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 haematologyRoche 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 www.roche.com.
All trademarks used or mentioned in this release are protected
by law.References[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:
https://www.medicines.org.uk/emc/product/9043/smpc[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:https://doi.org/10.1016/S2352-3026(19)30054-7.[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:
https://www.nhs.uk/conditions/haemophilia/symptoms/.[18] Franchini
M, et al. Haemophilia A in the third millennium. Blood Rev. 2013;
179-84.
Roche Global Media RelationsPhone: +41 61 688
8888 / e-mail: media.relations@roche.com
Hans Trees, PhDPhone: +41 79 407
72 58 |
Dr.
Rebekka SchnellPhone: +41 79 205 27 03 |
Simon
GoldsboroughPhone: +44 797 32 72 915 |
Karsten
KleinePhone: +41 79 461 86 83 |
Nina
MählitzPhone: +41 79 327 54 74 |
Kirti
PandeyPhone: +49 172 6367262 |
Sileia
UrechPhone: +41 79 935 81 48 |
|
Roche Investor Relations
Dr. Bruno
Eschli Phone: +41 61 68-75284 e-mail:
bruno.eschli@roche.com |
Dr.
Sabine BorngräberPhone: +41 61 68-88027 e-mail:
sabine.borngraeber@roche.com |
Dr.
Birgit MasjostPhone: +41 61 68-84814e-mail:
birgit.masjost@roche.com |
Dr.
Gerard Tobin Phone: +41 61 68-72942 e-mail:
gerard.tobin@roche.com |
Investor Relations North America
Loren
KalmPhone: +1 650 225 3217 e-mail:
kalm.loren@gene.com |
- 09122023_MR_ASH Hemlibra HAVEN 7_en
Roche (LSE:0QQ6)
Historical Stock Chart
From May 2024 to Jun 2024
Roche (LSE:0QQ6)
Historical Stock Chart
From Jun 2023 to Jun 2024