Swissmedic grants Roche authorisation extension for prophylactic treatment in people with moderate or mild haemophilia A

• Hemlibra is now also approved for the treatment of people of all ages with moderate or mild haemophilia A.
  
  
• First and only haemophilia prophylaxis that can be administered subcutaneously and at different dosing intervals (weekly, every two weeks or every four weeks).¹

Basel, 5 September 2022 – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that Swissmedic, the Swiss Agency for Therapeutic Products, has extended the authorisation of Hemlibra® (emicizumab) to include the treatment of moderate or mild haemophilia A without factor VIII inhibitors.

Subcutaneous treatment with the bispecific antibody Hemlibra has been approved in Switzerland for patients with severe haemophilia A with antibodies to coagulation factor VIII (FVIII), called inhibitors, since 6 November 2018 and for people with severe haemophilia A without inhibitors since August 2019. Hemlibra can be used in all approved indications regardless of age.¹ The authorisation extension is based on the multicentre, open-label, single-arm phase III study HAVEN 6 (BO41423).²

Haemophilia is a serious inherited blood clotting disorder that can cause uncontrolled and often spontaneous bleeding. As well as being at increased risk from external injuries, people with haemophilia A can also experience spontaneous internal bleeding into muscles and joints. Particularly often this involves the knees, elbows and ankles. Hemlibra has the potential to prevent these bleeds.^2,3

Dr Jean-Marc Häusler, Country Medical Director, Roche Pharma Switzerland, comments: “We are very pleased that Hemlibra has now also been approved for people with moderate or mild haemophilia A. Preventing bleeds can be particularly challenging for young children and their families. With less frequent treatments the drug can potentially lead to freedom from bleeding and thus a better quality of life.”

Haemophilia A is caused by a coagulation factor VIII (FVIII) deficiency that prevents the blood from clotting normally. It affects about 600 people in Switzerland. Treatment with Hemlibra differs fundamentally from previous replacement therapy with factor VIII preparations that are injected intravenously. As a monoclonal antibody, Hemlibra is administered subcutaneously and because of its long half-life of more than 26 days,¹ treatment intervals of one, two or four weeks are available with Hemlibra.¹ Hemlibra binds to both activated coagulation factor IX (FIXa) and factor X (FX), thereby mimicking the function of FVIII and thus largely restoring blood clotting.⁴

Easier handling means improved quality of life
Hemlibra offers people with congenital haemophilia A a treatment option that provides effective bleeding control and ease of handling, whatever their inhibitor status and disease severity. Besides the clinically relevant reduction in bleeds^2,3,5-8, another advantage is the ease of administration: the active substance emicizumab is injected into subcutaneous fatty tissue, for example in the thigh or abdomen.¹ This method of administration particularly benefits people with congenital haemophilia A whose veins are difficult to access, such as some children or elderly individuals. Thanks to the three treatment intervals (once weekly, every two weeks or every four weeks), those affected by haemophilia A can choose the treatment schedule that best suits their daily routine and current life situation. The HAVEN trial programme showed that over 90% of study participants preferred emicizumab to their previous haemophilia treatment.^2,7,8 

Clinical trials
The authorisation extension is based on the multicentre, open-label, single-arm phase III study HAVEN 6 (BO41423).² The aim was to supplement the safety and efficacy data from the previous HAVEN trial programme evaluating the use of emicizumab in people with severe congenital haemophilia A (including randomised studies BH29884 [HAVEN 1]³ and BH30071 [HAVEN 3]⁶) with data from the mild and moderate haemophilia A population in order to demonstrate a consistent risk-benefit balance. Over a median (IQR) treatment period of 55.6 (8.7-89.9) weeks, emicizumab (Hemlibra) showed consistent efficacy across all analysed bleeding endpoints and clinically significant bleed control in the study population with mild and moderate haemophilia A, in keeping with earlier HAVEN studies.^2,3,5-8 No new safety signals were identified with emicizumab in the non-severe haemophilia A population. Emicizumab (Hemlibra) thus offers an effective treatment option with a favourable safety profile for people with mild and moderate congenital haemophilia A without factor VIII inhibitors who require prophylactic therapy.

About Hemlibra® (emicizumab)
Hemlibra is an antibody, which is designed to bring together factor IXa and factor X, proteins involved in the natural coagulation cascade, thus restoring the blood clotting process for people with haemophilia A. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once-weekly, every two weeks, or every four weeks (after an initial once-weekly dose for the first four weeks). Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai, Roche and Genentech. 

About haemophilia A
Haemophilia A is a serious inherited disorder in which the blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Haemophilia A affects around 815,000 people worldwide⁸, approximately 35-39% of whom have a severe form of the disorder. People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa and X, which is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their disorder, people with haemophilia A can bleed frequently, especially into their joints or muscles. These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility and long-term joint damage. A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies. Inhibitors are antibodies developed by the body’s immune system that bind to replacement factor VIII, thereby blocking its function and interfering with blood clotting. Depending on the number of inhibitors formed, blood clotting may be suppressed so severely that bleeds can no longer be promptly and adequately controlled by a factor VIII replacement therapy.

About Roche in haematology
Roche has been developing medicines for people with malignant and non-malignant blood diseases for over 20 years; our experience and knowledge in this therapeutic area runs deep. Today, we are investing more than ever in our effort to bring innovative treatment options to patients across a wide range of haematological diseases.

About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.

In recognising our endeavor to pursue a long-term perspective in all we do, Roche has been named one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for the thirteenth consecutive year. This distinction also reflects our efforts to improve access to healthcare together with local partners in every country we work.

Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.

For more information, please visit www.roche.com.

All trademarks used or mentioned in this release are protected by law.

References
[1] Hemlibra® (Emicizumab) Fachinformation, Stand der Information Juli 2022, www.swissmedicinfo.ch
[2] Hermans C, et al. Emicizumab Prophylaxis for the Treatment of People with Moderate or Mild Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 6 Study. Presented at: International Society on Thrombosis and Haemostasis (ISTH) Congress; 2022 July 11. Abstract OC 30.5
[3] Callaghan M, et al. Long term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies. Blood 2021; 137(16):2231-2242
[5] Oldenburg, Johannes et al. “Emicizumab Prophylaxis in Hemophilia A with Inhibitors.” The New England journal of medicine vol. 377,9 (2017): 809-818. doi:10.1056/NEJMoa1703068
[6] Young, Guy et al. “A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors.” Blood vol. 134,24 (2019): 2127-2138. doi:10.1182/blood.2019001869
[4] Sampei, Zenjiro et al. “Identification and multidimensional optimization of an asymmetric bispecific IgG antibody mimicking the function of factor VIII cofactor activity.” PloS one vol. 8,2 (2013): e57479. doi:10.1371/journal.pone.0057479
[7] Mahlangu, Johnny et al. “Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors.” The New England journal of medicine vol. 379,9 (2018): 811-822. doi:10.1056/NEJMoa1803550
[8] Pipe, Steven W et al. “Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study.” The Lancet. Haematology vol. 6,6 (2019): e295-e305. doi:10.1016/S2352-3026(19)30054-7
[9] World Federation of Hemophilia. World Federation of Hemophilia report on the annual global survey 2019


Roche Group Media Relations
Phone: +41 61 688 8888 / e-mail: media.relations@roche.com

Attachment

• 05092022_Swissmedic_Hemlibra_en