F. Hoffmann-La Roche Ltd / FDA approves Roche's Hemlibra for haemophilia
A without factor VIII inhibitors . Processed and transmitted by West
Corporation. The issuer is solely responsible for the content of this
announcement.
-- First medicine to significantly reduce treated bleeds compared to prior
factor VIII prophylaxis based on an intra-patient comparison
-- Only medicine that can be self-administered subcutaneously once weekly,
every two weeks or every four weeks for haemophilia A with and without
factor VIII inhibitors
-- The efficacy and safety of Hemlibra has been demonstrated in one of the
largest pivotal clinical trial programmes in haemophilia A
Basel, 4 October 2018 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced
today that the US Food and Drug Administration (FDA) has approved
Hemlibra(R) (emicizumab-kxwh) for routine prophylaxis to prevent or
reduce the frequency of bleeding episodes in adults and children, ages
newborn and older, with haemophilia A without factor VIII inhibitors.
Hemlibra is now the only prophylactic treatment for people with
haemophilia A with and without factor VIII inhibitors that can be
administered subcutaneously (under the skin) and at multiple dosing
options (once weekly, every two weeks or every four weeks). This
approval is based on positive results from the phase III HAVEN 3 and
HAVEN 4 studies. Hemlibra prophylaxis led to statistically significant
and clinically meaningful reductions in treated bleeds compared to no
prophylaxis (primary endpoint) and across all other bleed-related
endpoints in the HAVEN 3 study, and showed a clinically meaningful
control of bleeding in the HAVEN 4 study.
"Many preventative treatment options for people with haemophilia A
without factor VIII inhibitors require intravenous infusions several
times a week. Even then, people can still experience bleeds, and there
has been a need for more treatment options," said Michael Callaghan, MD,
haematologist, Children's Hospital of Michigan, Detroit. "The approval
of Hemlibra is an important advancement for the entire haemophilia A
community, as we now have a new class of medicine for the first time in
nearly 20 years. Hemlibra can reduce bleeds, and it offers a new
subcutaneous administration once weekly, every two weeks or every four
weeks."
"Today's approval of Hemlibra reflects our commitment to groundbreaking
science and the development of medicines with the potential to redefine
the standard of care," said Sandra Horning, MD, Roche's Chief Medical
Officer and Head of Global Product Development. "Hemlibra is now the
only FDA-approved medicine for people with haemophilia A with and
without factor VIII inhibitors, based on the efficacy and safety profile
demonstrated across four pivotal studies. We want to thank the
haemophilia community for their partnership in helping us bring this new
option to everyone living with haemophilia A."
In the phase III HAVEN 3 study, adults and adolescents aged 12 years or
older with haemophilia A without factor VIII inhibitors who received
Hemlibra prophylaxis once weekly (n=36) or every two weeks (n=35)
experienced a 96% (95% CI: 92.5; 98.0, p<0.0001) and 97% (95% CI: 93.4;
98.3, p<0.0001) reduction in treated bleeds, respectively, compared to
those who received no prophylaxis (n=18). Hemlibra is the first medicine
to significantly reduce treated bleeds compared to prior factor VIII
prophylaxis, which has been the recommended standard of care, as
demonstrated by a statistically significant reduction of 68% (95% CI:
48.6; 80.5, p<0.0001) in treated bleeds in a prospective intra-patient
comparison (n=48) of people who previously received factor VIII
prophylaxis in a non-interventional study and switched to Hemlibra
prophylaxis. In the single-arm phase III HAVEN 4 study of adults and
adolescents aged 12 years or older with haemophilia A with factor VIII
inhibitors (n=5) and without factor VIII inhibitors (n=36), Hemlibra
prophylaxis every four weeks (n=41) led to clinically meaningful control
of bleeding. The most common adverse reactions occurring in 10% or more
of people treated with Hemlibra in pooled studies (n=391) were injection
site reactions (n=85), headache (n=57) and joint pain (arthralgia;
n=59).
Hemlibra was granted Breakthrough Therapy Designation by the FDA for
haemophilia A without factor VIII inhibitors. It was also granted
Priority Review, a designation given to medicines that the FDA has
determined to have the potential to provide significant improvements in
the treatment, prevention or diagnosis of a serious disease. The
company's Marketing Authorisation Application (MAA) variation for
haemophilia A without factor VIII inhibitors, which includes data from
the HAVEN 3 and HAVEN 4 studies, is under review by the European
Medicines Agency (EMA). Submissions to other regulatory authorities
around the world are ongoing.
Hemlibra was approved by the FDA in November 2017 for adults and
children with haemophilia A with factor VIII inhibitors. It has been
studied in one of the largest pivotal clinical trial programmes in
people with haemophilia A with and without factor VIII inhibitors,
including four pivotal HAVEN studies (HAVEN 1, HAVEN 2, HAVEN 3 and
HAVEN 4).
