New supplementary applications for TRIKAFTA, SYMDEKO and KALYDECO HCFA-TEAM 1 Οκτωβρίου, 2020

New supplementary applications for TRIKAFTA, SYMDEKO and KALYDECO

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Vertex submits new supplementary applications for TRIKAFTA, SYMDEKO and KALYDECO for Additional CFTR Mutations

On 1st of September Vertex announced that FDA has accepted three new supplemental applications for TRIKAFTA (elexacaftor / tezacaftor / ivacaftor and ivacaftor), SYMDEKO® (tezacaftor / ivacaftor andivacaftor) and KALYDECO (ivacaftor). These regulatory submissions are intended to expand the labels for TRIKAFTA, SYMDEKO and KALYDECO to include additional rare CFTR mutations, allowing people with cystic fibrosis (CF) not previously eligible for these medicines an opportunity to benefit from treatment that targets the underlying cause of their disease. In addition, these regulatory submissions may also allow certain people with CF who are currently eligible for KALYDECO to become eligible for SYMDEKO or TRIKAFTA and certain people currently eligible for SYMDEKO may become eligible for TRIKAFTA. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 30, 2020. 

“We have spent the last 20 years discovering, developing and bringing new medicines to thousands of people with CF, and the regulatory submissions announced today are an important next step in our commitment to bring transformative medicines to everyone living with this disease” said David Altshuler, Executive Vice President, Global Research and Chief Scientific Officer. “Using our well-established in vitro approach, we have been able to generate data providing evidence that people with certain rare mutations could benefit from treating the underlying cause of their disease with CFTR modulators.”

Approximately 600 people in the U.S. who have certain rare CF mutations may benefit from TRIKAFTA, SYMDEKO or KALYDECO for the first time. In addition, more than 1,100 people with CF in the U.S. currently eligible for SYMDEKO or KALYDECO may have the option of an additional CFTR modulator. These sNDAs are based on in vitro data from a validated cell assay model showing that many rare mutations in the CFTR gene are responsive to one or more of Vertex’s medicines. Data generated from this model, along with Phase 3 clinical data, have already led to the inclusion of nearly 30 additional ultra-rare and rare mutations in the U.S. for KALYDECO and SYMDEKO, including the first ever FDA approval based on in vitro data for a KALYDECO label expansion in patients with residual function CFTR mutations.



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