Ivacaftor

Pictured: Vertex Pharmaceuticals' headquarters in Boston, Massachusetts/iStock, hapabapa Vertex Pharmaceuticals on Thursday announced that it has entered into an extended, long-term reimbursement agreement with England’s National Health Service, granting access to its cystic fibrosis treatments Kaftrio (ivacaftor/tezacaftor/elexacaftor), Symkevi (ezacaftor/ivacaftor) and Orkambi (lumacaftor/ivacaftor). The biotech did not disclose specific details of the deal, only revealing that it would also apply to future license extensions of these drugs. Vertex is working on similar deals in Scotland, Wales and Northern Ireland. Ludovic Fenaux, senior vice president of Vertex International, said in a statement that the company is “delighted” to have reached a deal with the National Health Service (NHS), adding that the reimbursement agreement will ensure “long-term access” to Kaftrio, Symkevi and Orkambi for cystic fibrosis (CF) patients in England. Vertex is currently in talks with NHS England as well as the drug pricing watchdog National Institute for Health and Care Excellence (NICE) to ensure rapid access to the biotech’s next-generation triple combination CF treatment. In a Phase III readout posted in February 2024, the investigational therapy, dubbed vanza triple, matched Kaftrio in terms of improving patients’ lung function, and outperformed the approved treatment in terms of sweat chloride levels. Thursday’s reimbursement deal comes after the NICE in October 2023 declined to recommend Kaftrio, Symkevi and Orkambi for use in CF patients. At the time, the watchdog said that while the drugs’ efficacy data support their clinical benefit, they were too expensive to be covered. “The most likely cost-effectiveness estimates for [Kaftrio, Orkambi and Symkevi] are above the range that NICE considers an acceptable use of NHS resources,” the agency wrote. However, earlier this week, NICE reversed its recommendation. In its final draft guidance, the watchdog said that given the severity of CF and its effects of quality and length of life, the cost-effectiveness estimates for Kaftrio, Symkevi and Orkambi now fell within its threshold of acceptable use of NHS resources. While NICE’s recommendation and the NHS deal names three of Vertex’s CF drugs, the centerpiece is Kaftrio, which the watchdog said yielded better short- and long-term improvements than Symkevi and Orkambi. Kaftrio is also known as Trikafta in the U.S. and is a three-drug combo that first won the FDA’s approval in October 2019, allowing its use in patients aged 12 years and older bearing at least one copy of the F508del mutation in the CTFR gene. Trikafta works by increasing the number and improving the function of F508del-CFTR proteins at the cell surface. Trikafta won additional approvals in 2021, for use in CF patients ages 6 to 11 years, and in 2023, for patients 2 through 5 years. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

Executive Chairperson and former CEO of Vertex Pharmaceuticals to help guide the development of a high-value pipeline of first-in-class covalent protein drugs from Enlaza’s War-Lock™ platform LA JOLLA, Calif.--(BUSINESS WIRE)-- Enlaza Therapeutics, the first covalent biologic platform company, today announced the appointment of Dr. Jeffrey Leiden, Executive Chairperson of Vertex Pharmaceuticals, as a key advisor to the company. Dr. Leiden has more than 40 years of experience in the biopharma industry developing life-improving medicines and building companies that transform the industry. “Jeff is one of the most accomplished and admired leaders in our industry. He has dedicated his career to developing important first-in-class medicines from novel breakthrough technologies that have helped millions of people with serious diseases,” said Sergio Duron, Ph.D., CEO of Enlaza Therapeutics. “With our War-Lock platform, we can create covalent protein drugs with the potential to improve treatments for many diseases. Jeff’s guidance will be invaluable as we establish a diverse pipeline and advance our lead assets toward clinical development.” "While covalency has been a long-standing strategy in small molecule drug development, Enlaza is the first to incorporate covalency into biologics, unlocking a new class of protein therapeutics," said Dr. Leiden. "I look forward to working closely with the Enlaza team to realize the full potential of this exciting new drug discovery and development platform and help create meaningful new medicines for patients.” As CEO and President of Vertex from 2012 through 2020, Dr. Leiden built a robust pipeline of drug candidates, with a particular focus on therapies that cure or modify the course of disease. Under his leadership, Vertex delivered the first and only medicines to treat the underlying cause of cystic fibrosis for patients with certain forms of the disease, including KALYDECO®, ORKAMBI®, SYMDEKO® and TRIKAFTA®. In 2015, he established a collaboration with CRISPR Therapeutics to develop transformative therapies for sickle cell disease (SCD), beta thalassemia (TDT) and other diseases; and in 2023, CASGEVY™ was approved as the first gene-editing therapy in the world for SCD and TDT. In 2019, he led the Vertex acquisition of Semma Therapeutics to develop a clinical program to investigate what may be the first potentially curative stem cell-based therapy for type 1 diabetes. Prior to joining Vertex, Dr. Leiden was a managing director at Clarus Ventures, a life sciences venture capital firm, from 2006 through 2012. Dr. Leiden was President, Chief Operating Officer and Chief Scientific Officer of Abbott Laboratories, Pharmaceuticals Products Group, and a member of the board of directors of Abbott Laboratories from 2000 to 2006. At Abbott he led the development and commercialization of HUMIRA®. From 1987 to 2000, Dr. Leiden held several academic appointments, including the Rawson Professor of Medicine and Pathology and Chief of Cardiology and Director of the Cardiovascular Research Institute at the University of Chicago, the Elkan R. Blout Professor of Biological Sciences at the Harvard School of Public Health, and Professor of Medicine at Harvard Medical School. He is an elected member of both the American Academy of Arts and Sciences and the National Academy of Medicine. About the War-Lock™ Platform Enlaza’s covalent biologic platform, called War-Lock, creates highly specific therapeutic warheads that covalently bind to drug targets of interest. This white-space technology enables, for the first time, a covalent-acting protein drug that retains the selectivity of small-format biologics. Protein drugs produced by the platform can be modified to incorporate various payloads and achieve specific delivery to target tissues with high fidelity. About Enlaza Therapeutics Enlaza Therapeutics is pioneering the field of covalent biologics. This novel class of protein therapeutics has the potential to delivers more efficacious and safer treatments for patients. The company's proprietary War-Lock™ platform leverages leading synthetic biology technology for site-specific covalent coupling driven by drug binding. Enlaza is building a pipeline of first-in-class covalent biologics. For more information, please visit .