WINREVAIR is a breakthrough biologic for
this rare, progressive disease
WINREVAIR on top of background therapy
significantly improved exercise capacity and multiple important
secondary outcome measures compared to background therapy
alone
Merck (NYSE: MRK), known as MSD outside of the United States and
Canada, announced today that the U.S. Food and Drug Administration
(FDA) has approved sotatercept-csrk (U.S. Brand Name: WINREVAIR™,
for injection, 45mg, 60mg) for the treatment of adults with
pulmonary arterial hypertension (PAH, World Health Organization
[WHO] Group 1) to increase exercise capacity, improve WHO
functional class (FC), and reduce the risk of clinical worsening
events. WINREVAIR was previously granted Breakthrough Therapy
Designation by the FDA. WINREVAIR is the first FDA-approved activin
signaling inhibitor therapy for PAH, representing a new class of
therapy that works by improving the balance between pro- and
anti-proliferative signaling to regulate vascular cell
proliferation underlying PAH.
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WINREVAIR logo (Graphic: Merck & Co.,
Inc.)
“Pulmonary arterial hypertension is a rare, progressive and
ultimately life-threatening disease in which blood vessels in the
lungs thicken and narrow, causing significant strain on the heart,”
said Dr. Marc Humbert, Professor of Medicine and Director of the
Pulmonary Hypertension Reference Center at the Université
Paris-Saclay and investigator on the Phase 3 STELLAR study. “Based
on the Phase 3 STELLAR trial, adding WINREVAIR to background PAH
therapy demonstrated significant clinical benefits compared to
background PAH therapy alone. This approval is an important
milestone, as it offers healthcare providers a novel therapeutic
option that targets a new PAH treatment pathway.”
The approval is based on the Phase 3 STELLAR trial, which
compared WINREVAIR (n=163) to placebo (n=160), both in combination
with background standard of care therapies in adult patients with
PAH (WHO Group 1 FC II or III). Results showed adding WINREVAIR to
background therapy increased six-minute walk distance from baseline
by 41 meters (95% CI: 28, 54; p<0.001; placebo-adjusted) at Week
24 and significantly improved multiple important secondary outcome
measures, including reducing the risk of death from any cause or
PAH clinical worsening events by 84% versus background therapy
alone (number of events: 9 vs 42, hazard ratio=0.16; 95% CI: 0.08,
0.35; p<0.001).
Healthcare providers should monitor hemoglobin and platelets
before each dose of WINREVAIR for the first 5 doses, or longer if
values are unstable, and periodically thereafter to determine if
dose adjustments are required. WINREVAIR may increase hemoglobin
and may lead to erythrocytosis, which if severe may increase the
risk of thromboembolic events or hyperviscosity syndrome. WINREVAIR
also may decrease platelet count and lead to severe
thrombocytopenia, which may increase the risk of bleeding;
thrombocytopenia occurred more frequently in patients also
receiving prostacyclin infusion. Treatment should not be initiated
if platelet count is <50,000/mm3. See additional Selected Safety
Information below.
“The Pulmonary Hypertension Association welcomes the development
of new therapies for those with PAH,” said Matt Granato, president
and chief executive officer, Pulmonary Hypertension Association. “A
diagnosis of PAH is a life-changing experience for patients and
families due to its chronic, progressive nature. Patients with PAH
experience limiting symptoms such as shortness of breath and
fatigue. We are excited to see industry research leading to a
better understanding of PAH and the development of a medicine in a
novel treatment pathway that expands options for the patient
community.”
“New treatment options continue to be needed for patients with
pulmonary arterial hypertension that support important clinical
goals, including increasing exercise capacity and improving
functional class,” said Dr. Aaron Waxman, Executive Director of the
Center for Pulmonary Heart Diseases at Brigham and Women’s Hospital
and investigator on the Phase 3 STELLAR study. “Sotatercept added
to background therapy has the potential to become a new standard of
care option for patients with pulmonary arterial hypertension.”
WINREVAIR is given once every three weeks by subcutaneous
injection and may be administered by appropriate patients or
caregivers with guidance, training and follow-up from a healthcare
provider. Healthcare providers and patients/caregivers should refer
to the Instructions for Use for information on the proper
preparation and administration of WINREVAIR. Merck estimates that
WINREVAIR will be available for dispensing by select specialty
pharmacies in the U.S. by the end of April.
