Vertex Pharmaceuticals Incorporated operates as a global biotechnology company. The company invests in scientific innovation to create transformative medicines for people with serious diseases, with a focus on specialty markets.
The company has seven approved medicines: five that treat the underlying cause of cystic fibrosis (CF), a life-threatening genetic disease, one that treats severe sickle cell disease (SCD) and transfusion dependent beta thalassemia (TDT), life shortening inherited bloo...
Vertex Pharmaceuticals Incorporated operates as a global biotechnology company. The company invests in scientific innovation to create transformative medicines for people with serious diseases, with a focus on specialty markets.
The company has seven approved medicines: five that treat the underlying cause of cystic fibrosis (CF), a life-threatening genetic disease, one that treats severe sickle cell disease (SCD) and transfusion dependent beta thalassemia (TDT), life shortening inherited blood disorders, and one that treats moderate-to-severe acute pain. The company’s clinical-stage pipeline includes programs in CF, SCD, beta thalassemia, acute and peripheral neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy and other autoimmune renal diseases and cytopenias, type 1 diabetes, myotonic dystrophy type 1, and autosomal dominant polycystic kidney disease.
The company’s marketed CF medicines are ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), which was approved by the U.S. Food and Drug Administration (the “FDA”) in December 2024, TRIKAFTA/KAFTRIO (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO/SYMKEVI (tezacaftor/ivacaftor and ivacaftor), ORKAMBI (lumacaftor/ivacaftor) and KALYDECO (ivacaftor).
The company has received approval to market CASGEVY (exagamglogene autotemcel), a gene-therapy, for the treatment of SCD or TDT in the United States (U.S.), the European Union (E.U.), the United Kingdom (U.K.), the Kingdom of Saudi Arabia, the Kingdom of Bahrain, the United Arab Emirates, Canada and Switzerland. CASGEVY was initially approved by the FDA in December 2023.
In January 2025, the FDA approved JOURNAVX (suzetrigine), the company’s selective non-opioid NaV1.8 pain signal inhibitor, for the treatment of moderate-to-severe acute pain.
In December 2024, the U.S. Food and Drug Administration (the FDA) approved ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), the company’s fifth medicine for people with CF. In addition to ALYFTREK, its marketed medicines that treat people with CF are TRIKAFTA/KAFTRIO (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO/SYMKEVI (tezacaftor/ivacaftor and ivacaftor), ORKAMBI (lumacaftor/ivacaftor) and KALYDECO (ivacaftor). Collectively, the company’s five marketed CF medicines are being used to treat nearly three quarters of the approximately 94,000 people with CF in the U.S., Europe, Australia, and Canada.
Through approval of new medicines, label expansions, and expanded reimbursement, the company focuses on increasing the number of people with CF who are eligible and able to receive its medicines. The company is evaluating its medicines in additional patient populations, including younger children, with the goal of having small molecule treatments for all people who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to its CFTR modulators. In December 2024, the FDA approved the expanded use of TRIKAFTA for the treatment of people with CF. With this approval, 94 additional non-F508del CFTR mutations have been added to the TRIKAFTA label. The company is evaluating VX-522, an investigational messenger ribonucleic acid (mRNA) therapeutic that it is developing in collaboration with Moderna, Inc. (Moderna) in a Phase 1/2 clinical trial in people with CF. The company is continuing its research and development of additional CFTR modulators, with the aim of developing best-in-class medicines that can help more patients achieve normal levels of CFTR function, and it is investigating additional potential treatments for people with CF who do not make full-length CFTR protein and cannot benefit from CFTR modulators.
The company’s marketed therapy is CASGEVY (exagamglogene autotemcel), an ex-vivo, non-viral CRISPR/Cas9 gene-edited cell therapy, which has been approved in the United States (U.S.), the European Union (E.U.), the United Kingdom (U.K.), the Kingdom of Saudi Arabia (Saudi Arabia), the Kingdom of Bahrain (Bahrain), the United Arab Emirates (the UAE), Canada and Switzerland for the treatment of people 12 years of age and older with SCD or TDT. The company estimates approximately 60,000 people with severe SCD or TDT are or could become eligible for CASGEVY in the U.S., Canada, Europe, Saudi Arabia and Bahrain. The company is evaluating CASGEVY as a treatment for children 5 to 11 years of age with SCD or TDT in global Phase 3 clinical trials. The company is progressing preclinical assets for gentler conditioning for CASGEVY, which could broaden the eligible patient population, and investigating small molecules for the potential treatment of SCD and TDT.
