Tezacaftor

-With this expansion, any variant that results in production of CFTR protein is now included in the indication for ALYFTREK and TRIKAFTA, reinforcing the impact these medicines have, regardless of the location of the variant in the CFTR protein- -Approximately 800 more people with CF in the US are now eligible for a CFTR modulator for the first time- BOSTON --(BUSINESS WIRE)--Apr. 1, 2026-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved expanded use of ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor) for the treatment of people with cystic fibrosis (CF) ages 6 and older with a variant in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. Additionally, the U.S. FDA has also expanded the indication statement for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in patients ages 2 and older. This label expansion was supported by clinical and/or in vitro data from 564 variants demonstrating response to ALYFTREK and 521 variants demonstrating response to TRIKAFTA. As such, approximately 800 more people with a clinical diagnosis of CF in the U.S. are now eligible for a medicine that treats the underlying cause of their disease for the first time. This extension means approximately 95% of people with CF in the U.S. are now eligible for treatment with a CFTR modulator. “This groundbreaking approval represents more than 20 years of innovation in CF, including testing over 600 variants in our laboratory, demonstrating clinical effects in large clinical trials, and studying younger people with CF so they can be treated as early as possible,” said Carmen Bozic , M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex . “With this label expansion, any variant that results in production of CFTR protein is now included in the ALYFTREK and TRIKAFTA labels, validating that these medicines can restore CFTR function and provide clinical benefit to patients regardless of where in the CFTR protein a variant is located. We thank the CF community and investigators for their trust and look forward to bringing ALYFTREK and TRIKAFTA to more patients than ever before.” For more information on ALYFTREK or TRIKAFTA, patient assistance programs, or eligibility details, visit ALYFTREK.com, TRIKAFTA.com, VertexGPS.com or vertextreatments.com. IMPORTANT SAFETY INFORMATION WARNING: DRUG-INDUCED LIVER INJURY AND LIVER FAILURE Elevated transaminases have been observed in patients treated with ALYFTREK. TRIKAFTA can cause serious and potentially fatal drug-induced liver injury. Cases of liver failure leading to transplantation and death have been reported in both clinical trials and the postmarketing setting in patients with and without a history of liver disease taking TRIKAFTA, a fixed-dose combination drug containing elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA), the same or similar active ingredients as ALYFTREK. Liver injury has been reported within the first month of therapy and up to 15 months following initiation of TRIKAFTA. Assess liver function tests (ALT, AST, alkaline phosphatase, and bilirubin) in all patients prior to initiating ALYFTREK or TRIKAFTA, then every month during the first 6 months of treatment, every 3 months for the next 12 months, and at least annually thereafter. Consider more frequent monitoring for patients with a history of liver disease or liver function test (LFT) elevations at baseline. Interrupt ALYFTREK or TRIKAFTA for significant elevations in LFTs or in the event of signs or symptoms of liver injury. Consider referral to a hepatologist. Follow patients closely with clinical and laboratory monitoring until abnormalities resolve. If resolved, resume treatment only if benefit is expected to outweigh risk. Closer monitoring is advised after resuming treatment. ALYFTREK or TRIKAFTA should not be used in patients with severe hepatic impairment (Child- Pugh Class C ). ALYFTREK or TRIKAFTA is not recommended in patients with moderate hepatic impairment (Child- Pugh Class B ). ALYFTREK or TRIKAFTA 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. WARNINGS AND PRECAUTIONS DRUG-INDUCED LIVER INJURY AND LIVER FAILURE Elevated transaminases have been observed in patients treated with ALYFTREK. TRIKAFTA can cause serious and potentially fatal drug-induced liver injury. Liver failure leading to transplantation and death has been reported in patients with and without a history of liver disease taking TRIKAFTA. Liver injury has been reported within the first month of therapy and up to 15 months following initiation of TRIKAFTA Assess LFTs in all patients prior to initiating ALYFTREK or TRIKAFTA, then every month during the first 6 months of treatment, every 3 months for the next 12 months, and at least annually thereafter. Consider more frequent monitoring for patients with a history of liver disease or LFT elevations at baseline, or a history of elevated LFTs with drugs containing ELX, TEZ, and/or IVA Interrupt ALYFTREK or TRIKAFTA in the event of signs or symptoms of liver injury, which may include: Significant elevations in LFTs (e.g., ALT or AST >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, 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 Significant elevations in LFTs (e.g., ALT or AST >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) 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 NEUROPSYCHIATRIC EVENTS, INCLUDING SUICIDAL THOUGHTS AND BEHAVIORS Serious neuropsychiatric events, including symptoms of anxiety, depression, suicidal ideation and behavior, and sleep disturbances, have been reported in the postmarketing setting in patients with and without a previous history of