- Oral presentation on outcomes following treatment with CFTR modulators show that improvements in CFTR function, as measured by lower sweat chloride, correlate with better outcomes for people with cystic fibrosis -
- Oral presentation on new post hoc data from randomized, controlled and open-label trials suggest improved quality of life results with ALYFTREK compared to TRIKAFTA® –
BOSTON--(BUSINESS WIRE)--
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data across multiple studies demonstrating positive clinical and quality of life benefits of treatment with CFTR modulators and, in particular, ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor), which is approved in the United States and United Kingdom and is currently under review with health authorities in the EU, Canada, Australia, New Zealand and Switzerland. These data were presented at this year’s European Cystic Fibrosis Society’s (ECFS) 48th European Cystic Fibrosis Conference held June 4-7, 2025, in Milan, Italy.
During the conference, the Company presented data from a pooled analysis across CFTR modulators, including ALYFTREK, which demonstrate that reduction in sweat chloride (SwCl), and therefore greater restoration of CFTR function, is associated with improved outcomes in people with cystic fibrosis (CF). For all clinical outcomes in the study, SwCl levels below 60 mmol/L were associated with greater benefit including better and more stable lung function, fewer pulmonary exacerbations, better nutritional status and better quality of life. SwCl levels below 30 mmol/L generally demonstrated greater numerical benefit than all other groups, with confidence intervals that overlapped with the ≥30 to 5x the upper limit of normal (ULN) or ALT or AST >3x ULN with bilirubin >2x ULN)
Clinical symptoms suggestive of liver injury (e.g., jaundice, right upper quadrant pain, nausea, vomiting, altered mental status, ascites)
Consider referral to a hepatologist and follow patients closely with clinical and laboratory monitoring until abnormalities resolve. If resolved, and if benefit is expected to outweigh risk, resume treatment with close monitoring
ALYFTREK and TRIKAFTA should not be used in patients with severe hepatic impairment. ALYFTREK and TRIKAFTA are not recommended in patients with moderate hepatic impairment and should only be considered when there is a clear medical need and benefit outweighs risk. If ALYFTREK is used, monitor patients closely. If TRIKAFTA is used, use with caution at a reduced dosage and monitor patients closely
HYPERSENSITIVITY REACTIONS, INCLUDING ANAPHYLAXIS
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Hypersensitivity reactions, including cases of angioedema and anaphylaxis, have been reported in the post marketing setting for TRIKAFTA. If signs or symptoms of serious hypersensitivity reactions develop during treatment, discontinue ALYFTREK or TRIKAFTA and institute appropriate therapy. Consider benefits and risks for the individual patient to determine whether to resume treatment
PATIENTS WHO DISCONTINUED OR INTERRUPTED ELX-, TEZ-, OR IVA-CONTAINING DRUGS DUE TO ADVERSE REACTIONS
ALYFTREK
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There are no available safety data for ALYFTREK in patients who previously discontinued or interrupted treatment with drugs containing ELX, TEZ, or IVA due to adverse reactions. Consider the benefits and risks before using ALYFTREK in these patients. If ALYFTREK is used in these patients, closely monitor for adverse reactions as clinically appropriate
DRUG INTERACTIONS
Use With CYP3A Inhibitors
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Exposure to vanzacaftor, tezacaftor, and deutivacaftor, or elexacaftor, tezacaftor, and ivacaftor are increased when used concomitantly with strong or moderate CYP3A inhibitors. The dose of ALYFTREK or TRIKAFTA should be reduced when used concomitantly with moderate or strong CYP3A inhibitors
Use With CYP3A Inducers
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Following concomitant use of strong or moderate CYP3A inducers with ALYFTREK, exposures of vanzacaftor, tezacaftor, and deutivacaftor were decreased, which may reduce ALYFTREK effectiveness. Concomitant use with strong or moderate CYP3A inducers is not recommended
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Exposure to ivacaftor is significantly decreased and exposure to elexacaftor and tezacaftor are expected to decrease by concomitant use with CYP3A inducers, which may reduce therapeutic effectiveness of TRIKAFTA. Concomitant use with strong CYP3A inducers, such as rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, and St. John’s wort, is not recommended
CATARACTS
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Non-congenital lens opacities/cataracts have been reported in pediatric patients treated with TRIKAFTA, which contain ivacaftor (similar to an active ingredient in ALYFTREK). Baseline and follow-up ophthalmological examinations are recommended in pediatric patients initiating treatment
ADVERSE REACTIONS
ALYFTREK
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Serious adverse reactions that occurred more frequently with ALYFTREK than with ELX/TEZ/IVA in 2 or more patients (≥0.4%) were influenza (1.5%), increased AST (0.4%), increased GGT (0.4%), depression (0.4%), and syncope (0.4%)
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The most common adverse reactions occurring in ≥5% of patients and at a frequency higher than ELX/TEZ/IVA by ≥1% were cough, nasopharyngitis, upper respiratory tract infection, headache, oropharyngeal pain, influenza, fatigue, increased ALT, rash, increased AST, and sinus congestion
TRIKAFTA
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Serious adverse reactions that occurred more frequently in patients treated with TRIKAFTA compared to placebo included rash (1% vs
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