Last update 28 Dec 2024
Vanzacaftor/Tezacaftor/Deutivacaftor
Last update 28 Dec 2024
Overview
Basic Info
Drug Type Small molecule drug |
Synonyms Vanza Triple, Vanzacaftor Triple, VX-121/TEZ/D-IVA + [5] |
Target |
Mechanism CFTR stimulants(Cystic fibrosis transmembrane conductance regulator stimulants) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseApproved |
First Approval Date US (20 Dec 2024), |
RegulationPriority Review (US), Fast Track (US), Orphan Drug (US), Orphan Drug (AU) |
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Structure
Molecular FormulaC26H27F3N2O6 |
InChIKeyMJUVRTYWUMPBTR-MRXNPFEDSA-N |
CAS Registry1152311-62-0 |
View All Structures (3)
Related
8
Clinical Trials associated with Vanzacaftor/Tezacaftor/DeutivacaftorA Phase 1, Open-label Taste Assessment Study of Vanzacaftor/Tezacaftor/Deutivacaftor in Healthy Adult Panelists
The purpose of this study is to evaluate the flavor (basic tastes, aroma, texture, mouthfeel) of VNZ/TEZ/D-IVA fixed dose combination (FDC) granules.
Start Date26 Mar 2024 |
Sponsor / Collaborator |
A Phase 1, Randomized, Open-Label, 2-part Study to Evaluate the Relative Bioavailability, Food Effect, and Dose Proportionality of a Granule Formulation of Vanzacaftor in Combination With Tezacaftor and Deutivacaftor in Healthy Adult Subjects
The purpose of this study is to evaluate the relative bioavailability, food effect, and dose proportionality of a granule formulation of VNZ/TEZ/D-IVA.
Start Date13 Mar 2024 |
Sponsor / Collaborator |
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year of Age and Older
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).
Start Date11 Aug 2023 |
Sponsor / Collaborator |
100 Clinical Results associated with Vanzacaftor/Tezacaftor/Deutivacaftor
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100 Translational Medicine associated with Vanzacaftor/Tezacaftor/Deutivacaftor
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100 Patents (Medical) associated with Vanzacaftor/Tezacaftor/Deutivacaftor
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24
News (Medical) associated with Vanzacaftor/Tezacaftor/Deutivacaftor22 Dec 2024
The FDA on Friday issued two simultaneous cystic fibrosis approvals for Vertex Pharmaceuticals but included high-level safety warnings for both.
The first approval is for Vertex’s “vanza triple” combination, a new product that will be branded as Alyftrek, for use in cystic fibrosis patients aged six years and older. Like some of Vertex’s older products, it is a modulator of the CFTR gene. Its list price is about $370,000 per year, according to
SEC documents
.
Vertex’s second approval is a label expansion for Trikafta, an older CFTR modulator that covers 94 new cystic fibrosis mutations. Alyftrek includes all mutations covered by Trikafta, as well as an additional 31 cystic fibrosis-causing mutations.
Both therapies will come with boxed warnings for potential drug-induced liver injury and failure. Previously, Trikafta came with a regular warning and precaution to watch for elevated liver enzymes, but the FDA elevated that to a boxed warning.
Alyftrek is made up of three different compounds: vanzacaftor, tezacaftor and deutivacaftor. The combo therapy
succeeded in its Phase 3 trials
earlier this year, and Vertex used a priority review voucher to reduce the FDA’s review time for Alyftrek to six months.
Trikafta brought in $9.8 billion in sales last year, and $7.5 billion through the first three quarters of 2024. It’s not yet clear how or if the new boxed warning will affect Trikafta sales going forward. Vertex is expected to release updated financial guidance during its fourth-quarter earnings call early next year.
Drug ApprovalPhase 3Priority ReviewClinical ResultAccelerated Approval
20 Dec 2024
- ALYFTREK™ is approved for patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies -
- In head-to-head clinical trials, ALYFTREK was non-inferior on ppFEV1 and further decreased sweat chloride compared to TRIKAFTA® -
BOSTON--(BUSINESS WIRE)--Dec. 20, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), a once-daily next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator for the treatment of cystic fibrosis (CF) in people 6 years and older who have at least one F508del mutation or another mutation in the CFTR gene that is responsive to ALYFTREK. See below for Important Safety Information, including a Boxed Warning.