In January 2025, the FDA approved JOURNAVX (suzetrigine), the copany’s selective non-opioid NaV1.8 pain signal inhibitor, for the treatment of moderate-to-severe acute pain. The company has begun its commercial launch of JOURNAVX in eligible adults in the U.S. In addition, it is enrolling and dosing patients in a Phase 3 clinical trial evaluating suzetrigine for the treatment of diabetic peripheral neuropathy, a common form of peripheral neuropathic pain. The company is enrolling and dosing patients in two Phase 2 clinical trials evaluating VX-993, a next-generation selective NaV1.8 pain signal inhibitor, for the treatment of acute pain and for the treatment of diabetic peripheral neuropathy. In December 2024, the company announced results from a Phase 2 clinical trial evaluating suzetrigine in people with lumbosacral radiculopathy (LSR).
Beyond CF, SCD, TDT and pain, the company is advancing programs across multiple disease areas and modalities, including:
APOL1-Mediated Kidney Disease: The company is evaluating inaxaplin, its small molecule for the treatment of APOL1-mediated kidney disease (AMKD). The company continues to enroll and dose people with AMKD in the Phase 3 portion of the global Phase 2/3 clinical trial.
IgA Nephropathy: The company is eveloping povetacicept, a dual inhibitor of the B cell activating factor (BAFF) and a proliferation-inducing ligand (APRIL) pathways, as a potentially best-in-class approach to treat IgA nephropathy (IgAN), a serious progressive, autoimmune kidney disease that can lead to end-stage renal disease. The company isenrolling and dosing patients in the Phase 3 clinical trial evaluating povetacicept in people with IgAN.
Type 1 Diabetes: Zimislecel is an allogeneic stem-cell derived, fully differentiated islet cell therapy in pivotal development for the treatment of type 1 diabetes (T1D). The company expects to complete enrollment and dosing in the Phase 3 portion of this Phase 1/2/3 clinical trial in 2025. The company’s second clinical program in T1D, VX-264, in which zimislecel is encapsulated in an immunoprotective device, is ongoing. The company is also pursuing alternative approaches to immunosuppression that could be used with zimislecel, as well as hypoimmune cells.
Myotonic Dystrophy Type 1: The company is exploring multiple approaches to address the underlying causal biology for myotonic dystrophy type 1 (DM1), including small molecules. The company completed the single ascending dose portion of the global Phase 1/2 clinical trial evaluating VX-670, an oligonucleotide-based approach that the company has in-licensed from Entrada Therapeutics, Inc. (Entrada). The company is enrolling and dosing the multiple ascending dose portion of the trial, which will evaluate the safety and efficacy of VX-670.
Autosomal Dominant Polycystic Kidney Disease: The company is nearing completion of a Phase 1 clinical trial in healthy volunteers evaluating VX-407, its first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in people with a subset of variants in the PKD1 gene. The company expects to advance VX-407 into a Phase 2 proof-of-concept study in people with ADPKD in 2025.
In addition to the programs, the company has additional research programs aimed at diseases that fit its research and development strategy and follow-on programs in its existing disease areas in accord with its serial innovation approach.
The ocmpany’s core strategy is to discover and develop innovative medicines by combining transformative advances in the understanding of human disease and the science of therapeutics to dramatically advance human health. That strategy focuses on validated targets that address causal human biology, predictive lab assays and clinical biomarkers, rapid paths to registration and approval, and product candidates that hold the potential for transformative patient benefit. The company’s approach includes advancing multiple compounds or therapies from each program into early clinical trials to obtain patient data that can inform selection of the most promising therapies for later stage development as well as inform its ongoing discovery and development effort. The company plans to continue investing to advance its strategy, fostering scientific innovation by identifying additional product candidates through internal research efforts, and investing in business development transactions to access emerging technologies, products and product candidates.