“PAH remains a debilitating disease with high morbidity and
mortality,” said Dr. Eliav Barr, senior vice president and head of
global clinical development, chief medical officer, Merck Research
Laboratories. “This approval of WINREVAIR is an important milestone
and a testament to our science-led strategy and focus on the
development of innovations that can help people affected by rare
diseases like PAH. We are proud to bring this novel medicine to
patients.”
Merck offers support to patients who are prescribed WINREVAIR,
including information about insurance coverage and help for
eligible patients with out-of-pocket costs and co-pay assistance
options, through the Merck Access Program. For additional
information, healthcare providers and patients can call
1-888-637-2502 or visit www.merckaccessprogram-WINREVAIR.com in the
coming days.
STELLAR Study Results
The primary efficacy endpoint in the STELLAR trial was the
change from baseline at Week 24 in 6-Minute Walk Distance (6MWD).
In the WINREVAIR treatment group, the placebo-adjusted median
increase in 6MWD was 41 meters (95% CI: 28, 54; p<0.001,
Hodges-Lehmann estimate). Additionally, patients who added
WINREVAIR had statistically significantly greater improvements
compared to placebo across multiple secondary endpoints:
- Treatment with WINREVAIR led to an improvement in FC from
baseline at Week 24 in 29% of patients compared to 14% of patients
treated with placebo (p<0.001).
- Treatment with WINREVAIR resulted in an 84% reduction in the
occurrence of death or PAH clinical worsening events compared to
placebo (median duration of exposure 33.6 weeks; number of events:
9/163 vs 42/160, hazard ratio=0.16; 95% CI: 0.08, 0.35;
p<0.001).
- Treatment with WINREVAIR led to an improvement from baseline in
pulmonary vascular resistance (PVR). The median treatment
difference in PVR between WINREVAIR and placebo was -235
dynes*sec/cm5 (95% CI: -288, -181; p<0.001).
- Treatment with WINREVAIR led to an improvement from baseline in
N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels. The
median treatment difference in NT-proBNP between WINREVAIR and
placebo was -442 pg/mL (95% CI: -574, -310; p<0.001).
About STELLAR
The STELLAR study (NCT04576988) was a global, double-blind,
placebo-controlled, multicenter, parallel-group clinical trial in
which 323 patients with PAH (WHO Group 1 FC II or III) were
randomized 1:1 to WINREVAIR (target dose 0.7 mg/kg) (n=163) or
placebo (n=160) plus stable background therapy administered
subcutaneously once every 3 weeks.
The most common PAH etiologies were idiopathic PAH (59%),
heritable PAH (18%), and PAH associated with connective tissue
diseases (CTD) (15%). Most participants were receiving either three
(61%) or two (35%) background drugs for PAH, and 40% were receiving
prostacyclin infusions. The mean time from PAH diagnosis was 8.8
years. Patients had a WHO FC II (49%) or III (51%) at baseline.
About WINREVAIR™ (sotatercept-csrk) for injection, for
subcutaneous use, 45 mg, 60 mg
WINREVAIR is FDA-approved for the treatment of adults with
pulmonary arterial hypertension (PAH, WHO Group 1) to increase
exercise capacity, improve WHO functional class (FC) and reduce the
risk of clinical worsening events. WINREVAIR is the first activin
signaling inhibitor therapy approved to treat PAH. WINREVAIR
improves the balance between pro-proliferative and
anti-proliferative signaling to modulate vascular proliferation. In
preclinical models, WINREVAIR induced cellular changes that were
associated with thinner vessel walls, partial reversal of right
ventricular remodeling, and improved hemodynamics.
WINREVAIR is the subject of a licensing agreement with Bristol
Myers Squibb.
Selected Safety Information
WINREVAIR may increase hemoglobin and may lead to
erythrocytosis. Severe erythrocytosis may increase the risk of
thromboembolic events or hyperviscosity syndrome. Monitor Hgb
before each dose for the first 5 doses, or longer if values are
unstable, and periodically thereafter, to determine if dose
adjustments are required.
WINREVAIR may decrease platelet count and lead to severe
thrombocytopenia, which may increase the risk of bleeding;
thrombocytopenia occurred more frequently in patients also
receiving prostacyclin infusion. Do not initiate treatment if
platelet count is <50,000/mm3. Monitor platelets before each
dose for the first 5 doses, or longer if values are unstable, and
periodically thereafter to determine if dose adjustments are
required.