“ALYFTREK is our fifth CFTR modulator to secure FDA approval and represents another significant milestone in our journey to serially innovate and to improve the lives of people living with cystic fibrosis,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “Our north star for more than 20 years has been to address the underlying cause of cystic fibrosis, treat more people with this disease, and bring more people to normal levels of CFTR function — ALYFTREK, with once-daily dosing, efficacy in 31 additional mutations, and lower sweat chloride levels than TRIKAFTA, is another step in achieving this goal.”
This approval is based on the most comprehensive Phase 3 pivotal program ever conducted in CF, including more than 1,000 patients across more than 20 countries and more than 200 sites. These data were previously released at the conclusion of the studies and presented at the North American Cystic Fibrosis Conference in September of this year. The Phase 3 studies in people with CF ages 12 years and older met their primary endpoint (non-inferiority on absolute change from baseline in ppFEV1 compared to TRIKAFTA) and all key secondary endpoints (including absolute change from baseline in sweat chloride [SwCl] compared to TRIKAFTA). In the Phase 3 study of children with CF ages 6-11 years, ALYFTREK demonstrated safety, the primary endpoint. Secondary endpoints, such as absolute change from baseline in ppFEV1 and absolute change from baseline in SwCl, were presented, supporting the benefit of ALYFTREK in this age group. ALYFTREK was generally well tolerated across all studies.
“In Phase 3 clinical trials, across a broad range of genotypes, once-daily ALYFTREK demonstrated non-inferiority to TRIKAFTA in ppFEV1 response and statistically significant improvement in SwCl, a welcomed advancement for the treatment of CF,” said Claire L. Keating, M.D., Co-Director of the Gunnar Esiason Adult Cystic Fibrosis and Lung Program at Columbia University and investigator in the ALYFTREK clinical trial program. “ALYFTREK has the potential to improve the care of patients with CF.”
ALYFTREK is the first, once-daily CFTR modulator. In a recent survey, approximately 75% of physicians reported that more convenient dosing is a very high unmet need for people with CF. Specifically, people with CF will have the added benefit from a once-daily dosing regimen, given the need to take CFTR modulators with fat-containing food. ALYFTREK also offers a potentially transformative option for approximately 150 people with CF in the U.S. with one of 31 mutations who are now eligible for a CFTR modulator for the first time.
ALYFTREK was also submitted to global health authorities and is under regulatory review in the European Union, the United Kingdom, Canada, Switzerland, Australia and New Zealand.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. 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 68,000 people with CF across more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.
ALYFTREK U.S. INDICATIONS
ALYFTREK is indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of at least one indicated mutation.
IMPORTANT SAFETY INFORMATION
BOXED WARNING: DRUG-INDUCED LIVER INJURY AND LIVER FAILURE
Elevated transaminases have been observed in patients treated with ALYFTREK. Cases of serious and potentially fatal drug-induced liver injury and liver failure were reported in patients taking a fixed-dose combination drug containing elexacaftor, tezacaftor, and ivacaftor, which contains 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 elexacaftor/tezacaftor/ivacaftor.
Assess liver function tests (ALT, AST, alkaline phosphatase, and bilirubin) in all patients prior to initiating ALYFTREK, 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 elevated liver function tests (LFTs) at baseline.
Interrupt ALYFTREK 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 ALYFTREK.
ALYFTREK should not be used in patients with severe hepatic impairment (Child-Pugh Class C). ALYFTREK is not recommended in patients with moderate hepatic impairment (Child-Pugh Class B) and should only be considered when there is a clear medical need, and benefit outweighs risk. If used, monitor patients closely.