The company is working to ensure broad access for eligible patients with these conditions in all countries with regulatory approval. As the company continues to invest in its serial innovation strategy, launch new products, advance its diverse pipeline, and expand geographically.
Marketed Products
CF
The company’s CF medicines are collectively being used by nearly three quarters of the approximately 94,000 people with CF in the U.S., Europe, Australia, and Canada. Additionally, the company continues to secure formal reimbursement in multiple additional countries that collectively comprise approximately 15,000 additional people with CF. Approximately 10,000 of those additional people with CF are eligible for treatment with CFTR modulators.
The company’s CFTR regimens target the underlying cause of disease and have been shown to improve CFTR protein function in people with CF, and as such have been shown to provide transformative benefit for people living with CF. The company’s CF medicines are used by patients in over 60 countries, and TRIKAFTA/KAFTRIO is approved and reimbursed or accessible in more than 50 of these countries. The company continues to increase the number of patients eligible and able to receive its medicines through approvals of new medicines, label expansions and expanded reimbursement. In December 2024, the FDA approved the expanded use of TRIKAFTA for the treatment of people with CF 2 years of age and older who have at least one F508del mutation in the CFTR gene or a mutation that is responsive to TRIKAFTA based on clinical and/or in vitro data.
Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
CASGEVY, the company’s therapy for SCD and TDT, is approved in the U.S., the E.U., the U.K., Saudi Arabia, Bahrain, the UAE, Canada and Switzerland. SCD and TDT are hemoglobinopathies, a group of inherited blood disorders that result from gene mutations that alter hemoglobin, a protein in red blood cells that delivers oxygen throughout the body. The company estimates approximately 60,000 people with severe SCD or TDT are or could become eligible for CASGEVY in the U.S., Canada, Europe, Saudi Arabia and Bahrain.
CASGEVY, the company’s ex-vivo, non-viral CRISPR/Cas9-based gene-editing therapy, was developed for the treatment of severe SCD and TDT, with its collaborator, CRISPR Therapeutics AG (CRISPR). Patients first undergo a treatment at an authorized treatment center (ATC) that mobilizes a population of hematopoietic stem and progenitor cells (HSPC) from the bone marrow into the bloodstream. These cells are collected from the patient’s bloodstream and transferred to a manufacturing facility where the HSPCs are purified and CRISPR/Cas9 gene-editing is performed.
The company’s global launch strategy for CASGEVY continues to focus on countries with high unmet medical need and infrastructure to support treatment with this CRISPR/Cas9-based gene-editing therapy, including the U.S., the Middle East, and major markets in Europe. The company is working with ATCs to enable patient initiation, supporting the patient journey through infusion with CASGEVY, and working with payors to secure broad and equitable access for patients. With more than 50 ATCs activated globally, its strategy is focused on activation of a global ATC network to treat patients and expansion of its globally-enabled manufacturing and supply chain to meet growing patient demand. The company’s teams are also focused on working to educate patients, physicians and policymakers on the treatment journey and CASGEVY clinical data, where appropriate.
Acute Pain
In January 2025, the FDA approved JOURNAVX for the treatment of moderate-to-severe acute pain in adults. The company’s launch strategy for JOURNAVX focuses on securing broad access for people with acute pain, educating patients and physicians on the clinical profile of JOURNAVX, and making investments to provide a seamless treatment experience for physicians and people with acute pain. The company is engaging with payors and formulary decision makers to secure reimbursement and access to JOURNAVX. The company is working to secure national retail distribution to facilitate broad availability of JOURNAVX as well as financial and co-pay assistance programs for patients that will support access to JOURNAVX.
Research and Development Programs
The company’s development-stage product candidates are focused on the treatment of serious diseases, including CF, SCD, TDT, acute and peripheral neuropathic pain, AMKD, IgAN and other autoimmune renal diseases and cytopenias, T1D, DM1, and ADPKD. In pursuit of serial innovation, its research and development approach includes advancing multiple candidates into clinical trials and pursuing multiple modalities with the goal of bringing first-in-class and/or best-in-class therapies to patients.
