Roche showed off additional efficacy and safety data for fast-growing hemophilia A drug Hemlibra. (Roche)
Roche’s Hemlibra has been growing at eye-catching speed. Now, the Swiss drugmaker has padded the drug’s case with a mountain of data, just as its plan to buy into hemophilia gene therapy hit some regulatory road bumps.
In total, Roche presented 21 abstracts from its hemophilia program at the International Society on Thrombosis and Haemostatsis meeting in Melbourne. Data from the four pivotal trials of Hemlibra showcased the antibody drug’s long-term safety and efficacy benefits.
Across the four Haven studies on altogether 400 hemophilia A patients either with or without factor VIII inhibitors, more than 87% of participants didn’t have to be treated for bleeds over a median of 83 weeks, Roche reported Tuesday.
In a retrospective analysis, people on Hemlibra also saw a reduced need for preventative coagulation factor treatment before undergoing minor surgeries, Roche found. Specifically, of the 215 minor operations, 141 (65.6%) were done without factor VIII replacement intervention, and among them, 90.8% of patients didn’t have to undergo additional treatment to remedy post-operative bleed.
What’s more, results from the phase 3b Stasey trial showed that none of the 88 hemophilia A patients with factor VIII inhibitors experienced microvascular thrombosis or thrombotic events, which can be attributed to blood coagulation.
Giving Hemlibra as a preventative therapy also provided “a clinically meaningful improvement in long-term health-related quality of life” versus traditional factor VIII treatment, Roche said. In the Haven 3 and Haven 4 studies, 76% and 79% of patients reported no missed days of work a month before taking Hemlibra. About half a year since treatment, the numbers increased to 91% and 93% of patients, respectively.
“Data presented at ISTH continues to reinforce Hemlibra’s potential to redefine the standard of care for people living with hemophilia A,” Roche chief medical officer Sandra Horning said in a statement.
Hemlibra is growing at Street-beating speed as it extends its reach among factor VIII noninhibitor patients. In the first quarter, sales of the drug jumped to CHF 219 million ($220 million), 62% above consensus. But just as Hemlibra is stealing share from clotting factor replacement therapies, it could itself face competition from newer drugs. So Roche decided to snatch up Spark Therapeutics along with its pipeline of hemophilia gene therapies, including SPK-8011 and SPK-8016 for hem A.
That deal, though, has been delayed several times due to unexpected antitrust scrutiny. After the U.S. Federal Trade Commission sent an unusual “second quest” for additional information, Roche and Spark agreed to push back their closing deadline. Previously, the two could have killed the transaction if it wasn’t done by Jan. 31, 2020, and now, the new deadline is April 30, 2020.
In a message to Spark employees, Roche CEO Severin Schwan reiterated that the two still very much intend to complete the deal this year—which itself marks a postponement from the initial goal of the first half of 2019.
“While we still expect this transaction to close in 2019, we want to ensure that we proactively identify and remove any potential future obstacles to achieving this outcome,” he said. “Good things almost always take time, so as we work to bring this agreement to a close, I ask for your continued patience.”