neuropsychiatric symptoms taking ALYFTREK or TRIKAFTA. Symptoms may occur within the first 3 months of treatment. Assess patients for baseline neuropsychiatric symptoms and monitor for new or worsening symptoms. Consider the benefits and risks to determine if treatment should be interrupted at symptom occurrence or resumed with symptom improvement 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 <1%) and influenza (1% vs 0%) The most common adverse reactions occurring in ≥5% of patients treated with TRIKAFTA and at a rate higher than placebo by ≥1% were headache; URTI; abdominal pain; diarrhea; rash; increased ALT, blood creatine phosphokinase, AST, and blood bilirubin; nasal congestion; rhinorrhea; rhinitis; influenza; sinusitis; and constipation USE IN SPECIFIC POPULATIONS PEDIATRIC USE Safety and effectiveness have not been established for ALYFTREK in patients <6 years, nor for TRIKAFTA in patients <2 years. The use in children under these ages is not recommended Please see full Prescribing Information, including Boxed WARNING, for ALYFTREK and TRIKAFTA. About Cystic Fibrosis Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 112,000 people, including approximately 97,000 people in the United States , Europe , Australia and Canada . CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving. Learn more about the importance of sweat chloride (SwCl) in cystic fibrosis. Today Vertex CF medicines are treating over 75,000 people with CF across more than 60 countries on six continents. This represents approximately 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston , with international headquarters in London . Additionally, the company has research and development sites and commercial offices in North America , Europe , Australia , Latin America and the Middle East . Vertex is consistently recognized as one of the industry's top places to work, including 16 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex 's history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and X. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Carmen Bozic , M.D., in this press release, statements regarding the anticipated benefits of ALYFTREK and TRIKAFTA, expectations regarding the eligible patient population, and expectations for patient access to ALYFTREK and TRIKAFTA. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that patients may not have access to ALYFTREK or TRIKAFTA on the anticipated timeline, and other risks listed under the heading “Risk Factors” in Vertex 's annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) View source version on businesswire.com: https://www.businesswire.com/news/home/20260401690804/en/ Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com Media: mediainfo@vrtx.com or U.S. : 617-341-6992 or International: +44 20 3204 5275 Source: Vertex Pharmaceuticals Incorporated

AUSTIN, Texas and TOKYO, Dec. 1, 2025 /PRNewswire/ -- According to DataM Intelligence, the Cystic Fibrosis (CF) Therapeutics Market Size accelerated to USD 10.60 billion in 2024, up from USD 9.22 billion in 2023, and is now projected to expand aggressively to USD 35.09 billion by 2033, recording a CAGR of 14.2% during 2025–2033. The surge is driven by transformational advancements in CFTR modulator therapies, higher treatment access across the U.S. and Europe, the expansion of personalized medicine programs, improvements in newborn screening, and stronger R&D investments targeting rare genetic diseases. Cystic fibrosis, a life-long autosomal recessive disorder caused by mutations in the CFTR gene, affects more than 162,000 patients globally, with rising diagnostic rates enabling broader therapeutic adoption. The past five years have shifted the market from symptomatic management to disease-modifying treatments, marking the most significant clinical progress in CF care in decades. Download PDF Brochure: Browse in-depth TOC on "Cystic Fibrosis Therapeutics Market" 71 – Tables 67 – Figures 192 – Pages Precision Medicine, Breakthrough Modulators & Improved Survival Rates Accelerate Market Adoption Cystic fibrosis has moved from a once-fatal childhood disease to a highly treatable chronic condition, thanks to advanced therapeutic classes. Today's market growth is being propelled by three major dynamics: 1. CFTR Modulator Innovations Are Reshaping the Standard of Care CFTR modulators such as ivacaftor, tezacaftor, elexacaftor, and combination regimens have expanded eligibility to nearly 90% of CF patients, dramatically improving lung function, BMI, respiratory exacerbation rates, and life expectancy. 2. Rising Diagnosis Rates and Earlier Intervention With neonatal screening improving globally, early treatment initiation is raising adherence and increasing long-term therapeutic demand. Countries in Europe and Asia have expanded newborn testing coverage by 12–20% YoY. 3. Growth of Symptom-Relief Therapies in Untreated CFTR Mutations A subset of patients still relies on mucolytics, bronchodilators, pancreatic enzyme replacement, and inhaled antibiotics-ensuring sustained demand across legacy CF drug segments. Market Segmentation: By Drug Class (CFTR Modulators, Pancreatic Enzyme Supplements, Mucolytics, Bronchodilators, Others) CFTR Modulators CFTR modulators represented the dominant segment in 2024, accounting for nearly 72% of total revenue (USD 7.63 billion). This class continues to see explosive uptake because of its disease-modifying capabilities and expanded label approvals across adolescents and pediatric patients. Pancreatic Enzyme Supplements Pancreatic enzyme replacement therapies (PERT) accounted for 11% (USD 1.17 billion). They