The company’s research and development strategy has been validated through its success in moving novel product candidates into clinical trials and obtaining marketing approvals for ALYFTREK, TRIKAFTA/KAFTRIO, KALYDECO, ORKAMBI, and SYMDEKO/SYMKEVI for the treatment of CF, CASGEVY for the treatment of SCD and TDT, and JOURNAVX for the treatment of acute pain. The company’s approach to drug discovery has been further validated by its successful demonstration of clinical proof-of-concept in four additional disease areas: in diabetic peripheral neuropathy with its NaV1.8 inhibitors, in AMKD with inaxaplin, in T1D with zimislecel, and in IgAN with povetacicept.
To augment its internal programs, the company acquires businesses and technologies and collaborate with biopharmaceutical and technology companies, leading academic research institutions, government laboratories, foundations and other organizations to advance research in its disease areas of interest, as well as to access technologies needed to execute on its strategy. The company’s internal and external innovation approaches are based on the same strategy, which enables the company to effectively integrate and execute on new internal capabilities as the company invests in external innovation. The company’s investments in external innovation include its collaboration with CRISPR, which resulted in the successful development and approval of CASGEVY and the establishment and advancement of other pipeline programs, including its T1D program through its acquisition of Semma Therapeutics, Inc. (Semma), its expansion of its renal programs through its acquisition of Alpine Immune Sciences, Inc. (Alpine), its mRNA therapeutic, VX-522, for the treatment of CF through its collaboration with Moderna, and its intracellular therapeutics for DM1, including VX-670, through its collaboration with Entrada.
CF
The company is enrolling and dosing in a Phase 3 clinical trial evaluating ALYFTREK in children with CF 2 to 5 years of age who have at least one F508del mutation or a mutation responsive to triple combination CFTR modulators.
The company estimates that approximately 90% of people with CF could benefit from its five approved medicines, including TRIKAFTA/KAFTRIO and the recently approved ALYFTREK. The company continues to identify and develop additional CFTR modulators with the goal of developing best-in-class medicines that can help more patients who respond to CFTR modulators achieve normal levels of CFTR function; its next generation of CFTR modulators have completed, or are in the process of completing, Phase 1 clinical trials.
In order to treat people with CF who do not make full-length CFTR protein, and as a result, cannot benefit from its CFTR modulators, the company is researching and developing genetic therapies, such as mRNA, and gene-editing approaches to CF. In collaboration with Moderna, the company is developing VX-522, a nebulized CF mRNA therapeutic designed to treat the underlying cause of CF lung disease for these people by enabling cells in the lungs to produce functional CFTR protein. The multiple ascending dose portion of the clinical trial is ongoing and the company expects data in the first half of 2025.
Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
The company has completed enrollment in these two clinical trials, and the company expects to complete dosing in 2025.
In connection with its serial innovation approach, the company is advancing preclinical assets for myeloablative conditioning agents with improved tolerability profiles. The company is also investigating in vivo gene-editing approaches and small molecules for the potential treatment of SCD and TDT.
Pain
In acute pain, the company is evaluating an oral formulation of VX-993, its next-generation NaV1.8 pain signal inhibitor, for the treatment of moderate-to-severe acute pain following bunionectomy surgery in a Phase 2 clinical trial. The company is also enrolling and dosing healthy volunteers in a Phase 1 clinical trial evaluating an intravenous formulation of VX-993. The FDA has granted Fast Track Designation to VX-993 in acute pain in both the oral and intravenous formulations.
In neuropathic pain, the company is enrolling and dosing in the Phase 3 pivotal program evaluating suzetrigine in people with diabetic peripheral neuropathy, a common form of peripheral neuropathic pain, with the ultimate goal of securing a broad label for the treatment of peripheral neuropathic pain. In addition, the company is enrolling and dosing patients in a Phase 2 clinical trial for the oral formulation of VX-993, for the treatment of diabetic peripheral neuropathy.
In December 2024, the company announced Phase 2 clinical trial results showing that treatment with suzetrigine demonstrated a statistically significant and clinically meaningful within-group reduction in pain on the numeric pain rating scale for people with LSR, and that suzetrigine was safe and generally well-tolerated in the trial. The clinical trial also included a placebo reference arm, which showed a similar within-group reduction. The company hypothesizes that a high placebo response in this clinical trial led to a lack of separation of the suzetrigine and placebo response curves.
