Monday, July 15, 2024

Vertex Expected to Solidify Dominance in Cystic Fibrosis Market with Vanza Triple Approval

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Vertex Pharmaceuticals is anticipated to solidify its leading position in the cystic fibrosis (CF) therapy market with the submission of regulatory marketing applications for its once-daily vanzacaftor triple (vanzacaftor/tezacaftor/deutivacaftor) to the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). The approval and success of the vanza triple could further cement Vertex’s dominance in CF treatment, according to GlobalData, a prominent data and analytics firm.

Vertex’s cystic fibrosis portfolio, which includes CFTR modulators such as Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), Symdeko/Symkevi (tezacaftor/ivacaftor and ivacaftor), and Trikafta/Kaftrio (elexacaftor/tezacaftor/ivacaftor and ivacaftor), has transformed the CF market by targeting the root cause of the disease. These treatments have set new standards in managing CF, greatly improving patients’ quality of life and prognosis.

Sravani Meka, Senior Immunology Analyst at GlobalData, commented: “Among Vertex’s therapies, Trikafta/Kaftrio stands out as the most effective, addressing a broader range of mutation profiles compared to earlier treatments. The potential approval of vanza triple could extend Vertex’s leadership in the CF market, particularly with Trikafta’s patent not expiring until 2037. Filed in February 2022, the patent for vanza triple positions Vertex to maintain its dominance with a new standard of care for cystic fibrosis patients.”

Vanza Triple’s Success in Phase III Trials Positions Vertex for Continued Dominance in Cystic Fibrosis Market

The submission of regulatory marketing applications follows successful Phase III trials (SKYLINE 102, SKYLINE 103, and RIDGELINE 105), where vanza triple met its primary endpoints, demonstrating noninferiority to Trikafta in improving lung function and superiority in reducing sweat chloride (SwCl) levels. While the direct correlation between SwCl levels and lung function improvement is still being studied, vanza triple presents a promising alternative for patients experiencing side effects with Trikafta, such as rash, liver enzyme dysregulation, and central nervous system issues, or those who prefer once-daily dosing regimen.

GlobalData projects Trikafta sales to reach $10.4 billion by 2030, with vanza triple expected to generate $5.5 billion in the same period, surpassing other Vertex CFTR modulators. These projections are driven by the high cost of treatment, with Trikafta averaging $948 per treatment day, equating to approximately $346,000 annually. The significant revenue potential highlights the economic importance of Vertex’s cystic fibrosis therapies.

Despite its success, Vertex faces pricing pressures and challenges regarding drug affordability and access. In April 2024, Vertex reached an agreement in South Africa to provide access to select patients via a private health plan following criticism over patent practices and the lack of Trikafta generics in South Africa, despite holding patents since 2019. This move underscores the broader global issue of drug accessibility and affordability.

Cystic Fibrosis

Reinforcing Commitment to Innovation and Accessibility in Cystic Fibrosis Care

Recently, Vertex appointed Dr. Jennifer Schneider, Co-founder & CEO of Homeward Health, to its board of directors. Her expertise in health systems, policy, and reimbursement is expected to help Vertex navigate these challenges. Dr. Schneider’s addition to the board reflects Vertex’s commitment to addressing these complex issues and ensuring broader access to their therapies. Meka concluded: “Advances in understanding and targeted therapies have transformed cystic fibrosis treatment. Vertex, with its CFTR modulators, has revolutionized care, notably with Trikafta/Kaftrio. The company’s regulatory submission for the once-daily vanza triple underscores its commitment to innovation. While vanza triple shows clinical promise, its market impact remains uncertain amid pricing pressures and advocacy group challenges.

“Vertex’s strategic moves, including negotiations over Trikafta access in South Africa and leadership enhancements, reflect its proactive stance in maintaining market leadership. The company’s ongoing efforts to expand access and improve affordability will be critical in sustaining its market position and ensuring that patients worldwide benefit from its groundbreaking therapies.”

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The potential approval of the vanza triple marks a significant milestone for Vertex, reinforcing its status as a leader in cystic fibrosis treatment innovation. As the company continues to navigate the complex landscape of drug pricing and accessibility, its commitment to improving patient outcomes remains clear. The success of vanza triple will not only solidify Vertex’s market dominance but also set a new standard of care for CF patients, offering hope for better management of this challenging disease.


Resource: GlobalData, June 24, 2024

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