WARNINGS AND PRECAUTIONS
Drug-Induced Liver Injury and Liver Failure
Elevated transaminases have been observed in patients treated with ALYFTREK. Cases of serious and potentially fatal drug-induced liver injury and liver failure have been reported in patients with and without a history of liver disease taking a fixed-dose combination drug containing elexacaftor, tezacaftor, and ivacaftor (ELX/TEZ/IVA), which contains 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 ELX/TEZ/IVA Assess LFTs (ALT, AST, alkaline phosphatase, and bilirubin) in all patients prior to initiating ALYFTREK, 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 in patients with a history of liver disease, elevated LFTs at baseline, or a history of elevated LFTs with drugs containing ELX, TEZ, and/or IVA Interrupt ALYFTREK 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 or normal (ULN) or ALT or AST >3x ULN with bilirubin >2x ULN) Clinical signs or 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 ALYFTREK with close monitoring ALYFTREK should not be used in patients with severe hepatic impairment. ALYFTREK is 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 used, monitor patients closely
Significant elevations in LFTs (e.g., ALT or AST >5x the upper limit or normal (ULN) or ALT or AST >3x ULN with bilirubin >2x ULN) Clinical signs or 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 anaphylaxis, have been reported in the postmarketing setting of drugs containing ELX, TEZ, and/or IVA (same or similar active ingredients in ALYFTREK). If signs or symptoms of serious hypersensitivity reactions develop during ALYFTREK treatment, discontinue ALYFTREK and institute appropriate therapy. Consider benefits and risks for the individual patient to determine whether to resume ALYFTREK
Patients Who Discontinued or Interrupted Elexacaftor-, Tezacaftor-, or Ivacaftor-Containing Drugs Due to Adverse Reactions
There are no available safety data for ALYFTREK in patients who previously discontinued or interrupted treatment with drugs containing ELX/TEZ/IVAr 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
Reduced Effectiveness with Concomitant Use With CYP3A Inducers
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
Adverse Reactions with Concomitant Use With CYP3A Inhibitors
Following concomitant use of strong or moderate CYP3A inhibitors with ALYFTREK, exposures of vanzacaftor, tezacaftor, and deutivacaftor were increased, which may increase the risk of adverse reactions associated with ALYFTREK. Reduce the ALYFTREK dosage with concomitant use of strong or moderate CYP3A inhibitors
Cataracts
Non-congenital lens opacities have been reported in pediatric patients treated with drugs containing ivacaftor (similar to an active ingredient in ALYFTREK). Baseline and follow-up ophthalmological examinations are recommended in pediatric patients treated with ALYFTREK
ADVERSE REACTIONS
Serious Adverse Reactions
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%)
Most Common Adverse Reactions
The most common adverse reactions to ALYFTREK (≥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
USE IN SPECIFIC POPULATIONS
Pediatric Use
The safety and effectiveness of ALYFTREK in patients <6 years of age have not been established
Please click here to see the full U.S. Prescribing Information, including Boxed WARNING for ALYFTREK.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. 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 acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes 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 15 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 Reshma Kewalramani, M.D., and Claire L. Keating, M.D., in this press release, statements regarding the eligible patient population for ALYFTREK, expectations for ALYFTREK regulatory submissions to global health authorities, and statements regarding the potential benefits of ALYFTREK, including for patients eligible for a CFTR modulator for the first time. 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 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/20241220133127/en/
Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com Susie Lisa, CFA: +1 617-341-6108 or Manisha Pai: +1 617-961-1899
Media: mediainfo@vrtx.com or U.S.: 617-341-6992 Heather Nichols: +1 617-839-3607 or International: +44 20 3204 5275
Source: Vertex Pharmaceuticals Incorporated
Clinical ResultPhase 3Drug Approval
05 Nov 2024
Shares in Vertex rose following the lifted guidance for 2024. Image credit: Shutterstock / JHVEPhoto.
Vertex Pharmaceuticals
has seen a 12% revenue uptick in Q3 compared to the same period in 2023 as it prepares launches for two new separate therapies in cystic fibrosis and pain.
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Reporting on its Q3 financials this year, Vertex said its full-year product revenue rose from $10.65bn-$10.85bn to $10.8bn-$10.9bn.
The 12% revenue increase has notched sales up to $2.77bn, as per a 4 November press release.
Shares in the US company closed 0.35% higher on 4 November compared to the previous close pre-announcement. Shares rose again by 2.11% at market open on 5 November compared to the previous market close. Vertex has a market cap of $122bn.
Vertex’s lead cystic fibrosis treatment Trikafta (elexacaftor / tezacaftor / ivacaftor) was once again the primary driver behind the drugmaker’s positive financial performance. The triple-combination therapy generated nearly $2.6bn in Q3 sales, compared to $2.27bn made in the same period last year.