In keeping with its serial innovation approach, the company is advancing multiple additional NaV1.8 inhibitors and NaV1.7 inhibitors, which could be used alone or in combination, for the treatment of acute pain and chronic neuropathic pain.
APOL1-Mediated Kidney Disease
The company is evaluating multiple novel small molecules that inhibit the function of the mutant APOL1 protein with the potential to treat APOL1-mediated kidney disease. Based on positive Phase 2 data, the company initiated pivotal development of inaxaplin in a single Phase 2/3 adaptive clinical trial in patients with AMKD in 2022. The company completed the Phase 2B portion of the Phase 2/3 clinical trial and initiated the Phase 3 portion of the study in 2024. The company continues to enroll and dose people with AMKD in the Phase 3 portion of the global Phase 2/3 pivotal clinical trial (AMPLITUDE). The company expects to complete enrollment in the interim analysis cohort in 2025 and apply for potential accelerated approval in the U.S., assuming a positive interim analysis. In addition, the company has initiated a Phase 2 proof-of-concept clinical trial (AMPLIFIED) evaluating inaxaplin as a treatment for people with AMKD.
IgA Nephropathy and Other B Cell-Mediated Diseases
In 2024, the company acquired Alpine and its lead asset, povetacicept, which has the potential of being a pipeline-in-a-product. In 2024, the company initiated RAINIER, its global Phase 3 pivotal trial of povetacicept versus placebo in approximately 480 people with IgAN, which is enrolling and dosing people in the U.S., Europe and Asia. The company expects to complete enrollment in the interim analysis cohort in 2025 and apply for potential accelerated approval in the U.S., assuming a positive interim analysis.
In January 2025, the company entered into a collaboration agreement with Zai Lab Limited (Zai) for the development and commercialization of povetacicept in mainland China, Hong Kong SAR, Macau SAR, Taiwan region and Singapore. Under this collaboration, Zai will help advance the povetacicept clinical trials and make the regulatory submissions in the licensed territories. Zai will also be responsible for commercialization activities in the licensed territories, if povetacicept becomes an approved product.
In addition to IgAN, the company is evaluating povetacicept as a treatment for other B cell-mediated autoimmune kidney diseases in the RUBY-3 basket trial and as a treatment for autoimmune cytopenias in the RUBY-4 basket trial. Both of these basket trials are ongoing, and the company expects data in some of these conditions in 2025.
Type 1 Diabetes
The company is developing non-autologous (allogeneic) fully differentiated, stem-cell derived islet cell therapies designed to replace insulin-producing islet cells that are destroyed in people with T1D, with the goal of delivering a functional cure. The company is pursuing multiple programs for the transplant of functional islets into patients, including: transplantation of islet cells alone followed by standard, chronic immunosuppression to protect the implanted cells, implantation of the islet cells inside a novel immunoprotective device, alternative approaches to immunosuppression that could be used with the implanted islet cells, and development of hypoimmune islet cells to optimize protection of the implanted islet cells from the immune system.
Zimislecel, the company’s first program, is a stem cell-derived, allogeneic, fully differentiated, insulin-producing islet cell replacement therapy, using standard immunosuppression to protect the implanted cells. The Phase 1/2/3 clinical trial is designed as a sequential, multi-part clinical trial to evaluate the safety and efficacy of zimislecel, and has been expanded to include a total of 50 people. The company expects to complete enrollment and dosing in the Phase 3 portion of this clinical trial in 2025 and, assuming positive data, the company expects to file for approval after patients have completed one year of insulin-free follow-up. Data presented demonstrate unprecedented efficacy and curative potential. Safety data are consistent with the immunosuppressives, the perioperative period, and past medical history. In addition, the company has initiated a clinical trial evaluating zimislecel in people with T1D who have had a kidney transplant.
In its second program, the company is evaluating VX-264, in which zimislecel is encapsulated in an immunoprotective device that is implanted in patients. The company is enrolling and dosing patients with VX-264 in a Phase 1/2 clinical trial to evaluate the safety, tolerability and efficacy of VX-264. Part A of the study dosed patients with a partial dose of cells/devices and a stagger between patients, Part B dosed patients with a full target dose and a stagger between patients, and Part C will dose at full target dose without a stagger between patients. The company expects to share Part B full-dose data from this clinical trial in 2025.
