FDA Approves Merck’s WINREVAIR™ (sotatercept-csrk), a First-in-Class Treatment for Adults with Pulmonary Arterial Hypertension (PAH, WHO* Group 1)
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
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240326077272/en/
WINREVAIR logo (Graphic:
“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.
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 (
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
“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.
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.
“PAH remains a debilitating disease with high morbidity and mortality,” said Dr.
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
- 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 (
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
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,
WINREVAIR is the subject of a licensing agreement with
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
At
Forward-Looking Statement of
This news release of
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 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
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.
*
View source version on businesswire.com: https://www.businesswire.com/news/home/20240326077272/en/
Media Contacts:
(617) 519-6264
(917) 691-6218
Investor Contacts:
(732) 594-1579
(732) 594-1583
Source: