Health Canada Giving Priority Review to Trikafta

Health Canada Giving Priority Review to Trikafta
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Health Canada is reviewing an application that seeks the approval of Trikafta, a triple combination reported to treat 90% of all with cystic fibrosis (CF), or those with least one F508del mutation.

The request from Trikafta’s developer, Vertex Pharmaceuticals, covers eligible patients ages 12 and older, and has been granted priority review. This means a review timeline of 180 days rather than the conventional 300 days; a final decision from Health Canada is expected in the first half of this year.

“We are pleased this submission has been accepted for Priority Review by Health Canada, and we anticipate this accelerated review process will enable access for patients as early as possible,” Carmen Bozic, MD, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex, said in a press release.

The nonprofit patient group Cystic Fibrosis Canada has pushed for Trikafta’s approval, and a study based on Canadian data suggested that, if the medication is approved, CF-related deaths in Canada could drop by 15% over the next 10 years.

CF is caused by mutations in the gene CFTR, which encodes a protein of the same name. Normally, the CFTR protein acts like a “gate” on cell membranes, controlling the flow of certain ions in and out of cells. However, because the protein is mutated in people with CF, it does not work as it should.

Trikafta is a CFTR modulator, meaning it helps mutated CFTR to function correctly. Specifically, Trikafta is a combination of three therapies: elexacaftortezacaftor, and ivacaftor.

Elexacaftor and tezacaftor are correctors. They bind to the mutated CFTR protein and help it fold correctly, preventing the protein from being degraded and getting more of it to a cell’s membrane. Ivacaftor is a potentiator; it binds to CFTR and helps to keep the “gate” open.

Because these medications correct specific problems with the CFTR protein, Trikafta is designed for people with specific mutations in the CFTR gene, but this includes the F508del mutation, the most common CF-causing mutation. In clinical trials, Trikafta was shown to significantly improve lung function in CF patients with at least one F508del mutation.

The medication was approved in the U.S. in 2019 for CF patients ages 12 and older with either two F508del mutations, or one F508del mutation and one minimal function mutation. It was approved in Europe, under the name Kaftrio, for the same indication in 2020.

Vertex recently announced plans to seek expansion of the U.S. approval to cover children with CF as young as 6 years old.

Marisa, a science writer, holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
Total Posts: 336

Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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Marisa, a science writer, holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
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