GlobeNewswire by notified

New data reinforce the benefit of early preventative treatment with Roche’s Hemlibra for babies with severe haemophilia A

  • Phase III HAVEN 7 primary data presented at ASH 2023 provide additional confidence in the favourable efficacy and safety profile of subcutaneous Hemlibra given soon after birth 1
  • At nearly two years median follow-up in the descriptive, single-arm study, no babies experienced spontaneous bleeds requiring treatment, and all treated bleeds were as a result of trauma 1
  • Safety results were consistent with previous studies of Hemlibra, with no new safety signals observed 1
  • The HAVEN 7 study was developed in collaboration with the haemophilia A community, to generate additional evidence for the prophylactic treatment of infants with haemophilia A

Basel, 09 December 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the primary analysis of the Phase III HAVEN 7 study reinforced the efficacy and safety of Hemlibra® (emicizumab) in previously untreated or minimally treated infants with severe haemophilia A without factor VIII inhibitors. Results showed that Hemlibra achieved meaningful bleed control in babies up to 12 months of age, and was well tolerated.1 The new data were presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition taking place 9-12 December 2023, in San Diego, California, and included in the press programme.

“Haemophilia A can have a devastating impact on any patient, but this is especially true for infants, where the emotional and physical stress due to frequent hospital visits, treatment administration and other worries can be distressing for babies and their parents and caregivers,” said Steven Pipe, MD, professor of paediatrics and pathology at the University of Michigan. “These results reinforce the benefit of starting prophylaxis as soon as possible after birth, as well as for the use of subcutaneous treatments, which are especially valuable in young babies where access to veins can be very difficult.”

The burden of severe haemophilia A in babies and on their parents and caregivers is significant. The World Federation of Haemophilia treatment guidelines consider the standard of care in haemophilia to be regular prophylaxis initiated at a young age, as studies have shown this improves long-term outcomes, while reducing the risk of intracranial haemorrhage.[2-4] However, for many babies with haemophilia A, prophylaxis is not started until after the first year of life because of the high treatment burden.[5-8] Hemlibra, which is already approved and being used to treat babies with haemophilia A, provides a flexible treatment option that can be administered subcutaneously from birth at different dosing frequencies.9

“The results of HAVEN 7 provide additional confidence in the efficacy and safety profile of Hemlibra for babies with severe haemophilia A, and add to its extensive clinical and real-world evidence across all ages,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “Conducted in collaboration with the haemophilia A community, this trial reflects our ongoing commitment to listen and respond to the needs of those impacted by this condition, in hopes of advancing treatment standards even further.”

The HAVEN 7 study is a Phase III, descriptive, single-arm study, set up in collaboration with the haemophilia A community to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of subcutaneous Hemlibra in infants with severe haemophilia A without factor VIII inhibitors. These results, which included data from 55 participants, showed that at 101.9 weeks median follow-up, 54.5% of participants (n=30) did not have any bleeds that required treatment, while 16.4% (n=9) did not have any treated or untreated bleeds at all. There were no spontaneous bleeds requiring treatment in any participant, and all treated bleeds were as a result of trauma. A total of 207 bleeds occurred in 46 participants (83.6%); 87.9% of these were as a result of trauma. Model-based annualised bleeding rate (95% CI) was 0.4 (0.30–0.63) for treated bleeds. No new safety signals were observed and there were no treatment-related serious adverse events, intracranial haemorrhages or deaths reported. Just 3.6% of participants (n=2) tested positive for factor VIII inhibitors, which may be a consequence of reduced factor VIII usage in participants treated with Hemlibra, and no participant tested positive for anti-drug antibodies.[1] Results were consistent with positive results from the interim analysis and from previous Phase III HAVEN studies.10-14

The results of additional research on biomarkers in the HAVEN 7 study were also presented at ASH, and were supportive of the study’s primary efficacy analysis. This additional research showed that the pharmacodynamic profiles of Hemlibra in babies were consistent with those previously observed in older children and adults with haemophilia A. The data showed that Hemlibra exhibits the expected pharmacodynamic response, despite the reduced presence of the clotting factors that Hemlibra binds to in this age group.15

The Phase III HAVEN 7 study results complement data from the broader, pivotal HAVEN clinical programme, providing insights into the evolution of haemophilia A in babies, and the impact of initiating preventative treatment from birth. The primary analysis is being followed by a seven year extension period.1 Hemlibra continues to redefine standards of care in haemophilia A, as a flexible treatment option approved across all ages and stages of life, regardless of inhibitor status and at different dosing frequencies. It is approved for the routine prophylaxis of people with haemophilia A in more than 115 countries worldwide. It has been studied in one of the largest clinical trial programmes in people with haemophilia A with and without factor VIII inhibitors, including eight Phase III studies.

About Hemlibra® (emicizumab)
Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins involved in the natural coagulation cascade, and restore 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).9 Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai, Roche and Genentech. It is marketed in the United States by Genentech as Hemlibra (emicizumab-kxwh), with kxwh as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration.

