- Results from Roche's HAVEN 3 Phase 3 study of newly approved Hemlibra in patients with hemophilia A without inhibitors to Factor VIII showed that the once-weekly treatment had both statistically significant and clinically meaningful reduction in the number of treated bleeds compared with patients on no prophylaxis.
- Hemlibra also showed a statistically significant reduction in the number of treated bleeds when it was dosed every two weeks compared to no prophylaxis. Notably, the drug showed superiority to Factor VIII prophylaxis.
- There were also no new safety signals spotted. Data from HAVEN 3 will be submitted to regulatory authorities.
Editor's Note: This story was updated to include a statement from Roche regarding litigation with Shire.
Roche's Hemlibra (emicizumab) snagged early approval from the Food and Drug Administration last week for adult and pediatric patients with hemophilia A who have developed Factor VIII inhibitors, supported by data from the HAVEN 1 and HAVEN 2 studies.
Approval is pending in Europe in that patient population as well, under the European Medicines Agency's accelerated assessment process.
"[The HAVEN 3] results in people with hemophilia A without inhibitors represent the next step forward in our clinical trial program," said Sandra Horning, Roche's CMO and head of global product development. "We look forward to working with health authorities to make this treatment available for all people with hemophilia A as soon as possible."
As a once-weekly subcutaneous drug that has potential to reduce the frequency of bleeds, Hemlibra's combination of convenience and effectiveness, along with data in patients both with and without inhibitors to Factor VIII, could have a major impact on the hemophilia market, especially as, according to the company, it is "the first product to show superior efficacy to Factor VIII prophylaxis."
"We understand that the Hemlibra price will remain unchanged once it launches in the non-inhibitor patients and currently model $5 billion in peak de-risked sales across all indications for Hemlibra, assuming a protracted launch trajectory due to multiple clinical trial read outs needed to build out the label as well as conservative adoption of this therapy," wrote Jefferies analyst Jeffrey Holford in a note to clients.
Other companies are also working to disrupt the current hemophilia market, though they are well behind Roche. This included Alnylam Pharmaceutical Inc.'s fitusiran, which also cuts the bleed rate. However, clinical trials of this are currently on hold after a patient death in an open-label extension of a mid-stage study.
Shire plc is fighting back against Hemlibra, which has potential to knock a hole in its hemophilia market. The Irish pharma claims Roche is disparaging its Feiba prophylaxis agent, and has filed an injunction. The claims accuse Roche of "inaccurate and misleading characterization of the serious adverse events that occurred in the HAVEN 1 Phase 3 trial of emicizumab" and spreading misinformation about "the appropriate management of breakthrough bleeds uncontrolled by emicizumab."
"An oral hearing of the Court of Hamburg occurred on September 14, 2017. The court reviewed the evidence presented by both parties and concluded that Roche had not engaged in any inappropriate promotional activities related to the HAVEN 1 abstract. Following initial comments from the judges, Shire chose to withdraw their application. As a result, the case is formally closed and Shire will incur all court and legal fees for Roche in relation to these proceedings," said a Roche representative in a statement to BioPharma Dive.