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FDA Accepts Vertex's NDA for Next-Gen Cystic Fibrosis Triple Therapy

• The FDA has accepted Vertex's NDA for vanzacaftor/tezacaftor/deutivacaftor, a triple combination therapy for cystic fibrosis (CF) patients aged 6 and older. • This therapy targets those with at least one F508del mutation or another mutation responsive to the drug in the CFTR gene. • The FDA granted priority review, setting a PDUFA target action date for January 2, 2025, potentially expediting the availability of this treatment option. • Clinical trials suggest vanzacaftor could lower sweat chloride levels below the diagnostic threshold for CF, potentially improving patient outcomes.

Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for the investigational once-daily vanzacaftor/tezacaftor/deutivacaftor triple combination therapy, aimed at treating cystic fibrosis (CF) in patients aged 6 years and older. This next-generation therapy targets individuals with at least one F508del mutation or another mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the vanza triple.
The FDA's acceptance includes a priority review voucher, which shortens the review period from ten to six months, setting the Prescription Drug User Fee Act (PDUFA) target action date for January 2, 2025. Vertex has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the same patient population, with submissions also made in Canada, Australia, Switzerland, and the U.K.

Addressing CFTR Mutations

Cystic fibrosis, a rare and life-shortening genetic disease, affects over 92,000 people globally. It is caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. The most common mutation is F508del. These mutations disrupt the flow of salt and water across cell membranes, leading to the buildup of thick, sticky mucus in the lungs and other organs.
Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex, stated, “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.”

Mechanism of Action

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.

Clinical Significance

Diagnosis of CF is confirmed by testing sweat chloride (SwCl) levels, where a level ≥60 mmol/L indicates CF. Levels between 30-59 mmol/L suggest CF is possible, warranting further testing. Restoring CFTR function leads to lower SwCl levels, which are associated with improved outcomes, including better lung function and fewer pulmonary exacerbations. Vertex aims to achieve SwCl levels below 30 mmol/L, typical of CF carriers without the disease.
The investigational vanzacaftor/tezacaftor/deutivacaftor has been granted Fast Track and Orphan Drug Designations by the FDA. This new triple combination therapy may offer a significant advancement in CF treatment, potentially improving the quality of life and survival rates for affected individuals. The vanza triple will also be subject to a lower single-digit royalty obligation, compared to Vertex’s current CF portfolio.
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Reference News

[1]
Vertex Announces FDA Acceptance of New Drug Application for Vanzacaftor/Tezacaftor/Deutivacaftor, a Next-In-Class Triple Combination Treatment for Cystic Fibrosis
drugs.com · Jul 2, 2024

Vertex's vanzacaftor/tezacaftor/deutivacaftor triple therapy NDA accepted by FDA for cystic fibrosis, targeting those ag...

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