VERTEX PHARMACEUTICALS INC / MA
For the period ended Sep 30, 2026. Filed Nov 3, 2026.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2026 · Filed Nov 3, 2026
VRTX$547.09+0.8%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2026-06-30; filed 2026-08-04.
0000875320-26-000259
Item 2. Management's Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-09-30; filed 2026-11-03.
0000875320-26-000291
Item 2. Management's Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 379 previous paragraphs and 415 current paragraphs.
212 unchanged; 89 reworded; 111 added; 71 removed; 7 previous and 3 current paragraphs rearranged.
Tables are not compared: 11 previous and 11 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (111)
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targeting CF, SCD, beta thalassemia, IgAN, neuropathic pain, APOL1-mediated kidney disease, primary membranous
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nephropathy, congenital adrenal hyperplasia, adrenocorticotropic hormone-dependent Cushing’s syndrome, autosomal
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of age. Since approval, multiple children have initiated treatment.
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•In October, the United Arab Emirates and Kingdom of Bahrain approved CASGEVY for the treatment of children 2
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treatment of children with SCD or TDT.
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retail settings, representing a more than 40% increase compared to the second quarter of 2026 and more than four
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times the 170,000 JOURNAVX prescriptions that were filled in the third quarter of 2025.
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increase since the second quarter of 2026, and nearly tripling the$19.6 million of JOURNAVX product revenues in
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thethird quarter of 2025.
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Acromegaly
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•PALSONIFY (paltusotine) is a first-in-class, small molecule, once-daily oral somatostatin receptor type 2 agonist
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approved in the U.S. for the treatment of adults with acromegaly who had an inadequate response to surgery or for
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whom surgery is not an option. Acromegaly is a rare and debilitating condition caused by a benign pituitary tumor
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that secretes excess growth hormone. There are an estimated 12,000 adults with acromegaly in the U.S. for whom
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PALSONIFY is an option.
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•We recorded$10.1 million of PALSONIFY revenue in September 2026, following the closing of the Crinetics
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Acquisition on September 1, 2026.
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•More than 245 million individuals have access to PALSONIFY, representing approximately 80% of U.S. covered
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lives.
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•An estimated 3,200 endocrinologists treat the majority of people with acromegaly in the U.S. As of the end of the
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third quarter of 2026, approximately 500 of those target specialty endocrinologists are prescribing PALSONIFY.
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•Among patients starting or switching therapy, PALSONIFY has the leading brand share at more than 40%.
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•PALSONIFY was approved in the European Union in April 2026. We expect to begin launches in European markets
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in the second half of 2027. The regulatory submission for PALSONIFY in Japan was completed in April 2026, and
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the Ministry of Health, Labor and Welfare (the “MHLW”) has granted orphan drug designation in Japan.
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•We have initiated global regulatory submissions for ALYFTREK for the treatment of children with CF two to five
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years of age.
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•Povetacicept is under review for accelerated approval for adults with IgAN in Saudi Arabia, and the Saudi Food and
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analysis cohort has been treated for 48 weeks and to share data from the interim analysis in early 2027.
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Rare Endocrine Disorders
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•We are developing atumelnant, a once-daily oral adrenocorticotropic hormone (“ACTH”) receptor antagonist, for
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the treatment of classic congenital adrenal hyperplasia (“CAH”), which affects approximately 17,000 people in the
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U.S. Classic CAH is a rare, chronic, genetic condition in which the adrenal glands are unable to make enough
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cortisol. Current therapies force a choice between androgen control and steroid burden, representing a significant
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unmet need.
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•The Phase 3 clinical trial evaluating atumelnant in adults with CAH is a global, 32-week, randomized, double blind,
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placebo-controlled trial of 150 people. Enrollment and dosing are well underway, and we expect to complete
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enrollment in the trial in the first quarter of 2027. The FDA, the European Medicines Agency, and the MHLW in
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Japan have granted Orphan Drug designation to atumelnant in CAH.
