Shire International GmbH has obtained an injunction in a Hamburg court in Germany against F. Hoffman-La Roche (Roche) to address incomplete and misleading claims that may impact important patient safety issues regarding emicizumab, the investigational monoclonal antibody for the treatment of hemophilia A.
Emicizumab is being developed by Chugai Pharmaceutical Co. (Chugai), a subsidiary drug manufacturer operating in Japan. Roche owns 62% of Chugai.
A Phase I human trial found that emicizumab was well tolerated by healthy subjects. The drug binds to both the activated coagulation factor IX and to factor X, mediating the activation of factor X, which is normally the function of coagulation factor VIII. Patients with hemophilia A lack coagulation factor VIII.
Emicizumab side effects
Under the injunction, Shire PLC, a Jersey-registered, Irish-headquartered biopharmaceutical multinational giant, seeks to prevent further dissemination of the alleged inaccurate and misleading characterization of the serious side effects that occurred in the HAVEN 1 Phase III trials of emicizumab, specifically the assertion that “all events occurred when repeated high aPCC (activated Prothrombin Complex Concentrate) doses were used concurrently with emicizumab.”
In 2016, the New England Journal of Medicine (NEJM) explained that severe hemophilia A is commonly treated with infusions of Factor VIII. However, infusions are burdensome, with approximately 30% of patients developing antibodies. NEJM asked: “Would emicizumab, which mimics the function of factor VIII, reduce these problems?” (Image: adapted from nejm.org)
In a press release issued on Sunday, Shire added that the injunction also aims to correct promotion of the primary data results regarding ‘treated bleeds’ (a secondary endpoint) as compared to the primary endpoint of ‘number of bleeds over time’ established when the trial began.
Current emicizumab data insufficient
Based on Roche data that is publicly available, doctors, patients and caregivers may be misinformed regarding the appropriate management of the breakthrough bleeds that are uncontrolled by emicizumab, says Shire.
Shires also accuses Roche, through these actions, of unlawfully denigrating Shire’s proven bypassing agent FEIBA (Anti-Inhibitor Coagulant Complex).
Shire claims it made several requests to Roche in an effort to resolve these concerns ‘in an appropriate manner’, but received no response. “As a result, Shire made the decision to seek court intervention,” the company wrote.
Shire says it aims to make sure that the hemophilia community receives sufficient, accurate data from Roche regarding the reported SAEs (serious adverse events) in the Phase III emicizumab trial, thus enabling doctors and their patients to make proper and informed decisions regarding patient care.
Mariska, whose son has #hemophilia, talks about the burden of treatment. Watch the full video here: https://t.co/U0jtudMpOM pic.twitter.com/n0MTIe86Bt
— Roche (@Roche) July 8, 2017
Shire added:
“As the global leader in rare hematology, Shire advocates in the interest of patient safety and continually defends the demonstrated 40-year safety profile of FEIBA.”
Swiss pharmaceutical giant Roche wants to gain a slice of the $11 billion-per-year hemophilia drug market with emicizumab, which is also called ACE910.
Roche’s investigational medication is designed to compete with Shire’s and Novo Nordisk’s more traditional treatments.
Business Insider today quoted Roche, which said in an emailed statement (which did not respond to Shire’s statement):
“Our decisions and actions are always based on doing what is right for patients.”
Video – Emicizumab
This NEJM video explains what emicizumab is and how it could improve treatment options for patients with severe hemophilia A.