In its third program, research is directed toward developing hypoimmune cells by gene-editing zimislecel prior to differentiation into fully differentiated islets in order to cloak them from the immune system. This program continues to advance in pre-clinical development. The company is also pursuing alternative approaches to immunosuppression that could be used with zimislecel.
Myotonic Dystrophy Type 1
VX-670, its lead approach for DM1, holds the potential to address the underlying cause of DM1. VX-670 is an oligonucleotide connected to a cyclic peptide to promote effective delivery into cells. The company completed the single ascending dose portion of the global Phase 1/2 clinical trial evaluating the first candidate, VX-670, in patients with DM1. The company is enrolling and dosing the multiple ascending dose portion of the trial, which will evaluate the safety and efficacy of VX-670. The company also has a small molecule program for DM1 in preclinical development.
Autosomal Dominant Polycystic Kidney Disease
VX-407 is a first-in-class small molecule corrector that is designed to target the underlying cause of ADPKD in people with a subset of PKD1 variants. The company is nearing completion of a Phase 1 clinical trial in healthy volunteers evaluating VX-407 and expect to advance VX-407 into a Phase 2 proof-of-concept clinical trial in people with ADPKD in 2025.
Commercialization of Medicines
The company’s commercialization efforts focus on supporting the appropriate use of ALYFTREK, TRIKAFTA/KAFTRIO, SYMDEKO/SYMKEVI, ORKAMBI, KALYDECO, CASGEVY and JOURNAVX in the markets where these products have been approved. The company’s teams are responsible for promoting products to health care providers, ensuring its products are distributed effectively, ensuring the safe use of its products, and obtaining reimbursement for its products from third-party payors, including governmental organizations in the U.S. and ex-U.S. markets. In parallel, its government affairs and public policy group advocates for policies that promote life sciences innovation and increase awareness of the diseases on which the company is focusing with state and federal legislatures, government agencies, public health officials and other policymakers.
Commercialization of CF Medicines
In the U.S., the company primarily sells its CF products to a limited number of specialty pharmacy and specialty distributors. In international markets, the company sells its CF products primarily through distributor arrangements and to retail pharmacies, as well as to hospitals and clinics, many of which are government-owned or supported. The company also has established programs in the U.S. that provide its CF products to qualified uninsured or underinsured patients at no charge or at a reduced charge, based on specific eligibility criteria.
The company’s U.S. field-based CF commercial team consists of a small number of individuals to support commercialization of its medicines for CF. The company focuses its CF marketing efforts in the U.S. on a relatively small number of physicians and health care professionals who write most of the prescriptions for CF medicines. Many of these physicians and health care professionals are located at a limited number of accredited centers in the U.S. focused on the treatment of CF. In international markets, the company or its distributors has small sales forces that support TRIKAFTA/KAFTRIO, SYMDEKO/SYMKEVI, ORKAMBI and KALYDECO in jurisdictions where these products are approved.
The company is supporting the launch of ALYFTREK through its existing commercial infrastructure.
ALYFTREK is under regulatory review in the U.K., the E.U., Canada, Switzerland, Australia and New Zealand.
Commercialization of CASGEVY
The company is continuing the commercial launch of CASGEVY, following approvals in the U.S., the E.U., the U.K., Saudi Arabia, Bahrain, the UAE, Canada and Switzerland. The company expects to obtain additional regulatory approvals for CASGEVY in 2025. The company estimates approximately 60,000 people with severe SCD and TDT are or could become eligible for CASGEVY in the U.S., Canada, Europe, Saudi Arabia and Bahrain.
In the Middle East, the company has reached a hospital-based reimbursement for people with SCD or TDT in Saudi Arabia, and for national reimbursement for eligible people in Bahrain. The company is actively working to expand access to CASGEVY across Europe and the Middle East.
The company is treating patients across multiple geographies and more than 50 patients have initiated cell collection. The first commercial patient was infused with CASGEVY in the third quarter of 2024.