About HAVEN 3 (NCT02847637)
HAVEN 3 is a randomised, multicentre, open-label, phase III study
evaluating the efficacy, safety and pharmacokinetics of Hemlibra
prophylaxis versus no prophylaxis (episodic/on-demand factor VIII
treatment) in people with haemophilia A without factor VIII inhibitors.
The study included 152 patients with haemophilia A (12 years of age or
older) who were previously treated with factor VIII therapy either
on-demand or for prophylaxis. Patients previously treated with on-demand
factor VIII were randomised in a 2:2:1 fashion to receive subcutaneous
Hemlibra prophylaxis at 3 mg/kg/wk for four weeks, followed by 1.5
mg/kg/wk for at least 24 weeks (Arm A; n=36), subcutaneous Hemlibra
prophylaxis at 3 mg/kg/wk for four weeks, followed by 3 mg/kg/2wks (Arm
B; n=35) for at least 24 weeks or no prophylaxis (Arm C; n=18) for at
least 24 weeks. Patients previously treated with factor VIII prophylaxis
received subcutaneous Hemlibra prophylaxis at 3 mg/kg/wk for four weeks,
followed by 1.5 mg/kg/wk until the end of study (Arm D; n=48). Episodic
treatment of breakthrough bleeds with factor VIII therapy was allowed
per protocol.
HAVEN 3 met its primary endpoint and key secondary endpoints. Data from
the study showed:
-- Hemlibra prophylaxis once weekly or every two weeks resulted in a 96%
(95% CI: 92.5; 98.0, p<0.0001) and 97% (95% CI: 93.4; 98.3, p<0.0001)
reduction in treated bleeds, respectively, compared to no prophylaxis.
-- 55.6% (95% CI: 38.1; 72.1) of people treated with Hemlibra once weekly
and 60% (95% CI: 42.1; 76.1) of people treated with Hemlibra every two
weeks experienced zero treated bleeds, compared to 0% (95% CI: 0.0; 18.5)
of people treated with no prophylaxis.
-- 91.7% (95% CI: 77.5; 98.2) of people treated with Hemlibra prophylaxis
once weekly and 94.3% (95% CI: 80.8; 99.3) of people treated with
Hemlibra prophylaxis every two weeks experienced three or fewer treated
bleeds, compared to 5.6% (95% CI: 0.1; 27.3) of people treated with no
prophylaxis.
-- Hemlibra prophylaxis once weekly or every two weeks resulted in a 95%
(95% CI: 85.7; 98.4, p<0.0001) and 95% (95% CI: 85.3; 98.2, p<0.0001)
reduction in treated target joint bleeds, respectively, compared to no
prophylaxis.
-- Hemlibra prophylaxis once weekly or every two weeks resulted in a 95%
(95% CI: 90.1; 97.0, p<0.0001) and 94% (95% CI: 89.7; 97.0, p<0.0001)
reduction in all bleeds, respectively, compared to no prophylaxis.
-- Hemlibra prophylaxis once weekly demonstrated a statistically significant
reduction of 68% (95% CI: 48.6; 80.5, p<0.0001) in treated bleeds
compared to prior factor VIII prophylaxis based on a prospective
intra-patient comparison of people who were previously enrolled in a
non-interventional study.
-- The most common adverse reactions occurring in 10% or more of people
treated with Hemlibra in pooled studies (n=391) were injection site
reactions (n=85), headache (n=57) and joint pain (arthralgia; n=59).
About HAVEN 4 (NCT03020160)
HAVEN 4 is a single-arm, multicentre, open-label, phase III study
evaluating the efficacy, safety and pharmacokinetics (PK) of
subcutaneous administration of Hemlibra dosed every four weeks. The
study included 48 patients (12 years of age or older) with haemophilia A
with or without factor VIII inhibitors who were previously treated with
either factor VIII or bypassing agents, on-demand or as prophylaxis. The
study was conducted in two parts: a PK run-in; and an expansion cohort.
All patients in the PK run-in (n=7) were previously treated on-demand
and received subcutaneous Hemlibra at 6 mg/kg to fully characterise the
PK profile after a single dose during four weeks, followed by 6 mg/kg
every four weeks for at least 24 weeks. In the expansion cohort (n=41),
patients with haemophilia A with factor VIII inhibitors (n=5) and
without factor VIII inhibitors (n=36) received subcutaneous Hemlibra
prophylaxis at 3 mg/kg/wk for four weeks, followed by 6 mg/kg every four
weeks for at least 24 weeks. Episodic treatment of breakthrough bleeds
with factor VIII therapy or bypassing agents, depending on a patient's
factor VIII inhibitor status, was allowed per study protocol.
In the HAVEN 4 study, 56.1% (95% CI: 39.7; 71.5) of people with or
without factor VIII inhibitors treated with Hemlibra prophylaxis every
four weeks experienced zero treated bleeds and 90.2% (95% CI: 76.9;
97.3) experienced three or fewer treated bleeds.
About Hemlibra (emicizumab)
(MORE TO FOLLOW) Dow Jones Newswires
October 04, 2018 12:40 ET (16:40 GMT)
© 2018 Dow Jones News
