For Immediate Release: December 08, 2023 Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signali

Submission status for VERTEX PHARMS INC's drug TRIKAFTA (COPACKAGED) (SUPPL-2) with active ingredient ELEXACAFTOR, IVACAFTOR, TEZACAFTOR; IVACAFTOR has changed to 'Approval' on 08/03/2023. Application Category: NDA, Application Number: 217660, Application Classification: Labeling

Submission status for VERTEX PHARMS INC's drug TRIKAFTA (COPACKAGED) (SUPPL-11) with active ingredient ELEXACAFTOR, IVACAFTOR, TEZACAFTOR; IVACAFTOR has changed to 'Approval' on 08/03/2023. Application Category: NDA, Application Number: 212273, Application Classification: Labeling

Submission status for VERTEX PHARMS INC's drug ORKAMBI (SUPPL-5) with active ingredient IVACAFTOR; LUMACAFTOR has changed to 'Approval' on 08/03/2023. Application Category: NDA, Application Number: 211358, Application Classification: Labeling

Submission status for VERTEX PHARMS INC's drug SYMDEKO (COPACKAGED) (SUPPL-11) with active ingredient IVACAFTOR; IVACAFTOR, TEZACAFTOR has changed to 'Approval' on 08/03/2023. Application Category: NDA, Application Number: 210491, Application Classification: Labeling

Submission status for VERTEX PHARMS INC's drug KALYDECO (SUPPL-17) with active ingredient IVACAFTOR has changed to 'Approval' on 08/03/2023. Application Category: NDA, Application Number: 207925, Application Classification: Labeling

Submission status for VERTEX PHARMS INC's drug ORKAMBI (SUPPL-17) with active ingredient IVACAFTOR; LUMACAFTOR has changed to 'Approval' on 08/03/2023. Application Category: NDA, Application Number: 206038, Application Classification: Labeling

Submission status for VERTEX PHARMS INC's drug KALYDECO (SUPPL-16) with active ingredient IVACAFTOR has changed to 'Approval' on 05/03/2023. Application Category: NDA, Application Number: 207925, Application Classification: Efficacy

Submission status for VERTEX PHARMS INC's drug TRIKAFTA (ORIG-1) with active ingredient ELEXACAFTOR;TEZACAFTOR;IVACAFTOR;IVACAFTOR has changed to 'Approval' on 04/26/2023. Application Category: NDA, Application Number: 217660, Application Classification: Type 3 - New Dosage Form

Submission status for VERTEX PHARMS INC's drug TRIKAFTA (COPACKAGED) (SUPPL-9) with active ingredient ELEXACAFTOR, IVACAFTOR, TEZACAFTOR; IVACAFTOR has changed to 'Approval' on 04/26/2023. Application Category: NDA, Application Number: 212273, Application Classification: Labeling

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced multiple program updates ahead of upcoming investor meetings in January, including the company's scheduled webcast from the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 a.m. ET/7:30 a.m. PT. "2024 marked another year of excellent progress for Vertex, as we reached more people with CF than ever before, began a new era of commercial diversification, and advanced and broadened our clinical stage pipeline," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "In 2025, we are poised to diversify our business further across multiple dimensions: our revenue, as we

- Exclusive collaboration and licensing agreement for povetacicept in the region - - Zai Lab will leverage its local expertise and commercial footprint to accelerate development of povetacicept and bring the medicine to eligible patients in the region if approved - Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) and Zai Lab Limited (NASDAQ:ZLAB, HKEX: 9688)) today announced an exclusive collaboration and license agreement for the development and commercialization of Vertex's povetacicept (pove) in mainland China, Hong Kong SAR, Macau SAR, Taiwan region and Singapore (the licensed territory). Pove is a recombinant fusion protein therapeutic and dual antagonist of BAFF (B cell activatin

-- Exclusive collaboration and licensing agreement for povetacicept in the region -- Zai Lab will leverage its local expertise and commercial footprint to accelerate development of povetacicept and bring the medicine to eligible patients in the region if approved Zai Lab Limited (NASDAQ:ZLAB, HKEX: 9688)) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced an exclusive collaboration and license agreement for the development and commercialization of Vertex's povetacicept (pove) in mainland China, Hong Kong SAR, Macau SAR, Taiwan region and Singapore (the licensed territory). Pove is a recombinant fusion protein therapeutic and dual antagonist of BAFF (B cell activating

-- Collaboration to leverage Orna's differentiated lipid nanoparticle (LNP) delivery solutions for patients with SCD and TDT -- -- Orna to receive an upfront payment and is eligible to receive potential pre-clinical, clinical, and commercialization milestone payments and royalties -- WATERTOWN, Mass., Jan. 7, 2025 /PRNewswire/ -- Orna Therapeutics (through its wholly owned subsidiary ReNAgade Therapeutics Inc.) announced a three-year strategic research collaboration with Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) to utilize Orna's novel and proprietary LNP delivery solutions to enhance Vertex's efforts in developing next generation gene editing therapies for patients with SCD and TDT.