Commercialization of JOURNAVX
In January 2025, JOURNAVX was approved by the FDA as a treatment for adults with moderate-to-severe acute pain. The company’s commercial focus at launch for JOURNAVX is securing broad access and investing to ensure a seamless experience for patients and physicians. The company is engaging with key health care professionals, formulary decision makers and payers to establish the conditions for rapid patient access and uptake. The company’s commercial field team is engaging stakeholders to support the launch of JOURNAVX in hospitals and other healthcare settings in the U.S. In addition, the company is working to secure broad stocking agreements with national retail pharmacies and regional chains, to ensure access to JOURNAVX for patients across the U.S.
The company focuses on ensuring there is equal access to non-opioid options for pain relief. The company continues to see momentum from federal and state policymakers to support the appropriate use of non-opioid pain medications, including addressing financial and other administrative barriers, such as utilization management, to accessing non-opioid options. In addition, as a part of its commitment to patients, the company offers financial and copay support programs for eligible patients who are prescribed JOURNAVX.
Reimbursement of Approved Therapies
The company’s CF medicines are used by patients in more than 60 countries, and TRIKAFTA/KAFTRIO is reimbursed or accessible in more than 50 of these countries outside the U.S. The company expects to continue to focus significant resources to maintain reimbursement and obtain expanded reimbursement for its CF medicines and pipeline therapies in ex-U.S. markets.
For CASGEVY in ex-U.S. markets, the company anticipates some early access programs initially and in parallel the company is pursuing long-term reimbursement arrangements. The company has successfully achieved national reimbursement in England, Luxembourg, and Bahrain for people with TDT or SCD, and secured hospital-based reimbursement for patients in Saudi Arabia and Austria. The company continues to engage with payors in the U.K., the E.U., and the Middle East.
For JOURNAVX in the U.S., the company has been working with government and commercial payors pre- and post-approval to support rapid and broad access, including to accelerate targeted contracting and formulary additions at both select payors and targeted hospital systems. In addition, the company is finalizing contracts with private and government payors, as well as hospital group purchasing organizations.
Strategic Transactions
In May 2024, the company acquired Alpine. Alpine’s lead molecule, povetacicept, is a highly potent and effective dual antagonist of BAFF and APRIL. The company is evaluating povetacicept for the potential treatment of IgAN in a Phase 3 clinical trial and for the treatment of other serious autoimmune diseases in Phase 2 clinical trials.
Collaboration and Licensing Arrangements
Joint Development and Commercialization Agreement with CRISPR
In December 2017, the company entered into a joint development and commercialization agreement (Original JDCA) with CRISPR, pursuant to which its is co-developing and co-commercializing CASGEVY for SCD and TDT. The company entered into the Original JDCA following its exercise of an option to co-develop and co-commercialize the hemoglobinopathies program that was contained in the collaboration agreement that the company entered into with CRISPR in 2015.
Pursuant to the A&R JDCA, the company lead global development, manufacturing and commercialization of CASGEVY, with support from CRISPR. Subject to the terms and conditions of the A&R JDCA, the company has the right to conduct all research, development, manufacturing, and commercialization activities relating to the product candidates and products under the A&R JDCA (including CASGEVY) throughout the world, subject to CRISPR’s reserved right to conduct certain activities.
In-License Agreements
The company’s in-license agreements include:
Moderna, Inc.: In 2016, the company entered into a collaboration with Moderna for the identification and development of mRNA therapeutics encoding CFTR for the treatment of CF. In December 2022, the FDA cleared its Investigational New Drug Application (IND) for VX-522, an mRNA therapeutic the company is developing with Moderna pursuant to this collaboration. The multiple ascending dose portion of the Phase 1/2 clinical trial of VX-522 in people with CF is ongoing. The company expects to share data from this clinical trial in the first half of 2025.
Entrada Therapeutics, Inc.: In 2022, the company established a collaboration with Entrada focused on enabling efficient intracellular delivery of an oligonucleotide for DM1. This collaboration includes VX-670, an investigational candidate for the treatment of DM1 that is in clinical development. The company completed the single ascending dose portion of the global Phase 1/2 clinical trial for VX-670 in people with DM1. The company is enrolling and dosing the multiple ascending dose portion of the trial, which will evaluate the safety and efficacy of VX-670.
Out-license Agreements
In January 2025, the company entered into a collaboration agreement with Zai for the development and commercialization of povetacicept in mainland China, Hong Kong SAR, Macau SAR, Taiwan region and Singapore. Under this collaboration, Zai will help advance the povetacicept clinical trials and make the regulatory submissions in the licensed territories. Zai will also be responsible for commercialization activities in the licensed territories, if povetacicept becomes an approved product.
