Suzetrigine

— JOURNAVX is the first and only approved non-opioid oral pain signal inhibitor and the first new class of pain medicine approved in more than 20 years — — JOURNAVX is an effective and well-tolerated medicine without evidence of addictive potential indicated for use across all types of moderate-to-severe acute pain — BOSTON, MA, USA I January 30, 2025 I Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved JOURNAVX™ (suzetrigine), an oral, non-opioid, highly selective NaV1.8 pain signal inhibitor for the treatment of adults with moderate-to-severe acute pain. JOURNAVX is an effective, well-tolerated medicine without evidence of addictive potential indicated for use across all types of moderate-to-severe acute pain. “Today’s approval is a historic milestone for the 80 million people in America who are prescribed a medicine for moderate-to-severe acute pain each year,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “With the approval of JOURNAVX, a non-opioid, pain signal inhibitor and the first new class of pain medicine approved in more than 20 years, we have the opportunity to change the paradigm of acute pain management and establish a new standard of care.” “This is an incredible day for patients and physicians alike who now have an approved non-opioid treatment that delivers effective acute pain relief and a favorable safety profile without addictive potential,” said Jessica Oswald, M.D., M.P.H., Associate Physician in Emergency Medicine and Pain Medicine in San Diego and Vertex Acute Pain Steering Committee Member. “I believe JOURNAVX could redefine the management of pain and become a foundational treatment option for people with all types of moderate-to-severe acute pain, where options aside from opioids have been so desperately needed.” As part of Vertex’s ongoing commitment to patients, the company has established patient support programs to help ensure that qualified patients can access JOURNAVX. For more information visit JOURNAVX.com . About Acute Pain Acute pain is a serious and potentially disabling condition often caused by surgery, accident or injury. Over 80 million Americans are prescribed medicine to treat their moderate-to-severe acute pain every year. Of these, about 40 million are prescribed an opioid. Nearly 10% of acute pain patients treated initially with an opioid will go on to have prolonged opioid use, and about 85,000 patients will develop opioid use disorder annually. Poorly controlled acute pain can lead to reduced quality of life, development of chronic pain, and increased burden on the health care system and society. About JOURNAVX™ (suzetrigine) JOURNAVX (suzetrigine) is a first-in-class, oral, non-opioid, highly selective pain signal inhibitor that is selective for NaV1.8 relative to other NaV channels. NaV1.8 is a voltage-gated sodium channel that is selectively expressed in peripheral pain-sensing neurons (nociceptors), where its role is to transmit pain signals (action potentials). Because JOURNAVX blocks pain signals only found in the periphery, not in the brain, JOURNAVX provides effective relief of pain without the limitations of currently available therapies, including the addictive potential of opioids. The U.S. Food and Drug Administration approved twice-daily JOURNAVX for the treatment of adults with moderate-to-severe acute pain. Vertex has established a wholesale acquisition cost for JOURNAVX in the United States of $15.50 per 50mg pill. Vertex is also evaluating suzetrigine in peripheral neuropathic pain (PNP). The company’s Phase 3 pivotal program for suzetrigine in patients with painful diabetic peripheral neuropathy is ongoing, and Vertex plans to advance its pivotal program evaluating suzetrigine in patients with painful lumbosacral radiculopathy pending discussions with regulators. INDICATION and IMPORTANT SAFETY INFORMATION FOR JOURNAVX ™ (suzetrigine) INDICATION AND USAGE JOURNAVX is a prescription medicine used to treat adults with moderate-to-severe short term (acute) pain. It is not known if JOURNAVX is safe and effective in children. IMPORTANT SAFETY INFORMATION Patients should not take JOURNAVX if they take certain medicines that are strong inhibitors of an enzyme called CYP3A. Patients should ask their healthcare providers if they are not sure. Before taking JOURNAVX, patients should tell their healthcare provider about all of their medical conditions, including if they: have liver problems. People with liver problems may have an increased risk of getting side effects from taking JOURNAVX;are pregnant or plan to become pregnant as it is not known if JOURNAVX will harm an unborn baby. Patients and their healthcare providers should decide if they will take JOURNAVX while they are pregnant,are breastfeeding, or are planning to breastfeed, as it is not known if JOURNAVX passes into breast milk. Patients and their healthcare providers should decide if they will take JOURNAVX while they are breastfeeding. Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking JOURNAVX with certain other medicines may affect the way JOURNAVX and the other medicines work and may increase patients’ risk of side effects. Patients should ask their healthcare provider or pharmacist for a list of these medicines if they are not sure. Patients should especially tell their healthcare provider if they take hormonal birth control medicine (contraceptives) containing progestins other than levonorgestrel or norethindrone. If they take one of these contraceptives (progestins other than levonorgestrel or norethindrone), they may not work as well during treatment with JOURNAVX. Patients should also use nonhormonal contraceptives such as condoms or use other forms of hormonal birth control during treatment with JOURNAVX and for 28 days after they stop taking JOURNAVX. Medicines that are substrates of the CYP3A enzyme may become less effective during treatment with JOURNAVX. Their healthcare provider may need to adjust the dose of patients’ medicine when starting or stopping JOURNAVX. Patients should know the medicines they take and keep a list of them to show their healthcare provider and pharmacist when they get a new medicine. Patients should not take food or drink containing grapefruit while taking JOURNAVX. JOURNAVX can cause side effects: The most common side effects for patients treated with JOURNAVX include itching, muscle spasms, increased blood level of creatine phosphokinase, and rash. JOURNAVX may temporarily reduce the chance of females becoming pregnant while on treatment. Patients should talk to their healthcare provider if they have concerns about becoming pregnant. If patients are using contraceptives, continue to use contraceptives during treatment with JOURNAVX. Patients should tell their healthcare provider if they have any side effect that bothers them or that does not go away. These are not all of the possible side effects of JOURNAVX. Patients should call their healthcare provider for medical advice about side effects. Patients may report side effects to the FDA at 1-800-FDA-1088. Please click here for the full Prescribing Information, including Patient Information, for JOURNAVX. 