About haemophilia A
Haemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Haemophilia A affects around 900,000 people worldwide.2,16 The burden of severe haemophilia A in infants and on their parents and caregivers is significant. 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 symptoms, people with haemophilia A can bleed frequently, especially into their joints or muscles.17 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.18 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 and block the efficacy of replacement factor VIII, making it difficult, if not impossible, to obtain a level of factor VIII sufficient to control bleeding.2

About Roche in haematology
Roche has been developing medicines for people with malignant and non-malignant blood diseases for more than 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 haematologic diseases. Our approved medicines include MabThera®/Rituxan® (rituximab), Gazyva®/Gazyvaro® (obinutuzumab), Polivy® (polatuzumab vedotin), Venclexta®/Venclyxto® (venetoclax) in collaboration with AbbVie, Hemlibra® (emicizumab), Lunsumio® (mosunetuzumab) and Columvi® (glofitamab). Our pipeline of investigational haematology medicines includes T-cell engaging bispecific antibody cevostamab, targeting both FcRH5 and CD3, Tecentriq® (atezolizumab), a monoclonal antibody designed to bind with PD-L1, and crovalimab, an anti-C5 antibody engineered to optimise complement inhibition. Our scientific expertise, combined with the breadth of our portfolio and pipeline, also provides a unique opportunity to develop combination regimens that aim to improve the lives of patients even further.

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 endeavour 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

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

[1] Pipe S, et al. Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study. Presented at American Society of Hematology (ASH) congress; 2023 December 10. Abstract 505.
[2] Srivastava A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26 (Suppl 6): 1-158.
[3] Manco-Johnson MJ, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. New England Journal of Medicine. 2007;357(6):535–544.
[4] Andersson NG, et al. Intracranial haemorrhage in children and adolescents with severe haemophilia A or B – the impact of prophylactic treatment. British Journal of Haematology. 2017;179(2):298–307; 4.
[5] Spagrud LJ, et al. Pain, distress, and adult-child interaction during venipuncture in pediatric oncology: an examination of three types of venous access. Journal of Pain and Symptom Management. 2008;36:173–84.
[6] Van den Berg HM, et al. Timing of inhibitor development in more than 1000 previously untreated patients with severe hemophilia A. Blood 2019;134:317–320.
[7] Valentino LA & Kapoor M. Central venous access devices in patients with hemophilia. Expert Rev Med Devices 2005;2:699–711.
[8] Mancuso M, et al. Prophylaxis in children with haemophilia in an evolving treatment landscape. Haemophilia. 2021;27:889–896.
[9] Hemlibra SmPC [Internet; cited 2023 December] Available from:
[10] Pipe S, et al. Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study. Presented at American Society of Hematology (ASH) virtual congress; 2022 December 10. Abstract 187.
[11] Mancuso ME, et al. Emicizumab Prophylaxis in Adolescent/Adult Patients with Hemophilia A Previously Receiving Episodic or Prophylactic Bypassing Agent Treatment: Updated Analyses from the HAVEN 1 Study. Blood. 2017;130 (Supplement 1):1071.
[12] Young G, et al. Emicizumab prophylaxis provides flexible and effective bleed control in children with hemophilia A with inhibitors: results from the HAVEN 2 study. Blood. 2018;132 (Supplement 1):632.
[13] Mahlangu J, et al. Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors. N Engl J Med. 2018;379:811-822.
[14] Pipe S, 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. 2019; DOI:
[15] Pipe S, et al. Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7. Presented at American Society of Hematology (ASH) congress; 2023 December 09. Abstract P1238.
[16] Iorio A, et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males. Ann Intern Med. 2019;171(8):540-546.
[17] NHS. Symptoms of haemophilia [Internet; cited 2023 December]. Available from:
[18] Franchini M, et al. Haemophilia A in the third millennium. Blood Rev. 2013; 179-84.

Roche Global Media Relations
Phone: +41 61 688 8888 / e-mail:

Hans Trees, PhD
Phone: +41 79 407 72 58
Dr. Rebekka Schnell
Phone: +41 79 205 27 03
Simon Goldsborough
Phone: +44 797 32 72 915
Karsten Kleine
Phone: +41 79 461 86 83
Nina Mählitz
Phone: +41 79 327 54 74
Kirti Pandey
Phone: +49 172 6367262
Sileia Urech
Phone: +41 79 935 81 48

Roche Investor Relations

Dr. Bruno Eschli
Phone: +41 61 68-75284
Dr. Sabine Borngräber
Phone: +41 61 68-88027

Dr. Birgit Masjost
Phone: +41 61 68-84814
Dr. Gerard Tobin
Phone: +41 61 68-72942

Investor Relations North America

Loren Kalm
Phone: +1 650 225 3217


To view this piece of content from, please give your consent at the top of this page.
To view this piece of content from, please give your consent at the top of this page.

About GlobeNewswire by notified

GlobeNewswire by notified
GlobeNewswire by notified
One Liberty Plaza - 165 Broadway
NY 10006 New York

GlobeNewswire by notified is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.

