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elexacaftor

Generic: ELEXACAFTOR, IVACAFTOR, TEZACAFTOR; IVACAFTOR·Brand: TRIKAFTA (COPACKAGED)

elexacaftor: sourced relationships with 1 company · 2 FDA decisions.

elexacaftor evidence translation

What we can confirm

What it means

Source-backed records connect elexacaftor to VRTX; that link alone does not prove the drug works.

What to watch

Watch for new trials or company disclosures after the FDA record.
BioSniper AIPage evidence connected

Prepared research question

How could elexacaftor → VRTX affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

Dated Pipeline Records (2)
CompanyDated record stage
VERTEX PHARMACEUTICALS INC / MA (VRTX)
Reported 2019-10-21 · Current development: unknownSource
Approved
VERTEX PHARMACEUTICALS INC / MA (VRTX)
Reported 2019-10-21 · Current development: unknownSource
Approved
FDA Decisions2 records
Approval1 INDICATIONS AND USAGE TRIKAFTA is indicated for the treatment of cystic fibrosis (CF) in adult and pediatric patients aged 2 years and older who have a clinical diagnosis of CF and who have at least one variant in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene that is either responsive based on clinical and/or in vitro data (see Table 6 ) or results in production of CFTR protein [see Clinical Pharmacology (12.1) ] . If the patient's genotype is unknown, an FDA-cleared CF genetic test should be used to confirm the presence of at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. TRIKAFTA is a combination of ivacaftor, a CFTR potentiator, tezacaftor, and elexacaftor indicated for the treatment of cystic fibrosis (CF) in adult and pediatric patients aged 2 years and older who have a clinical diagnosis of CF and who have at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. ( 1 , 12.1 ) If the patient's genotype is unknown, an FDA-cleared CF genetic test should be used to confirm the presence of at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. ( 1 )(VRTX)FDA ↗
NDA217660Type 3 - New Dosage FormOrphan DrugPriorityOralGranule
Approval1 INDICATIONS AND USAGE TRIKAFTA is indicated for the treatment of cystic fibrosis (CF) in adult and pediatric patients aged 2 years and older who have a clinical diagnosis of CF and who have at least one variant in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene that is either responsive based on clinical and/or in vitro data (see Table 6 ) or results in production of CFTR protein [see Clinical Pharmacology (12.1) ] . If the patient's genotype is unknown, an FDA-cleared CF genetic test should be used to confirm the presence of at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. TRIKAFTA is a combination of ivacaftor, a CFTR potentiator, tezacaftor, and elexacaftor indicated for the treatment of cystic fibrosis (CF) in adult and pediatric patients aged 2 years and older who have a clinical diagnosis of CF and who have at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. ( 1 , 12.1 ) If the patient's genotype is unknown, an FDA-cleared CF genetic test should be used to confirm the presence of at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. ( 1 )(VRTX)FDA ↗
NDA212273Type 1 - New Molecular Entity and Type 4 - New CombinationOrphan DrugPriorityOralTablet

Frequently asked questions

Which companies have sourced relationships with elexacaftor?

elexacaftor has dated SEC/FDA relationship records for VERTEX PHARMACEUTICALS INC / MA.

What is elexacaftor's latest FDA decision?

The most recent tracked FDA decision for elexacaftor is Approval for 1 INDICATIONS AND USAGE TRIKAFTA is indicated for the treatment of cystic fibrosis (CF) in adult and pediatric patients aged 2 years and older who have a clinical diagnosis of CF and who have at least one variant in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene that is either responsive based on clinical and/or in vitro data (see Table 6 ) or results in production of CFTR protein [see Clinical Pharmacology (12.1) ] . If the patient's genotype is unknown, an FDA-cleared CF genetic test should be used to confirm the presence of at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. TRIKAFTA is a combination of ivacaftor, a CFTR potentiator, tezacaftor, and elexacaftor indicated for the treatment of cystic fibrosis (CF) in adult and pediatric patients aged 2 years and older who have a clinical diagnosis of CF and who have at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. ( 1 , 12.1 ) If the patient's genotype is unknown, an FDA-cleared CF genetic test should be used to confirm the presence of at least one variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. ( 1 ), dated April 26, 2023.