remain standard-of-care for >80% of CF patients due to chronic exocrine pancreatic insufficiency. Mucolytics Mucolytics contributed 8% (USD 848 million), driven by the sustained use of inhaled agents such as dornase alfa for airway clearance. By Route of Administration (Oral, Inhaled, Parenteral) Oral Oral therapies held 69% (USD 7.31 billion) due to the dominance of oral CFTR modulators. This segment will grow fastest through 2033 driven by life-long use and expanded mutation coverage. Inhaled Inhaled therapies contributed 26% (USD 2.76 billion). This includes inhaled mucolytics, bronchodilators, antibiotics, and other respiratory-support agents still widely used in multidisciplinary CF care. By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies) Hospital Pharmacies Hospital pharmacies captured 58% (USD 6.15 billion) as CF remains primarily managed through specialty centers that coordinate multidisciplinary treatment plans. Retail Pharmacies Retail pharmacies represented 31% (USD 3.28 billion), largely driven by the growth of chronic outpatient CF management and expanded insurance coverage. Online Pharmacies Online pharmacies accounted for 11% (USD 1.17 billion) and are increasing rapidly due to home delivery convenience and improved digital prescription services for chronic diseases. Buy This Report with Year-End Offer ( Buy 1 report: Get 30% OFF | Buy 2 reports: Get 50% OFF each! Limited time offer): Regional Outlook: United States: Largest and Fastest-Expanding CF Therapeutics Market The U.S. accounted for 54% of global CF drug sales (USD 5.73 billion in 2024). U.S. Market Highlights CF patient registry size exceeds 40,000, representing 25% of global cases CFTR modulator usage penetration surpasses 88% Government and private insurers expanded coverage for next-generation modulators by 20% YoY CF care center expansions increased pulmonary inpatient capacity by 11% By 2033, the U.S. market is forecast to exceed USD 19 billion. Japan: Rapidly Modernizing CF Treatment Landscape Japan accounted for 6% (USD 636 million) of global value in 2024. Japan Market Dynamics Recent improvements in genetic screening increased CF detection rates by 16% YoY Rising access to imported CFTR modulators and specialty respiratory therapies Hospital-based CF clinics increased multidisciplinary patient management capacity by 14% Japan is on track to surpass USD 2.1 billion by 2033 as availability and early diagnosis improve. Competitive Landscape: Below are revenues and cystic-fibrosis or related respiratory/orphan-drug segment contributions based on public annual reports and investor disclosures. Vertex Pharmaceuticals Incorporated 2023 Revenue: USD 9.87 billion CF Franchise Contribution: > USD 9.3 billion (94% of total company revenue) Vertex is the global leader in CFTR modulators including Trikafta/Kaftrio, Symdeko/Symkevi, and Orkambi. F. Hoffmann-La Roche Ltd 2023 Revenue: USD 66.5 billion Respiratory & Infectious Diseases Portfolio: USD 3.4+ billion Expanding CF pipeline with inhaled and anti-inflammatory therapies. Novartis Pharmaceuticals Corporation 2023 Revenue: USD 45.4 billion Immunology & Respiratory Division: USD 8.1 billion Active in inhaled antibiotic programs and airway clearance technologies. Teva Pharmaceuticals USA, Inc. 2023 Revenue: USD 15.8 billion Respiratory Portfolio Contribution: USD 1.7 billion Strong presence in inhalation therapies supporting CF symptom relief. Gilead Sciences, Inc. 2023 Revenue: USD 27.1 billion Antiviral & Anti-Infective Division: USD 5.8 billion Previously developed Cayston (inhaled aztreonam), widely used for chronic lung infections in CF patients. Buy This Report with Year-End Offer ( Buy 1 report: Get 30% OFF | Buy 2 reports: Get 50% OFF each! Limited time offer): The Decade Ahead: What will Define the Future of Cystic Fibrosis Therapeutics The Cystic Fibrosis market is on track for the fastest evolution in its history, driven by: Next-gen CFTR modulators targeting rare mutations Combination RNA therapies for non-responders Gene-editing and gene-replacement programs, particularly using CRISPR Inhaled biologics addressing persistent lung infection Improved lipid nanoparticle (LNP) delivery systems enabling organ-specific targeting Pediatric expansion increasing long-term treatment demand Real-world evidence integration improving precision dosing By 2033, cystic fibrosis care will be defined by personalized molecular therapies, multi-modality regimens, and potentially curative genetic treatments-setting the stage for the CF therapeutics market to become one of the most valuable rare-disease drug markets globally. Related Report: Duchenne Muscular Dystrophy Therapeutics Market Size to Surge from US$2.19 B in 2024 to US$6.64 B by 2033 - 13.2% CAGR. Marfan Syndrome Treatment Market Size to Grow from US$203.5 M in 2023 to US$312.6 M by 2031 - Driven by Rising Diagnosis & Advanced Therapeutic & Surgical Care. Huntington's Disease Market Size to Grow from US$456 M in 2024 to US$3.23 B by 2033 - >7.5% CAGR Signals Surge in Therapeutic & R&D Activity. Sickle Cell Anaemia Testing Market Share to Grow at 12% CAGR - From US$0.43 B in 2024 to Over US$1.5 B by 2032. About DataM Intelligence DataM Intelligence is a renowned provider of market research, delivering deep insights through pricing analysis, market share breakdowns, and competitive intelligence. The company specializes in strategic reports that guide businesses in high-growth sectors such as nutraceuticals and AI-driven health innovations. To find out more, visit or follow us on Twitter, LinkedIn, and Facebook. 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