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Merck scores key FDA approval for pulmonary arterial hypertension drug Winrevair

2024-03-26

上市批准并购临床3期临床结果

Merck has earned an FDA approval for Winrevair (sotatercept) to treat patients with pulmonary arterial hypertension (PAH). Winrevair is the first disease-modifying treatment for the indication and is expected to achieve blockbuster sales.

Merck three years, tFDAcrown jewel ofWinrevair$1sotatercept acquisition of Acceleronpulmonary arterial hypertension (PAH)FDWinrevairval of Winrevair (sotatercept) to treat pulmonary arterial hypertension (PAH), Merck is set to launch the first disease-modifying treatment for the condition.

Initially developed by Acceleron and CMercke and investigated as a treatmenAcceleronorders such as osteoporosis, anemia aFDAmultiple myelomWinrevairrcsotaterceptnd its placpulmonary arterial hypertension (PAH) cMerckn a small but important indication.

PAH is a rare, rapidly Acceleronng disease in which the blood vessels narrow in the lungs, increasiosteoporosisssanemiad leamultiple myelomailsotatercepty 40,000 people in the United States have the disorder. PAH strikes more women than men and most are in middle age, killing nearly half within five years of diagnosis. The condition begins as a more common disorder, pulmonary hypertension (PH), then can progress into PAH.

PAHck will charge $14,000 for a vial of Winrevair, which is dosed subcutaneously every three weeks, putting its annual cost at $238,heart failure the label-recommended, weight-based target dose and data gatherPAHfrom the STELLAR trial, Merck projects that roughly two-thirds of patients will use a single-vial kit with others needed a double-vial package.pulmonary hypertension (PH)PAH

Merckonths ago, the Institute for ClinicWinrevaironomic Review (ICER) calculated a price range which matches the benefit patients are expected to receive for sotatercept at between $17,900 and $35,400 per year.Merck

"We believe the U.S. price of Winrevair represents the significant innovation this medicine can deliver as a first-in-class activin signaling inhibitor therapsotaterceptirst biologic approved by the FDA for PAH," Merck said in a statement.

Johnson & Johnson dominates the PAH market with Uptravi, a prostacyclin receptor (IP) agonist which generated sales of $1.6 billion last year on a 20% increase, and Opsumit, an endothelin receptor agFDAst whPAH produced $2 billion in revenue, with its sales up 14% from 2022.

Johnson & Johnsont four days agoPAHhe FDA approvUptravimbinprostacyclin receptor (IP)h combines Opsumit an United Therapeutics’ Adcirca (tadalafil), which are often Opsumitbed tendothelin receptor agonist endothelin receptorile all these therapies dilate the blood vessels to alleviate symptoms, they do little to ameliorate the disease.

As a physician who treated PAH patentsFDAo decades ago, Merck’s head of Opsynviclinical developmOpsumiterg United Therapeuticsurrent treatments have done little to extend lives.PAH

“When you make that diagnosPAH you essentially diagnose a disease that has a pretty high fatality,” Koglin said in an interview with Fierce Pharma. “What has happened over the last 20 years is while we had more and more tools to treat the symptoms of the disease—patients were able to walk a little bit longer and to deal with the disease better—but the mortality hasn’t really moved.”

But that could change with Winrevair. As a first-in-class activin signaling inhibitor, Winrevair acts directly on the underlying problem that causes the disease by targeting a type of protein that promotes inflammatory processes associated with the disease. Winrevair has been shown to modulate vascular cell proliferation, reversing vascular and right ventricle remodeling.

“We’ve learned that this narrowing of pulmonary vessels that leads to those increased pWinrevairin our lungs has to do with an imbalance in signaling factors,” Koglin said. “Once you develop an imbalance, you see an increase in pulmonary pressures and resistWinrevairthen all the downstream effects.”

In the phase 3 STELLAR trial, which conducted a 6-minute walk test as the primary endpoint, patients on sotatercept for 24 weeks as an add-on to standard of care improved their exercise capacity by an average distance of 41 meters.

Additionally, sotatercept provided statistically significant improvements in eight of nine secondary measotaterceptuding extending the time to a clinical worsening event, such as death, hospitalization or needing substantially more therapy.

“We were able sotaterceptduction in time to clinical worsening in those events—that’s a Kaplan-Meier analysis—by over 80%. That’s a dramatic effect size,” Koglin added. “And we saw that a substantial number of patients in our studies actually improved on the disease.”

The trial also showed that 24 weeks of sotatercept significantly reduced right heart size and improved right ventricle function and haemodynamic status.

After Bristol Myers Squibb bought out tsotaterceptther developer, Celgene, in 2019, Merck will now make royalty payments to BMS.

Merck Bristol Myers Squibbmmercialize Winrevair as it has had a PHCelgenen the markeMerck a decade in Adempas, which it sharBMSwith Bayer. Analysts have estimated Winrevair's peak sales potential is at least $2 billion, with J.P. Morgan’s Chris Schott estimating it at $3 billion to $4 billion.

Merckontrolled half of the $7.3 billioWinrevairket in 2023. Prophecy Market Insights projects the market will grow by 5.5% annually,Bayerhing $12.2 billion by 2032.J.P. Morgan

J&Jatercept also is under investigation in late-stage trials for PH due to left heart disease and for patients with PAH at intermediate or high risk of disease progression.