In clinical studies, serious bleeding (e.g., gastrointestinal,
intracranial hemorrhage)
was reported in 4% of patients taking WINREVAIR and 1% of
patients taking placebo. Serious bleeding was more likely in
patients on prostacyclin background therapy and/or antithrombotic
agents, or with low platelet counts. Advise patients about signs
and symptoms of blood loss. Do not administer WINREVAIR if the
patient is experiencing serious bleeding.
WINREVAIR may cause fetal harm when administered to a pregnant
woman. Advise pregnant women of the potential risk to a fetus.
Advise females of reproductive potential to use an effective method
of contraception during treatment with WINREVAIR and for at least 4
months after the final dose. Pregnancy testing is recommended for
females of reproductive potential before starting WINREVAIR
treatment.
Based on findings in animals, WINREVAIR may impair female and
male fertility. Advise patients on the potential effects on
fertility.
The most common adverse reactions occurring in the Phase 3
clinical trial (≥10% for WINREVAIR and at least 5% more than
placebo) were headache (24.5% vs 17.5%), epistaxis (22.1% vs 1.9%),
rash (20.2% vs 8.1%), telangiectasia (16.6% vs 4.4%), diarrhea
(15.3% vs 10.0%), dizziness (14.7% vs 6.2%), and erythema (13.5% vs
3.1%).
Because of the potential for serious adverse reactions in the
breastfed child, advise patients that breastfeeding is not
recommended during treatment with WINREVAIR, and for 4 months after
the final dose.
About Merck
At Merck, known as MSD outside of the United States and Canada,
we are unified around our purpose: We use the power of leading-edge
science to save and improve lives around the world. For more than
130 years, we have brought hope to humanity through the development
of important medicines and vaccines. We aspire to be the premier
research-intensive biopharmaceutical company in the world – and
today, we are at the forefront of research to deliver innovative
health solutions that advance the prevention and treatment of
diseases in people and animals. We foster a diverse and inclusive
global workforce and operate responsibly every day to enable a
safe, sustainable and healthy future for all people and
communities. For more information, visit www.merck.com and connect
with us on X (formerly Twitter), Facebook, Instagram, YouTube and
LinkedIn.
Forward-Looking Statement of Merck & Co., Inc., Rahway,
N.J., USA
This news release of Merck & Co., Inc., Rahway, N.J., USA
(the “company”) includes “forward-looking statements” within the
meaning of the safe harbor provisions of the U.S. Private
Securities Litigation Reform Act of 1995. These statements are
based upon the current beliefs and expectations of the company’s
management and are subject to significant risks and uncertainties.
There can be no guarantees with respect to pipeline candidates that
the candidates will receive the necessary regulatory approvals or
that they will prove to be commercially successful. If underlying
assumptions prove inaccurate or risks or uncertainties materialize,
actual results may differ materially from those set forth in the
forward-looking statements.
Risks and uncertainties include but are not limited to, general
industry conditions and competition; general economic factors,
including interest rate and currency exchange rate fluctuations;
the impact of pharmaceutical industry regulation and health care
legislation in the United States and internationally; global trends
toward health care cost containment; technological advances, new
products and patents attained by competitors; challenges inherent
in new product development, including obtaining regulatory
approval; the company’s ability to accurately predict future market
conditions; manufacturing difficulties or delays; financial
instability of international economies and sovereign risk;
dependence on the effectiveness of the company’s patents and other
protections for innovative products; and the exposure to
litigation, including patent litigation, and/or regulatory
actions.
The company undertakes no obligation to publicly update any
forward-looking statement, whether as a result of new information,
future events or otherwise. Additional factors that could cause
results to differ materially from those described in the
forward-looking statements can be found in the company’s Annual
Report on Form 10-K for the year ended December 31, 2023 and the
company’s other filings with the Securities and Exchange Commission
(SEC) available at the SEC’s Internet site (www.sec.gov).
Please see Prescribing Information for WINREVAIR
(sotatercept-csrk) at
http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_pi.pdf,
Patient Information for WINREVAIR at
http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ppi.pdf,
and Instructions for Use for WINREVAIR (1-vial kit, 2-vial kit)
at
https://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ifu_1-vial_2-vial_kits.pdf.
*WHO = World Health Organization
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