NEW YORK, NY - Pfizer Inc. announced it is voluntarily withdrawing all lots of OXBRYTA ^® (voxelotor) for the treatment of sickle cell disease (SCD) at this time, in all markets where it is approved. Pfizer is also discontinuing all active voxelotor clinical trials and expanded access programs worldwide.

Pfizer's decision is based on the totality of clinical data that now indicates the overall benefit of OXBRYTA no longer outweighs the risk in the approved sickle cell patient population. The data suggest an imbalance in vaso-occlusive crises and fatal events which require further assessment. Pfizer has notified regulatory authorities about these findings and its decision to voluntarily withdraw OXBRYTA from the market and discontinue distribution and clinical studies while further reviewing the available data and investigating the findings.

“The safety and well-being of patients is of the utmost importance to Pfizer, and we believe this action is in the best interest of patients,” said Aida Habtezion, Chief Medical Officer and Head of Worldwide Medical and Safety at Pfizer. “Our primary concern is for patients who suffer from SCD, which remains a very serious and difficult-to-treat disease with limited treatment options. We advise patients to contact their physicians to discuss alternative treatment while we continue to investigate the findings from our review of the data.”

Patients, physicians, pharmacists, or other healthcare professionals with additional questions about OXBRYTA should contact Pfizer Medical Information 1-800-438-1985. The company will keep patients, regulatory authorities, investigators and clinicians informed about actions and appropriate next steps for OXBRYTA.

CARLSBAD, CA -  Ionis Pharmaceuticals, Inc. announced  that the U.S. Food and Drug Administration (FDA) has granted zilganersen Fast Track designation for the treatment of children and adults with an ultra-rare, progressive and ultimately fatal neurological disorder known as Alexander disease (AxD). Topline data from the pivotal study of zilganersen is expected in the second half of 2025. The FDA grants investigational medicines Fast Track designation to facilitate the development and expedite the review of medicines that demonstrate the potential to treat serious conditions and fill an unmet medical need.

"With no approved treatments available for people living with AxD, receiving this Fast Track designation for zilganersen reflects the seriousness of this ultra-rare disease and the significant unmet need for treatment in this patient population," said Eugene Schneider, M.D., executive vice president and chief clinical development officer at Ionis. "Zilganersen was designed to address the underlying cause of disease and help improve the functioning of people living with AxD. We look forward to a data readout next year and working closely with the FDA to potentially bring forward the first approved AxD treatment."

The Phase 1-3 study of zilganersen in adults and children living with AxD completed enrollment earlier this year across 13 sites in eight countries

PRINCETON, N.J. - Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has approved COBENFY™ (xanomeline and trospium chloride), an oral medication for the treatment of schizophrenia in adults. COBENFY represents the first new class of medicine in several decades and introduces a fundamentally new approach to treating schizophrenia by selectively targeting M ₁ and M ₄ receptors in the brain without blocking D ₂ receptors. 

“Today’s landmark approval of our first-in-class treatment for schizophrenia marks an important milestone for the community, where after more than 30 years, there is now an entirely new pharmacological approach for schizophrenia — one that has the potential to change the treatment paradigm,” said Chris Boerner, PhD , board chair and chief executive officer at Bristol Myers Squibb. “As we reenter the field of neuropsychiatry, we are dedicated to changing the conversation around serious mental illness, beginning with today’s approval in schizophrenia.”

Schizophrenia is a persistent and often disabling mental illness affecting how a person thinks, feels and behaves. It is estimated to impact approximately 2.8 million people in the United States. Symptoms typically first appear in early adulthood and present differently in each person, making symptoms difficult to diagnose and manage. While the current standard of care can be effective in managing symptoms of schizophrenia, up to 60% of people experience inadequate improvement in symptoms or intolerable side effects during therapy. 

“For people living with schizophrenia, it's often difficult to find a treatment that works for them. Having a variety of treatment options gives patients and healthcare providers the tools to help manage this serious condition,” said Gordon Lavigne, chief executive officer of the Schizophrenia & Psychosis Action Alliance. “People living with schizophrenia want and deserve more. Today's approval provides a new option as people with schizophrenia move forward with proper support to rebuild their lives.”