The pharma company could soon be bolstering its cystic fibrosis portfolio in the form of a once-daily vanzacaftor triple therapy (vanzacaftor / tezacaftor / deutivacaftor). The treatment has a Prescription Drug User Fee Act (PDUFA) target action date of January 2025 in the US, with the US Food and Drug Administration (FDA) also granting it priority review. Vertex added it has also completed regulatory submissions for the vanzacaftor triple therapy in the EU, UK, and Canada, among others.
In development is an mRNA therapy for cystic fibrosis, with Vertex stating it has completed a single ascending dose (SAD) portion of a Phase I/II study evaluating the candidate named VX-522.
Vertex also has another PDUFA in January 2025 as the vanzacaftor triple therapy, with the
FDA set to review suzetrigine
for the treatment of moderate-to-severe pain. Suzetrigine is estimated to reach blockbuster status by 2029 and achieve $1.4bn in sales by 2030, according to an analysis by GlobalData’s Pharma Intelligence Centre.
GlobalData is the parent company of
Pharmaceutical Technology
.
Vertex also issued an update for its cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) cell therapy Casgevy (exagamglogene autotemcel). The company has now activated 45 treatment centres around the world for the therapy, which had its revenue from the first infused patient included in the Q3 financials. In August, the UK’s NHS
rolled out the treatment
following a positive recommendation by the National Institute for Health and Care Excellence (NICE), which Vertex said impacted the guidance change.
Also notching up the guidance was Vertex’s acquisition of
Alpine Immune Sciences
earlier this year, a
deal that saw the drugmaker spend $4.9bn
to bolster its immunotherapy pipeline.
Vertex CEO Reshma Kewalramani said: “The third quarter marked another period of strong progress […] and we are again increasing our full-year product revenue guidance. Launch preparedness is well underway as we look forward to the potential approvals of the vanzacaftor triple for cystic fibrosis and suzetrigine.”
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AcquisitionCell TherapyImmunotherapy
100 Deals associated with Vanzacaftor/Tezacaftor/Deutivacaftor
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R&D Status
10 top approved records. to view more data
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| Indication | Country/Location | Organization | Date |
|---|---|---|---|
| Cystic Fibrosis | US | 20 Dec 2024 |
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Clinical Result
Clinical Result
Indication
Phase
Evaluation
View All Results
| Study | Phase | Population | Analyzed Enrollment | Group | Results | Evaluation | Publication Date |
|---|
Phase 3 | 971 | (Trial 1, NCT05033080) | btmgmuqnbq(rpdjuupxgm) = adbbepuazp cblezktznh (fikifmlwle, 0.3) View more | Positive | 20 Dec 2024 | ||
(Trial 1, NCT05033080) | btmgmuqnbq(rpdjuupxgm) = uflebzhhte cblezktznh (fikifmlwle, 0.3) View more | ||||||
Phase 3 | 597 | (ELX/TEZ/IVA) | jzrpgarkbs(bjrgpspkqe) = ucjmqiettp fzzqskqrbe (qksozxvsmf, otcmpjjsyf - roanjtwltb) View more | - | 13 Jun 2024 | ||
Placebo (matched to ELX/TEZ/IVA)+VX-121/TEZ/D-IVA (VX-121/TEZ/D-IVA) | jzrpgarkbs(bjrgpspkqe) = uvzpdfvoec fzzqskqrbe (qksozxvsmf, pgnsbiooqx - twwwspraau) View more | ||||||
RIDGELINE 105 (NEWS) Manual  | Phase 3 | 78 | Vanzacaftor/Tezacaftor/Deutivacaftor | asukrwmxhj(dhwaudiwrz) = haukwfgpqk jbrlgajrrk (fgycmxgihi, -2.0 to 1.9) View more | Positive | 06 Feb 2024 | |
SKYLINE 103 (NEWS) Manual | Phase 3 | 971 | Vanzacaftor/tezacaftor/deutivacaftor (SKYLINE 102) | kloorostwf(kglftjtjuk): DIFFERENCE = 0.2 (95% CI, -0.7 to 1.1), P-Value = <0.0001 View more | Positive | 06 Feb 2024 | |
TRIKAFTA (SKYLINE 102) |
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