Subscribe to releases from GlobeNewswire by notified

Subscribe to all the latest releases from GlobeNewswire by notified by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from GlobeNewswire by notified

Generalforsamling - frist for fremsættelse af emner26.2.2024 11:53:00 CET | pressemeddelelse

Tivoli A/S afholder ordinær generalforsamling tirsdag den 23. april 2024, kl. 13:30 i Tivolis Koncertsal. Forslag fra aktionærer til emner behandlet på generalforsamlingen skal fremsættes senest mandag den 11. marts 2024. Eventuelle forslag sendes skriftligt til Tivolis aktionærer vil forventeligt modtage indkaldelse til generalforsamlingen i slutningen af marts 2024. Med venlig hilsen Susanne Mørch Koch Adm. direktør Kontaktperson: Juridisk chef, Julie Koefoed: Vedhæftet fil Tivoli AS - Fondsbørsmeddelelse nr 2 - Generalforsamling i 2024, frist for emner

Annual General Meeting - Deadline for submitting proposals for the agenda26.2.2024 11:53:00 CET | Press release

Tivoli A/S’ Annual General Meeting will take place on Tuesday 23 April 2024 at 1:30 pm CET at the Tivoli Concert Hall. Any proposals for the agenda of the Annual General Meeting must be submitted no later than Monday 11 March 2024. Any proposals must be submitted in writing to The invitations for the Annual General Meeting are expected to be sent to Tivoli’s shareholders by the end of March 2024. Best regards Susanne Mørch Koch CEO Contactperson: Head of Legal, Julie Koefoed: Attachment Tivoli AS - Stock Exchange Announcement no 2 - Annual General Meeting in 2024 submitting proposals for agenda

Sparinvest SICAV ophæver suspension af handel med alle afdelinger26.2.2024 11:49:04 CET | pressemeddelelse

København, Feb. 26, 2024 (GLOBE NEWSWIRE) -- Under henvisning til Nasdaq Copenhagens regler for udstedere af investeringsbeviser skal Sparinvest S.A. hermed på vegne af de berørte afdelinger i Sparinvest SICAV offentliggøre, at der igen kan foretages beregning af indre værdier for de pågældende afdelinger. De indre værdier er indberettet til Nasdaq Copenhagen. Suspension af handel med de berørte afdelinger ophæves hermed. Der er tale om følgende afdelinger: Fund NameISINOrder Book CodeEquitas EUR RLU0362354549SSIEEURREthical Global Value EUR RLU0362355355SSIEGVEURREuropean Value EUR RLU0264920413SSIEUVEURRGlobal Value EUR RLU0138501191SSIGVEURRSustainable Corporate Bonds IG EUR RLU0264925727SSIGLIGEURRLong Danish Bonds DKK RLU0138507396SSILDBDKKRBalance EUR RLU0650088072SSIBAEURRBalance DKK RLU0673458609SSIBDKKRProcedo EUR RLU0139792278SSIPEURRProcedo DKK RLU0686499277SSIPDKKRSecurus EUR RLU0139791205SSISEURRSecurus DKK RLU0686498972SSISDKKRGlobal Convertible Bonds EUR RLU1438960566SSI

NNIT A/S: Reporting of transactions in NNIT’s shares made by person discharging managerial responsibilities26.2.2024 11:30:00 CET | Press release

NNIT has pursuant to article 19 of the Market Abuse Regulation received notification of transactions by person with managerial responsibilities in NNIT. Reference is made to the attached table showing detailed information about the transactions. Contact for further information Carsten Ringius EVP & CFO Tel: +45 3077 8888 Media relations Tina Joanne Hindsbo Media Relations Manager Tel: +45 3077 9578 NNIT is a leading provider of IT solutions to life sciences internationally, and to the public and enterprise sectors in Denmark We focus on high complexity industries and thrive in environments where regulatory demands and complexity are high. We advise and build sustainable digital solutions that work for the patients, citizens, employees, end users or customers. We strive to build unmatched excellence in the industries we serve, and we use our domain expertise to represent a business first approach – strongly supported by a selection of partner technologies, bu

Ekstraordinære indfrielser (CK93) - Nykredit Realkredit A/S26.2.2024 11:22:00 CET | pressemeddelelse

Til Nasdaq Copenhagen Ekstraordinære indfrielser (CK93) I medfør af kapitalmarkedsloven § 24 offentliggør Nykredit Realkredit A/S hermed oplysninger om ekstraordinære indfrielser (CK93) pr. den 23. Februar 2024 i vedhæftede fil. Oplysningerne vil endvidere på sædvanlig måde blive formidlet via Nasdaq Copenhagen. Oplysningerne findes også i excel-format i Nykredits obligationsdatabase på For yderligere informationer om dataformat og -indhold henvises til Nasdaqs hjemmeside. Spørgsmål kan rettes til head of investor relations Morten Bækmand Nielsen på tlf. 44 55 15 21. Med venlig hilsen Nykredit Realkredit A/S Vedhæftede filer Ekstraordinære indfrielser - Nykredit Realkredit A_S - 26-02-2024XMLeordindf

HiddenA line styled icon from Orion Icon Library.Eye