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•Atumelnant has demonstrated therapeutic potential in people with ACTH-dependent Cushing’s syndrome, another
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neuropathic pain, type 1 diabetes, IgA nephropathy, primary membranous nephropathy and other autoimmune diseases and
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intent with the Pan-Canadian Pharmaceutical Alliance for reimbursement of ALYFTREK for eligible patients six
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SCD and TDT. Approximately 5,500 patients with SCD or TDT may be eligible for treatment with CASGEVY for
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•In May, we secured reimbursement for CASGEVY for eligible patients 12 years and older with SCD or TDT in
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Germany. We are committed to working with government and reimbursement authorities globally to ensure
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sustainable access for eligible patients.
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•In the second quarter and first six months of 2026, approximately 535,000 and 900,000 prescriptions, respectively,
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•We have reached agreements with two additional major pharmacy benefit managers for Medicare Part D coverage of
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JOURNAVX. As a result, seniors covered by three of the four major Medicare Part D pharmacy benefit managers
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have reimbursed access. Twenty-three states provide coverage for JOURNAVX via Medicaid. In total,
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approximately 260 million individuals have reimbursed access to JOURNAVX across a wide range of commercial
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and government payers.
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•Following positive results from the Phase 3 clinical trial evaluating ALYFTREK in children with CF two to five
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years of age, we initiated global regulatory submissions for this age group.
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•During the second quarter of 2026, Health Canada accepted our new drug submission for suzetrigine for the
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•We have completed our regulatory submission for accelerated approval of povetacicept in adults with IgAN in Saudi
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Phase 3 portion, which is underway.
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of 2026.
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•We expect to share data from the interim analysis of the AMPLITUDE clinical trial in early 2027. We expect to
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been treated for 48 weeks.
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•The FDA cleared the Investigational New Drug Application for VX-017, our stem cell-derived, fully differentiated
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•We expect to provide updated timelines for the zimislecel and VX-017 programs in 2026.
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•In July, we entered into an agreement and plan of merger (the “Crinetics Merger Agreement”) to acquire all of the
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issued and outstanding shares of common stock of Crinetics Pharmaceuticals, Inc. (“Crinetics”)for $85.00 per share
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in cash, for a total equity value of approximately $10.0 billion (the “Crinetics Acquisition”). We expect the
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transaction to close in the third quarter of 2026, subject to certain customary conditions. Crinetics’ PALSONIFY®
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(paltusotine) is a once-daily oral therapy for adults with acromegaly, a rare and debilitating condition caused by a
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pituitary tumor that secretes excess growth hormone, who had an inadequate response to surgery and/or for whom
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surgery is not an option. PALSONIFY is approved by the FDA and the European Medicines Agency, and is under
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review by other global regulatory bodies. Crinetics’ most advanced pipeline candidate, atumelnant, is a once-daily
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oral adrenocorticotropic hormone receptor antagonist in Phase 3 development for congenital adrenal hyperplasia.
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SCD, TDT and acute pain.
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In July 2026, we entered into the Crinetics Merger Agreement to acquire Crinetics as described above. Crinetics is a
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endocrine diseases and endocrine-related tumors. We will acquire Crinetics for $85.00 per share in cash, for a total equity
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value of approximately $10.0 billion. We expect to fund the acquisition with our cash, cash equivalents, andproceeds from
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the 2026 Term Loan, as defined below. The Crinetics Acquisition is not conditioned on our receipt of financing. We will
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account for the acquisition in the period that it closes.
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Therapeutics AG (“CRISPR”) and Entrada Therapeutics, Inc. (“Entrada”).
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In the second quarter of 2025, our other revenues included a $20.6 million upfront payment received from our
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collaboration agreement with Ono Pharmaceuticals Co., Ltd. In thefirst half of 2025, our other revenues also included a
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people with serious diseases, with a focus on specialty markets. We have seven approved medicines: five that treat the
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people with serious diseases, with a focus on specialty markets. We have eight approved medicines: five that treat the
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(“SCD”) and transfusion dependent beta thalassemia (“TDT”), life shortening inherited blood disorders, and one that treats
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(“SCD”) and transfusion dependent beta thalassemia (“TDT”), life shortening inherited blood disorders, one that treats
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moderate-to-severe acute pain. We are also preparing for the anticipated launch of povetacicept, a potential treatment for IgA
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moderate-to-severe acute pain, and one that treats acromegaly. We are also preparing for the anticipated launch of
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nephropathy (“IgAN”). Our clinical-stage pipeline spans a range of programs targeting CF, SCD, beta thalassemia,
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povetacicept, a potential treatment for IgA nephropathy (“IgAN”). Our clinical-stage pipeline spans a range of programs
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cytopenias, APOL1-mediated kidney disease, autosomal dominant polycystic kidney disease and myotonic dystrophy type 1,
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dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis and myotonic dystrophy type 1, reflecting
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reflecting our commitment to addressing significant unmet medical needs globally.