Cystic Fibrosis Foundation
In 2004, the company entered into a collaboration agreement with the Cystic Fibrosis Foundation, as successor in interest to the Cystic Fibrosis Foundation Therapeutics, Inc., to support research and development activities. Pursuant to the collaboration agreement, as amended, the company has agreed to pay tiered royalties ranging from single digits to sub-teens on covered compounds first synthesized and/or tested during a research term on or before February 28, 2014, including ivacaftor, lumacaftor and tezacaftor and royalties ranging from low-single digits to mid-single digits on net sales of certain compounds first synthesized and/or tested between March 1, 2014 and August 31, 2016, including elexacaftor. For combination products, such as ORKAMBI, SYMDEKO/SYMKEVI, TRIKAFTA/KAFTRIO, and ALYFTREK sales are allocated equally to each of the active pharmaceutical ingredients in the combination product, and royalties are then paid for any royalty-bearing components included in the combination.
Intellectual Property
The company has thirteen issued U.S. patents listed in the Orange Book that cover the active pharmaceutical ingredients in KALYDECO, its marketed formulations, and/or its approved indication.
The company has 22 issued U.S. patents listed in the Orange Book that cover the active pharmaceutical ingredients in ORKAMBI, its marketed formulations, and/or its approved indication. The company has 25 issued U.S. patents listed in the Orange Book that cover the active pharmaceutical ingredients in SYMDEKO, its marketed formulation, and/or its approved indication. The company has 28 issued U.S. patents listed in the Orange Book that cover the active pharmaceutical ingredients in TRIKAFTA, its marketed formulation, and/or its approved indication. The company has 35 issued U.S. patents listed in the Orange Book that cover the active pharmaceutical ingredients in ALYFTREK, its marketed formulation, and/or its approved indication. The company expects to have an issued patent listed in the Orange Book that covers the active pharmaceutical ingredient in JOURNAVX, its marketed formulation, and/or its approved indication.
Products approved by the FDA under a Biologics Licensing Application (BLA), including CASGEVY, receive 12 years of regulatory exclusivity in the U.S. from a product’s approval date. Additionally, the company has licenses to dozens of issued U.S. patents that cover CASGEVY, its approved indication, and/or its manufacture. Products approved by the FDA under a BLA are not subject to the Orange Book patent listing requirement.
In addition to protecting its marketed products, the company actively files patent applications in the U.S. and in foreign countries on inventions relating to its pipeline. For example, the company also owns and/or controls U.S. and foreign patents and/or patent applications relating to the following:
Other CF potentiators and correctors and many other related compounds, and the use of those compounds for the treatment CF.
VX-522 and other mRNA-based approaches for treating CF.
VX-993, VX-973, and other compounds being studied for the potential treatment of pain.
Inaxaplin and other compounds being studied for the potential treatment of AMKD.
Povetacicept for the treatment of IgAN.
Zimislecel, VX-264, and other cell-based approaches for treating T1D.
VX-670 for the treatment of DM1.
VX-407 and other compounds being studied for the potential treatment of ADPKD.
Other pre-clinical and clinical candidates and the use of such candidates to treat specified diseases.
The manufacture, pharmaceutical compositions, related solid forms, formulations, dosing regimens, and methods of use of many of the above compounds.
In December 2023, the company entered into an agreement with Editas Medicine, Inc. (Editas), providing the company a non-exclusive sublicense to certain patents relating to CRISPR/Cas9 technology, owned by Broad and Harvard, which are licensed to Editas. In addition to the patent portfolios licensed from Dr. Charpentier, Broad, and Harvard, the company owns patents and/or patent applications relating to the composition, manufacture, and use of CASGEVY.
VERTEX, KALYDECO, ORKAMBI, SYMDEKO, SYMKEVI, TRIKAFTA, KAFTRIO, CASGEVY, ALYFTREK, and JOURNAVX are registered trademarks of Vertex.
History
Vertex Pharmaceuticals Incorporated was founded in 1989. The company was incorporated in Massachusetts in 1989.
