Vertex Pharmaceuticals’ New Drug Application for vanzacaftor/tezacaftor/deutivacaftor, a next-generation triple combination therapy for cystic fibrosis, has been accepted by the FDA with priority review status.
Key points:
- The FDA has set a PDUFA target action date of January 2, 2025, reducing the review time from 10 to 6 months.
- The therapy is designed for CF patients ages 6 and older with at least one F508del mutation or another responsive mutation in the CFTR gene.
- Vertex has also received validation of its Marketing Authorization Application by the European Medicines Agency.
Market estimate: The global cystic fibrosis therapeutics market is expected to reach $17.7 billion by 2030, according to Grand View Research.
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In a significant development for cystic fibrosis (CF) patients worldwide, Vertex Pharmaceuticals (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for a groundbreaking triple combination therapy.
The treatment, known as vanzacaftor/tezacaftor/deutivacaftor (vanza triple), has been granted priority review status, potentially accelerating its path to market.
The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of January 2, 2025, reducing the standard review time from 10 months to just 6 months. This expedited timeline underscores the potential impact of the therapy on CF treatment.
Dr. Nia Tatsis, Executive Vice President and Chief Regulatory and Quality Officer at Vertex, emphasized the significance of this milestone: “The FDA acceptance of our vanza triple application represents an important step in the decades-long development of CFTR modulators and another example of our track record of serial innovation in CF.”
The vanza triple therapy is designed for CF patients aged 6 and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This broad applicability could potentially benefit a significant portion of the CF patient population.
Vertex’s new therapy aims to raise the bar set by its predecessor, TRIKAFTA®. According to Dr. Tatsis, the vanza triple gives more people with CF the chance to achieve levels of sweat chloride below the diagnostic threshold for CF, and even to levels seen in individuals without the disease. This could represent a significant leap forward in CF treatment efficacy.
The global implications of this development are substantial. Vertex has also received validation of its Marketing Authorization Application (MAA) by the European Medicines Agency (EMA) for patients ages 6 years and older. Additionally, the company has submitted applications in Canada, Australia, Switzerland, and the UK, signaling a comprehensive global rollout strategy.
The potential market impact of this therapy is considerable. While specific projections for the vanza triple are not available at the time of writing, the global cystic fibrosis therapeutics market is expected to reach $17.7 billion by 2030, according to Grand View Research. Vertex, already a leader in CF treatment, could further solidify its market position with this new therapy.
Cystic fibrosis, affecting over 92,000 people globally, is a rare, life-shortening genetic disease that impacts multiple organs, including the lungs, liver, and pancreas. The median age of death is in the 30s, underscoring the critical need for more effective treatments.
Vertex’s vanza triple therapy represents a potential paradigm shift in CF treatment. By targeting the underlying cause of the disease rather than just its symptoms, it offers hope for improved quality of life and potentially extended lifespans for CF patients.