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our commitment to addressing significant unmet medical needs globally.
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•In the second quarter of 2026, we secured reimbursement for ALYFTREK in four additional countries, including
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•In the third quarter of 2026, we secured reimbursement for ALYFTREK in seven additional countries, bringing the
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Spain, bringing the total number of countries where ALYFTREK is reimbursed to 25. We also signed a letter of
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total number of countries where ALYFTREK is reimbursed to 32.
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•In the second quarter of 2026, we recorded$76.4 million of CASGEVY product revenues, representing a 78%
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•In thethird quarter of 2026, we recorded$99.2 million of CASGEVY product revenues, representing a 30% increase
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increase compared to the first quarter of 2026 and a more than 300% increase compared to the second quarter of
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compared to the second quarter of 2026 and a more than 400% increase compared to thethird quarter of 2025.
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with SCD or TDT, making it the first genetic therapy indicated for children as young as two years of age for both
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with SCD or TDT, making it the first genetic therapy for SCD and TDT indicated for children as young as two years
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Arabia”) and the United Kingdom for the treatment of children five to eleven years of age.
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years of age and older, and the United Kingdom approved CASGEVY for the treatment of children 5 years of age
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the first time with this approval. We also completed regulatory submissions in the Kingdom of Saudi Arabia (“Saudi
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and older. We have completed regulatory submissions in the Kingdom of Saudi Arabia (“Saudi Arabia”) for the
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have been filled for JOURNAVX across the hospital and retail settings.
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•In the third quarter of 2026, more than 750,000 prescriptions were filled for JOURNAVX across the hospital and
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•In the second quarter of 2026, we recorded$49.6 million of JOURNAVX product revenues, representing a 71%
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•In thethird quarter of 2026, we recorded$58.3 million of JOURNAVX product revenues, representing an 18%
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neuropathy, a form of peripheral neuropathic pain, by the end of 2026.
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neuropathy, a form of peripheral neuropathic pain, by the end of 2026 and expect to share results in mid-2027.
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•The FDA accepted our biologics license application for accelerated approval of povetacicept for adults with IgAN
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•The biologics license application for accelerated approval of povetacicept for adults with IgAN is under priority
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and assigned a PDUFA target action date of November 30, 2026. If approved, povetacicept will become the first
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review by the FDA, with an assigned PDUFA target action date of November 30, 2026. If approved, povetacicept
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commercialized therapy in our emerging nephrology franchise.
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will become the first commercialized therapy in our emerging nephrology franchise.
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Arabia, and the Saudi Food and Drug Authority has granted Breakthrough Designation to povetacicept.
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Drug Authority has granted Breakthrough designation to povetacicept.
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nephropathy (“pMN”), another B cell-mediated disease. We completed the Phase 2B portion of the Phase 2/3
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nephropathy (“pMN”), another B cell-mediated disease. We are enrolling and dosing patients in the Phase 3 portion
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OLYMPUS pivotal trial evaluating povetacicept in people with pMN, and we confirmed the dose selection for the
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of the OLYMPUS Phase 2/3 pivotal trial evaluating povetacicept in people with pMN.
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•Inaxaplin is our small molecule for the treatment of APOL1-mediated kidney disease (“AMKD”). We expect to
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•Inaxaplin is our small molecule for the treatment of APOL1-mediated kidney disease (“AMKD”). We have
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complete full enrollment in the AMPLITUDE Phase 2/3 pivotal clinical trial evaluating inaxaplin in the second half
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completed full enrollment in the AMPLITUDE pivotal clinical trial evaluating inaxaplin.