The FDA approval of COBENFY is supported by data from the EMERGENT clinical program, which includes three placebo-controlled efficacy and safety trials and two open-label trials evaluating the long-term safety and tolerability of COBENFY for up to one year. In the Phase 3 EMERGENT-2 and EMERGENT-3 trials, COBENFY met its primary endpoint, demonstrating statistically significant reductions of schizophrenia symptoms compared to placebo, as measured by the Positive and Negative Syndrome Scale (PANSS) total score change from baseline to week five. COBENFY demonstrated a 9.6-point reduction (-21.2 COBENFY vs. -11.6 placebo, p<0.0001) and an 8.4-point reduction (-20.6 COBENFY vs. -12.2 placebo; p<0.0001) in PANSS total score compared to placebo at week five in EMERGENT-2 and EMERGENT-3, respectively. In EMERGENT-2, COBENFY demonstrated a statistically significant improvement in illness from baseline to week five, as measured by the Clinical Global Impression-Severity (CGI-S) score, a secondary endpoint in the trial. ¹

The safety and tolerability profile of COBENFY has been established across acute and long-term trials. In the Phase 3 EMERGENT-2 and EMERGENT-3 trials, the most common adverse reactions (≥5% and at least twice placebo) were nausea, dyspepsia, constipation, vomiting, hypertension, abdominal pain, diarrhea, tachycardia, dizziness and gastroesophageal reflux disease. COBENFY does not have atypical antipsychotic class warnings and precautions and does not have a boxed warning.

“Due to its heterogeneous nature, schizophrenia is not a one-size-fits-all condition, and people often find themselves in a cycle of discontinuing and switching therapies,” said Rishi Kakar, MD, chief scientific officer and medical director at Segal Trials and investigator in the EMERGENT program. “The approval of COBENFY is a transformative moment in the treatment of schizophrenia because, historically, medicines approved to treat schizophrenia have relied on the same primary pathways in the brain. By leveraging a novel pathway, COBENFY offers a new option to manage this challenging condition.”

The Company today also announced the launch of COBENFY Cares™, a program designed to support patients who have been prescribed COBENFY. Patients will be able to enroll in the COBENFY Cares program in late October corresponding with product availability. The COBENFY Cares phone number is 1-877-COBENFY.

COLUMBUS, OH - Safecor Health is pleased to announce the appointment of Mark Saxon as its new Chief Executive Officer.

Saxon comes to Safecor Health with nearly 30 years of experience as a healthcare executive, healthcare provider, and a military veteran helping health systems improve quality, drive efficiencies, and reduce costs. Most recently, Saxon served as Market President for Navvis Healthcare, where he led population health strategies and developed multiple joint ventures and partnerships to grow the business and drive performance in value-based care.

"As I learned more and more about Safecor Health, it became clear that this is an outstanding opportunity," explained Saxon. "There's a market need for what we do, and we're solving real healthcare challenges. We're reducing medical errors, improving logistics, speeding time to patients, and reducing pressures for hospital staff. Hospital expenditures are expected to reach $2 trillion in the U.S. in 2024 and increasing hospital utilization is creating stresses that our current and future solutions can help solve."

Steve Fischbach, Safecor Health's co-founder and current CEO, is moving to the role of Executive Vice Chairman of the company's board of directors.

"Safecor is expecting strong growth in the coming years as we continue to expand our offerings and value proposition to our customers," stated Fischbach, "and I'm excited to continue in this board role supporting the company as Mark leads us through this next growth phase of our company."

Mark Parrish, Chairman of the Board of Directors at Safecor Health, added, "We're very pleased to have Mark join the team. He brings the energy and experience we need to realize our objectives in both growth and customer satisfaction."

In addition to his experience in the healthcare industry, both as an executive and as a Chief Physician Assistant in cardiothoracic surgery, Saxon is a veteran of the Persian Gulf War. He earned his MBA from Lindenwood University, his BS as a Physician Assistant from the Medical University of South Carolina, and his bachelor's degree from Northeast Missouri State University (now Truman State).

Safecor Health is a Vesey Street Capital Partners (VSCP) portfolio company.

Adults with opioid use disorder who receive a higher daily dose of the opioid addiction treatment medication buprenorphine may have a lower risk of subsequent emergency department visits or use of inpatient services related to behavioral health (such as for mental health and substance use disorders) than adults receiving the recommended dose, according to an analysis funded by the National Institutes of Health (NIH). These findings suggest that higher buprenorphine doses could be more effective in managing opioid use disorder, which may be particularly relevant for improving treatment for those who use fentanyl, a major driver of the overdose crisis.

Researchers reviewed insurance claims data from over 35,000 people who were diagnosed with opioid use disorder and began buprenorphine treatment between 2016 and 2021. They found that among all people who started treatment with buprenorphine, 12.5% experienced an emergency department or inpatient visit related to behavioral health within the study period. After adjusting for patient demographics and medical history available in the data, researchers then analyzed how long it took for people receiving different doses of buprenorphine to use emergency care or have an inpatient stay after starting treatment.

