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Roche outlines plan for communicating new findings on emicizumab (Hemlibra)

In a letter to the global hemophilia community, Roche describes its plan for keeping healthcare professionals and patients informed of new efficacy and safety data and findings on emicizumab (Hemlibra) from ongoing studies and clinical use in the real world. The relatively new therapy is now available in more than 90 countries and used by more than 7,200 patients with hemophilia A to date. Glenn Pierce, MD, PhD, WFH Vice President, Medical, noted, “Emicizumab has become an important medicine in the armamentarium of treatments for individuals with hemophilia A, with and without inhibitors. However, it has a novel mechanism of action distinct from clotting factor replacement products, hence the widespread interest in both long-term safety and efficacy. The letter from Roche details additional plans aimed at more transparency of emicizumab effects, including publication of new data, for the broader bleeding disorders community.”

Read the letter from Roche here:

An update from Roche on the evolution of Hemlibra®▼ post-approval data communications

23 September 2020

To the haemophilia community,

At Roche we recognise and appreciate the interest that the haemophilia community has in regards to Hemlibra®▼(emicizumab), specifically around the subject of its long-term efficacy and safety, both now and in the future. We understand that the subject of long-term safety for haemophilia treatments is an important topic also for the community, and at Roche we will continue to listen to the needs of the community and aspire to provide information that leads to better clinical decision making and patient care.

Recognising that Hemlibra was the first new type of medicine to be developed for haemophilia A in 20 years, a key priority during clinical development was to address any questions related to its efficacy and safety from the ongoing clinical trials programme, specifically related to serious adverse events (SAEs). This information was transparently and readily made available to the community through regular updates from Roche and on the emicizumabinfo.com website in the US. Today, Hemlibra is approved and available for people with haemophilia A in more than 90 countries and more than 7,200 patients have been treated with Hemlibra in the post-approval setting. The transition from clinical development to approved access for patients in many countries has meant a move from single patient reports to large databases of information on the use of Hemlibra. Having a much broader availability of information from databases and individual experiences means we can see early signals, identify trends and improve our overall understanding of the safety and efficacy profile of Hemlibra. During this transition, the source of safety information has also expanded from controlled clinical trial reporting to ongoing voluntary adverse event (AE) reporting by healthcare professionals, patients and their caregivers, as per local regulatory needs. This also changes the level of detail of information about individual safety cases that we receive. In light of this transition, and so that we continue to ensure transparency, as well as aim to meet the community’s expectations and needs for information, we have evolved our communication of post-approval information on Hemlibra.

We did so by actively engaging and working closely with the haemophilia medical and patient community. Together, we developed a plan to share new data and findings from ongoing studies and real world use, that give a more comprehensive picture of Hemlibra’s long-term safety and efficacy, providing valuable information that will support informed treatment decision making. Importantly, we consider that any updates and further information on Hemlibra should be appropriately benchmarked, medically relevant and externally validated. This means that, together with members of the scientific community, we can provide a broader body of data in place of the quarterly data updates previously reported, including:

    • Long-term follow-up data from Hemlibra’s pivotal clinical development programme: HAVEN 1 (adult people with haemophilia A (PwHA) with inhibitors), HAVEN 2 (paediatric PwHA with inhibitors), HAVEN 3 (PwHA without inhibitors) and HAVEN 4 (every 4 weekly dosing, PwHA with and without inhibitors)
    • New data from STASEY (safety and efficacy in PwHA with inhibitors), the largest long-term safety study conducted in haemophilia A to date
    • New findings from HAVEN 5 (PwHA in the Asia-Pacific region)
    • Findings from HAVEN 6 (people with mild and moderate haemophilia A without inhibitors)
    • Findings from HAVEN 7 (infants with severe haemophilia A without inhibitors)
    • Real-world data from community operated registries (EUHASS, PedNet, ATHN7)
    • As well as other post-approval supported studies and real-world data which address evidence and long-term exposure to Hemlibra

In addition, a series of manuscripts reviewing mortality in haemophilia A, and using this to establish a framework to assess the reported fatalities in people receiving Hemlibra, will be submitted to a peer-reviewed journal with the aim of publishing later this year.

In close collaboration with medical experts we will continue to provide additional long-term Hemlibra data, specifically for the first five years after being approved and available in many countries, to keep the community updated on long-term efficacy and serious AEs, including thromboembolic events (TEs) and thrombotic microangiopathy (TMAs).

Published information on Hemlibra’s benefit/risk profile and safety data, together with educational materials on post-approval safety reporting and the pharmacovigilance process, will be collectively available on a global website, available to all healthcare professionals around the world, where local laws and regulations allow. We will also ensure that patient organisations for haemophilia and bleeding disorders continue to receive appropriate information regarding the safety and efficacy of Hemlibra in a timely manner, so they may inform the patient communities that they serve.

Hemlibra’s label, as approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), should always be the primary source of information on the safety and efficacy of the medicine. Should any AE in a person taking Hemlibra impact the overall benefit/risk profile of the medicine, we will share this information as quickly as possible, which may include changes to the prescribing information. Information about AEs in people taking Hemlibra can always be obtained globally through the local Roche affiliate in each country according to local regulations. (Roche affiliate worldwide contacts can be found at www.roche.com/about/business/roche_worldwide).

Our promise and commitment to the haemophilia community – to provide timely and transparent updates on Hemlibra – remain unchanged. We will continue to listen, respond and partner with members of this community, so that together we can make a collective effort for a continued dialogue to protect patient safety.

▼ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.