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conduct the pre-planned interim analysis for potential U.S. accelerated approval once the interim analysis cohort has
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•We are on track to conduct the pre-planned interim analysis for potential U.S. accelerated approval once the interim
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1/2 clinical trial to evaluate the safety and efficacy of VX-017 in people with T1D in the near term.
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•We are enrolling and dosing patients in a Phase 1/2 clinical trial to evaluate the safety and efficacy of VX-017 in
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•Zimislecel is an allogeneic, stem cell-derived, fully differentiated, insulin-producing islet cell replacement therapy,
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people with type 1 diabetes (“T1D”). VX-017 is an allogeneic, stem cell-derived, fully differentiated islet cell
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islet cell therapy designed to treat all eligible patients with T1D, regardless of blood type. We plan to initiate a Phase
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therapy designed to treat the targeted patient population with T1D regardless of blood type.
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In thefirst half of 2026, our total product revenues came primarily from the sale of our medicines for the treatment of
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In thefirst nine months of 2026, our total product revenues came primarily from the sale of our medicines for the
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CF. Our CF strategy involves continuing to develop and obtain approval and reimbursement for treatment regimens that will
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treatment of CF. Our CF strategy involves continuing to develop and obtain approval and reimbursement for treatment
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provide benefits to all people with CF and increasing the number of people with CF eligible and able to receive our
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regimens that will provide benefits to all people with CF and increasing the number of people with CF eligible and able to
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medicines. Outside of CF, we continue to advance the commercialization of CASGEVY for the treatment of SCD and TDT,
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receive our medicines. Outside of CF, we continue to advance the commercialization of CASGEVY for the treatment of SCD
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and JOURNAVX for the treatment of acute pain, and we are preparing for a potential launch of povetacicept for the treatment
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and TDT, and JOURNAVX for the treatment of acute pain. We added PALSONIFY for the treatment of acromegaly to our
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of IgAN. In addition, we are advancing our pipeline of product candidates for the treatment of serious diseases outside of CF,
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launch of povetacicept for the treatment of IgAN. In addition, we are advancing our pipeline of product candidates for the
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JOURNAVX, and, ultimately, our pipeline therapies, in U.S. and ex-U.S. markets.
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JOURNAVX, PALSONIFY, and, ultimately, our pipeline therapies, in U.S. and ex-U.S. markets.
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publicly traded biotechnology company focused on discovering, developing, and commercializing novel therapeutics for
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In September 2026, we acquired Crinetics, a publicly traded biotechnology company focused on discovering,
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and proceeds from the 2026 Term Loan described above.
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of proceeds from the 2026 Term Loan. We have accounted for the Crinetics Acquisition as a business combination, which
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Over the last several years, we entered into collaboration agreements with a number of companies, including CRISPR
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We have collaboration agreements with a number of companies, including CRISPR Therapeutics AG (“CRISPR”) and
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In thefirst half of 2026 and 2025, our AIPR&D included$21.9 million and$22.0 million, respectively, related to
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In thefirst nine months of 2026 and 2025, our AIPR&D included$62.5 million and$76.5 million, respectively, related
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upfront, contingent milestone, or other payments pursuant to our business development transactions, including the asset
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to upfront, contingent milestone, or other payments pursuant to our business development transactions, including the asset
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commercial portfolio in the third quarter of 2026 through the Crinetics Acquisition, and we are preparing for a potential
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treatment of serious diseases outside of CF, SCD, TDT, acute pain, and acromegaly.
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borrowed pursuant to our 2026 Term Loan partially offset by repurchases of our common stock pursuant to our share
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years of age and older in Canada.
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increase compared to the first quarter of 2026 and a 151% increase compared to the second quarter of 2025.
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treatment of moderate-to-severe acute pain, and review is underway.
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using standard immunosuppression to protect the implanted cells. We are enrolling and dosing patients in the Phase
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1/2/3 clinical trial of zimislecel in people with type 1 diabetes (“T1D”).
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thecontinued performance of our CF therapies.
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•To the extent we borrow amounts under the 2026 Term Loan discussed above, we will be required to repay a portion