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that Dr. Reshma Kewalramani, Chief Executive Officer and President, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 at 10:30 a.m. ET/7:30 a.m. PT. A live webcast of management's remarks will be available through the Vertex website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website. 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 t

- 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® - 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

- Approximately 300 more people with cystic fibrosis in the U.S. are now eligible for a medicine that treats the underlying cause of their disease for the first time - Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of people with cystic fibrosis (CF) ages 2 and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation that is responsive to TRIKAFTA based on clinical and/or in vitro data. In addition, safety information on liver injury and liver fai

– Treatment with the highly selective NaV1.8 pain signal inhibitor suzetrigine met the primary endpoint with a statistically significant and clinically meaningful 2.02 point within-group reduction from baseline in the Numeric Pain Rating Scale (NPRS) – – Placebo arm showed similar within-group reduction in NPRS – – Suzetrigine was generally well tolerated – – Advancement to Phase 3 in painful lumbosacral radiculopathy planned, pending discussions with regulators – – Vertex to host investor call on December 19 at 8:00 a.m. ET – Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced results from its Phase 2 study of suzetrigine, an investigational, oral, highly selective

- Data from long-term follow-up of patients in clinical trials further demonstrate durability of the transformative benefits of CASGEVY™ - - Safety profile consistent with busulfan conditioning and autologous hematopoietic stem cell transplant - - Vertex provides update on progress in bringing CASGEVY to patients - Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced longer-term data for CASGEVY™ (exagamglogene autotemcel) from global clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). CASGEVY is the first and only approved CRISPR/Cas9 gene-edited therapy. The results, presented at the American Society of Hema

— Product revenue of $2.77 billion, a 12% increase compared to Q3 2023 — — Raising full-year product revenue guidance to $10.8 billion to $10.9 billion — — Preparing for two potential near-term launches: vanzacaftor triple in CF and suzetrigine (VX-548) for moderate-to-severe acute pain — — Pipeline progress continuing with three additional programs advancing to Phase 3: suzetrigine in DPN, povetacicept in IgAN and VX-880 in T1D — Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today reported consolidated financial results for the third quarter ended September 30, 2024, and raised its full-year product revenue guidance to $10.8 billion to $10.9 billion. "The third quarter marked anot

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that Nancy Thornberry has been appointed to its Board of Directors as an independent director. Ms. Thornberry has more than 35 years of experience in the pharmaceutical and biotech industries, spanning drug discovery, development and commercialization. She founded and served as Chief Executive Officer of Kallyope until 2021 and remains on its Board of Directors and is Chair of Research & Development. Prior to joining Kallyope, Ms. Thornberry spent more than 30 years at Merck & Co., Inc., most recently as Senior Vice President and Global Franchise Head, Diabetes and Endocrinology, with responsibility for discovery and clinical

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that Michel Lagarde has been appointed to its Board of Directors as an independent director. Mr. Lagarde is a senior health care and business leader with global expertise, currently serving as Chief Operating Officer and Executive Vice President of Thermo Fisher Scientific Inc. Prior to Thermo Fisher, Mr. Lagarde served as President and Chief Operating Officer of Patheon, a contract manufacturer to the biopharma industry, and as a Managing Director at JLL Partners, a private equity firm investing in health care services. "We are delighted to have Michel join the Vertex Board. His deep experience across numerous segments of

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that E. Morrey Atkinson, Ph.D., has been appointed Executive Vice President, Chief Technical Operations Officer, Head of Biopharmaceutical Sciences and Manufacturing Operations, effective immediately. Dr. Atkinson has served as Senior Vice President of Global Commercial Manufacturing and Supply Chain since July 2020. This new role and function unifies responsibility for small molecule, cell and gene therapies across preclinical, clinical and commercial biopharmaceutical sciences, manufacturing operations and global supply chain. "Over the last three years, Morrey has played a vital role in building out our commercial manufa

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that Jonathan Biller has been appointed Chief Legal Officer, effective September 19, 2022. Mr. Biller will report directly to Vertex's Chief Executive Officer and President, Reshma Kewalramani, M.D., and join the company's Executive Committee. In this role, Mr. Biller will oversee all of Vertex's global legal and compliance functions. "Jonathan is an outstanding addition to Vertex with his deep legal, industry and business experience, and I am delighted to welcome him to the company," said Dr. Kewalramani. "Jonathan will lead an extraordinarily talented legal and compliance team and support our continued growth as we expand

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that Stuart A. Arbuckle has been appointed as the company's Executive Vice President, Chief Operating Officer (COO), effective immediately. Mr. Arbuckle has served as Vertex's Executive Vice President and Chief Commercial Officer since 2012 and expanded his role to include oversight of operations earlier this year. "This is an exciting time for Vertex, as we continue to expand access to our cystic fibrosis portfolio around the world and prepare to bring forward new therapies with the potential to transform the treatment of multiple serious diseases," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Ver

Patrik De Haes M.D. becomes Non-executive Chairman Leuven, BE and Boston, MA, US – May 17, 2021 – 7:30 AM CET – Oxurion NV (Euronext Brussels: OXUR), a biopharmaceutical company developing next generation standard of care ophthalmic therapies, with a clinical stage portfolio in retinal vascular disorders, announces in a planned succession, the promotion of Tom Graney, CFA from Chief Financial Officer (CFO) to Chief Executive Officer (CEO) of the company. He takes over from Dr. Patrik De Haes, who after 14 years as CEO, has decided to move away from day-to-day management of the company to become the Non-executive Chairman of Oxurion. Thomas Clay, the outgoing Chairman of Oxurion, will remain

WARREN, N.J., Feb. 09, 2021 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST), a pharmaceutical company focused on developing and commercializing differentiated products that address patients’ unmet needs and solve therapeutic problems, today announced the appointment of Julie Krop, M.D., Chief Medical Officer of Freeline Therapeutics (NASDAQ: FRLN), and Marco Taglietti, M.D., Director, President and Chief Executive Officer of SCYNEXIS (NASDAQ: SCYX), to the Board of Directors of the Company effective February 10, 2021. Aquestive also announced the resignation of Douglas K. Bratton from the Board of Directors after more than 17 years of service. Aquestive’s Board of Directors