Vertex Presents New Data Across Portfolio of Cystic Fibrosis Medicines Including ALYFTREK® at the North American Cystic Fibrosis Conference

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- Data presented on outcomes following treatment with CFTR modulators add to growing body of evidence that reduced level of sweat chloride is associated with improved clinical outcomes, particularly in younger people with cystic fibrosis - BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the presentation of multiple abstracts demonstrating the clinical benefits of treatment with CFTR modulators, including the Company’s most recently approved medicine, ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor), at the North American Cystic Fibrosis Conference (NACFC) held from October 22-25 in Seattle, Washington . Several of the abstracts that will be presented at this year’s conference demonstrate that improvement in CFTR function, as measured by reduction in sweat chloride (SwCl), is associated with improved outcomes in people with cystic fibrosis (CF). ALYFTREK has shown greater reductions in sweat chloride compared to TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Phase 3 trials, suggesting that ALYFTREK has the potential for even greater improvements in quality of life and other health-related outcomes. Data being presented on ALYFTREK and on correlation between lower SwCl and improved clinical outcomes with CFTR modulator treatment: “Decreased Pulmonary Exacerbations and Lower IV Antibiotic Usage Following Vanzacaftor/Tezacaftor/Deutivacaftor Treatment in People with CF with F508del-Minimal Function Genotypes” (Poster #868): Post-hoc analyses of two pivotal Phase 3 studies (SKYLINE 102 and SKYLINE 103) will be presented, showing that F580del/minimal function mutation (F/MF) subjects treated with ALYFTREK had lower rates of pulmonary exacerbations (PEx), PEx requiring hospitalization or IV antibiotics, and IV antibiotic use vs. F/MF subjects treated with TRIKAFTA. Friday, October 24,1:15–2:15 p.m. PDT. “Maximizing benefits with early CFTR modulator treatment: Lower sweat chloride is associated with improved clinical outcomes in children aged 6 to 11 years” (Poster #119): Data will be presented from a pooled analysis of data from participants in Phase 3 studies and open-label extension studies of CFTR modulators, including ALYFTREK. An analysis of three age cohorts (≥18 years, 12-17 years, and 6-11 years) with F/MF or F508del/F508del (F/F) mutations shows that participants who initiated therapy at a younger age achieved greater levels of CFTR function, as demonstrated by lower levels of SwCl. Among study participants in the 6-11 years cohorts, those that achieved lower SwCl levels experienced broad clinical benefits — as indicated by greater numerical improvements in lung function, respiratory symptoms, nutritional status and annual rates of PEx. Friday, October 24 , 12:15–1:15 p.m. PDT. “Changes in Sweat Chloride Concentrations Following CFTR Modulator Treatment and Association with Antibiotic Usage in Adolescents and Adults with Cystic Fibrosis” (Poster #120) : Data will be presented from a pooled analysis of over 2,000 people with CF 12 years and older with F/MF or F/F genotypes who took part in Phase 3 clinical trials of CFTR modulators, including ALYFTREK. These data demonstrate that, following the initiation of treatment with CFTR modulators, achieving sweat chloride levels below the diagnostic threshold (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, 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 Hypersensitivity reactions, including cases of angioedema and anaphylaxis, have been reported in the postmarketing 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 to determine whether to resume treatment PATIENTS WHO DISCONTINUED OR INTERRUPTED ELX-, TEZ-, OR IVA-CONTAINING DRUGS DUE TO ADVERSE REACTIONS ALYFTREK 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 benefits and risks before using ALYFTREK in these patients and if used, closely monitor for adverse reactions INTRACRANIAL HYPERTENSION (IH) IH has been reported in the postmarketing setting with TRIKAFTA, which contains the same or similar active ingredients as ALYFTREK. Clinical manifestations of IH include headache, blurred vision, diplopia, and potential vision loss; papilledema can be found on fundoscopy. If an unusual headache or visual disturbances occur during treatment, and IH is suspected, interrupt treatment and refer for prompt medical evaluation. Consider benefits and risks to determine whether to resume treatment. Patients should be monitored until IH resolution and for recurrence. Patients with elevated vitamin A levels may be at increased risk DRUG INTERACTIONS Use With CYP3A Inducers Following concomitant use of strong or moderate CYP3A inducers with ALYFTREK, exposures of vanzacaftor, TEZ, and deutivacaftor were decreased, which may reduce ALYFTREK effectiveness. Concomitant use with strong or moderate CYP3A inducers is not recommended Exposure to IVA is significantly decreased and exposure to ELX and TEZ are expected to decrease with concomitant use of CYP3A inducers, which may reduce effectiveness of TRIKAFTA. Concomitant use with strong CYP3A inducers is not recommended Use With CYP3A Inhibitors Exposure to vanzacaftor, TEZ, and deutivacaftor or ELX, TEZ, and IVA 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 CATARACTS Non-congenital lens opacities have been reported in pediatric patients treated with TRIKAFTA, which contains IVA (similar to an active ingredient in ALYFTREK). Baseline and follow-up ophthalmological examinations are recommended in pediatric patients ADVERSE REACTIONS ALYFTREK 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%) 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 (URTI), headache, oropharyngeal pain, influenza, fatigue, increased ALT and AST, rash, and sinus congestion TRIKAFTA Serious adverse reactions that occurred more frequently in patients treated with TRIKAFTA compared to placebo included rash (1% vs

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