with FDA breakthrough, ICER backing

Roche is relying on hemophilia newcomer Hemlibra to crank out major sales, and the company got two pieces of good news on that front early this week. 

Tuesday, the Swiss drugmaker said the FDA had tagged the drug with its “breakthrough” designation, speeding its trip through the clinic as a therapy for hemophilia A patients without factor VII inhibitors.

Right now, Hemlibra is restricted to a smaller population—those who do have those inhibitors—and a new approval would make its sales potential skyrocket. Consensus estimates including that broader nod sit at $2 billion by 2025, though some analysts, including the team at Jefferies, think the drug could soar much higher. They’re predicting $5 billion that same year.

Industry watchers are so optimistic about Hemlibra’s future, in fact, that shares of hemophilia competitor Shire have been struggling for months. The slide has refocused takeover talk on the company, though it’s still unclear whether Takeda—the only company that has publicly declared buyout interest—has the funds to pull off what would be a $40-billion-plus transaction.

But a recent Hemlibra safety scare could keep Shire patients in the non-inhibitor pool loyal. Late last month, the Basel-based pharma giant confirmed five patients taking Hemlibra had died. None of the deaths was determined to be linked to the drug, a spokesperson said at the time, but the fact that “some experts still do not fully understand the mechanism behind Hemlibra” could “potentially impact use in non-inhibitor patients who are generally well controlled” on Shire drug Advate, Jefferies’ David Steinberg wrote to clients.

RELATED: As hemophilia market heats up, Roche confirms 5 Hemlibra patient deaths

Meanwhile, Hemlibra also snagged a rare prize late Monday: a favorable review at the Institute for Clinical and Economic Review (ICER), a U.S.-based price watchdog that’s stirred the ire of several drugmakers with its cost-lowering recommendations. It issued a final evidence report on Hemlibra, concluding that the new treatment “improves health while lowering costs for certain people with hemophilia A.”

Hemlibra “is an exciting innovation,” ICER’s chief medical officer, David Rind, said in a statement, adding that “it addresses unmet clinical needs in a group of patients with a very high burden of illness, while producing overall cost savings in the health system.”

Roche is relying on Hemlibra, as well as fellow new drugs Ocrevus and Tecentriq, to step up as biosimilar attackers come after older cancer products including Herceptin and Avastin.