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 serious diseases and conditions — cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain — and continues to advance clinical and research programs in these areas. 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 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 . SOURCE: Vertex Pharmaceuticals

A novel Vertex Pharmaceuticals drug that takes a new approach to pain is now approved by the FDA, a landmark decision for a first-in-class product that brings patients a treatment alternative intended to avoid the addiction risks posed by opioid medications. The regulatory decision announced late Thursday covers the treatment of moderate-to-severe acute pain in adults. Acute pain is defined as pain that lasts less than three months, such as aches following an injury or a surgical procedure. The Boston-based company’s new drug, a twice-daily pill known in development as suzetrigine, will be marketed under the brand name Journavx. “Today’s approval is an important public health milestone in acute pain management,” Jacqueline Corrigan-Curay, acting director of the FDA’s Center for Drug Evaluation and Research, said in the agency’s approval announcement. “A new non-opioid analgesic therapeutic class for acute pain offers an opportunity to mitigate certain risks associated with using an opioid for pain and provides patients with another treatment option.” Exclusive Improving the Healthcare Financial Experience to Help Care Flow Zelis CEO Amanda Eisel shares her perspective on how the company is solving the problems of a fragmented health financial system to benefit all. By Stephanie Baum The addiction risks of opioids come from their targeting of receptors in the central nervous system. But before pain signals reach the brain, they travel across pathways in the peripheral nervous system. Journavx targets one of these pathways, a sodium channel called NaV1.8. The drug is a small molecule designed to selectively block this channel without hitting other related sodium channels, which could lead to adverse effects. The drug comes from a pain research program that began at Vertex about 25 years ago. FDA approval of Journavx is based on the results of two Phase 3 studies, one that tested the drug after abdominoplasty (tummy tuck) surgery and the other after bunionectomy surgery. Both studies met the main goal of showing statistically significant improvement compared to placebo according to a pain rating scale. The studies also included as a comparator the drug Vicodin, an inexpensive painkiller that pairs the opioid hydrocodone with the main pharmaceutical ingredient in Tylenol. On this secondary trial measure, the results were disappointing. In tummy tuck patients, the numerical improvement achieved by suzetrigine fell short of statistical significance compared to Vicodin. In bunion surgery patients, Vicodin beat suzetrigine. The Vertex drug was safe and well tolerated in these studies. Vertex reported no addiction problems in the studies. Vertex is also developing Journavx for chronic pain, but there have been mixed results so far. In a Phase 2 test in painful lumbosacral radiculopathy (lower back pain), data released last month showed that despite meeting the main goal according to a pain rating scale, the placebo arm posted a similar response. The study was not powered or designed to show differentiation from placebo, but Vertex’s stock price still took a hit. The company said it plans to advance the drug to a pivotal test in this indication, pending discussions with regulators. A Phase 3 test in painful diabetic peripheral neuropathy is ongoing. In a note sent to investors after the back pain trial readout, William Blair analyst Myles Minter said the lack of separation from placebo in that trial increases the risk for the ongoing Phase 3 diabetic peripheral neuropathy test. presented by Artificial Intelligence What Keeps Healthcare CIOs Up at Night: Balancing Technology Investments with Consumer Expectations When it comes to managing inbound phone calls, underperformance has devastating cost implications. By Stephanie Baum Vertex set a $15.50 per pill price for Journavx. That’s slightly higher than the $12 per pill price that Leerink Partners modeled. A December report by the Institute for Clinical and Economic Review concluded that compared to treatment with Vicodin, the Vertex drug would lead to savings that mainly come from avoiding the excess healthcare costs associated with opioid use disorder. A recent regulatory change should support reimbursement of the new Vertex product. The Non-Opioids Prevent Addiction in the Nation (NOPAIN) Act took effect on Jan. 1, requiring Medicare to provide separate reimbursement for qualifying non-opioid pain drugs. A goal of the legislation is to increase access to alternative pain drugs to reduce opioid use. Leerink analyst David Risinger wrote in a research note that the Journavx label represents the best-case scenario for Vertex. There is no language for addiction or abuse potential, which Leerink expects will be critical to the drug’s utilization. The firm sees Journavx as the first in a series of novel non-opioid pain medicines coming from Vertex. Risinger said Leerink projects Journavx and follow-on NaV1.8 inhibitors for acute and neuropathic pain could top $10 billion in peak sales. That’s important for Vertex, which is working to diversify its portfolio beyond the cystic fibrosis products that currently bring in the vast majority of its revenue. Photo by Vertex Pharmaceuticals