The investigational therapy has received FDA priority review, potentially accelerating its availability for cystic fibrosis patients aged 6 and older with certain CFTR mutations.

RT’s Three Key Takeaways:

  1. FDA Priority Review: The FDA has accepted Vertex Pharmaceuticals’ New Drug Application for the vanzacaftor/tezacaftor/deutivacaftor triple combination therapy.
  2. Target Population: The treatment is intended for cystic fibrosis patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene.
  3. Global Submissions: In addition to the FDA, Vertex has also submitted marketing authorization applications for the vanza triple in the European Union, Canada, Australia, Switzerland, and the United Kingdom.

Vertex Pharmaceuticals Inc announced that the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for investigational once-daily vanzacaftor/tezacaftor/deutivacaftor triple combination therapy (vanza triple) for people living with cystic fibrosis (CF).

The treatment is for people aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene responsive to the vanza triple. 

Vertex used a priority review voucher for this submission, reducing the review time from 10 months to six months. This results in a Prescription Drug User Fee Act (PDUFA) target action date of Jan 2, 2025.

Vertex also received validation of its marketing authorization application (MAA) submission by the European Medicines Agency (EMA) in the European Union for patients ages 6 years and older. The company has also submitted in Canada, Australia, Switzerland, and the United Kingdom.

“The FDA acceptance of our vanza triple application and the MAA validation by the EMA represent important milestones in the decades-long development of CFTR modulators and another example of our track record of serial innovation in CF,” says Nia Tatsis, PhD, executive vice president and chief regulatory and quality officer at Vertex, in a release. “Vanzacaftor raises the high bar set by Trikafta and gives more people with CF the chance to get to levels of sweat chloride below the diagnostic threshold for CF, and even to levels of sweat chloride seen in those without CF.”

The ‘Vanza Triple’

In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. 

Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane.

Investigational vanzacaftor/tezacaftor/deutivacaftor was granted Fast Track and Orphan Drug Designations from the FDA for the treatment of cystic fibrosis.

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