The recommended target dose for buprenorphine in the U.S. Food and Drug Administration (FDA)’s approved labeling is 16 mg per day. Researchers found that those taking higher daily doses of buprenorphine (>16 to 24 mg) took 20% longer to have a subsequent emergency department or inpatient health care visit related to behavioral health within the first year after receiving treatment, compared to those receiving >8 to 16 mg a day. Those taking daily doses of more than 24 mg of buprenorphine went 50% longer before having a subsequent emergency or inpatient health care visit related to behavioral health within the first year after receiving treatment, compared to those receiving >8 to 16 mg a day.

“As the overdose crisis evolves, particularly with the rise of fentanyl, it is crucial to investigate how to best adapt and deliver the life-saving and evidence-based treatments for opioid use disorder that we have available,” said Nora D. Volkow, M.D., director of NIH’s National Institute on Drug Abuse (NIDA). “The findings add to the growing evidence that higher doses of buprenorphine may have meaningful health impacts for people with opioid use disorder.”

In 2022, of the nearly 108,000 overdose deaths reported in the U.S., almost 70% were primarily due to fentanyl, a synthetic opioid that is approximately 50 times stronger than heroin. The ubiquity of fentanyl in the drug supply and associated overdose deaths have raised questions about whether existing dosing guidelines for buprenorphine should be modified to better address the unique challenges posed by such a potent opioid. Higher doses of buprenorphine may be necessary to effectively manage the more severe withdrawal symptoms, cravings, and tolerance associated with fentanyl use.

"Preventing or delaying the need for high-intensity, urgent health care among people with opioid use disorder has tremendous benefits on health and recovery," said Bradley D. Stein, M.D., Ph.D., a study co-author and director of the RAND-USC Schaeffer Opioid Policy Tools and Information Center. “As we continue to gather data across studies, findings suggest that higher doses of buprenorphine may have the ability to significantly improve treatment in the era of fentanyl, as both fatal and nonfatal overdoses remain unacceptably high.”

The authors also note that addressing barriers to accessing higher doses, such as state laws and insurance policies, will be important in ensuring that all patients receive effective care. In addition, revisiting guidelines that serve as barriers to higher doses could be beneficial, as these may limit access to potentially life-saving treatment for patients at high risk of relapse or overdose.

Notably, the data used in the study were pulled from a single commercial insurance company, which does not include uninsured people and those with Medicaid or fee-for-service Medicare coverage, and the sample of people included in the analysis was 75% non-Hispanic white. Further research is needed to explore the effects of higher buprenorphine doses in more diverse populations, including those with different insurance statuses or in different clinical settings. In addition, the authors note that future research should also investigate additional outcomes such as the long-term effects of high-dose buprenorphine on opioid use disorder treatment retention and overall health outcomes using similar data.

NEW HAVEN, CT — Biohaven Ltd. announced positive topline results from pivotal Study BHV4157-206-RWE (NCT06529146) demonstrating the efficacy of troriluzole on the mean change from baseline in the f-SARA after 3 years of treatment. The study achieved the primary endpoint and showed statistically significant improvements on the f-SARA at years 1 and 2 (Figure 1). SCA is a rare, progressively debilitating neurodegenerative disease that affects approximately 15,000 people in the United States and 24,000 in Europe and the United Kingdom. There are no FDA approved treatments for SCA.

Collectively, data across multiple analyses demonstrate a robust and clinically meaningful slowing of disease progression in SCA patients. These treatment benefits translate into a 50-70% slower rate of decline compared to untreated patients, representing 1.5-2.2 years delay in disease progression over the 3-year study period. Additionally, in a responder sensitivity analysis, disease progression when defined by a 2 point or greater worsening on the f-SARA at 3 years showed an odds ratio (OR) of 4.1 (95% CI: 2.1, 8.1) for the untreated external control arm versus troriluzole treated subjects (p < 0.0001; pooled analysis).

Dr. Susan Perlman, Director of Ataxia Clinic and Neurogenetics Clinical Trials at the David Geffen School of Medicine at UCLA stated, 

"SCA is a debilitating, relentlessly progressive disease that destroys quality of life, leaving patients unable to care for themselves, walk, or speak. Troriluzole is the very first treatment to show a delay in disease progression that can give patients additional years of independence, where they can walk without assistance, continue to work, play with their children, and participate in daily activities. This is an exciting and hopeful moment for the SCA community."

Study BHV4157-206-RWE was designed, in discussion with the US Food and Drug Administration (FDA), to assess the effectiveness of troriluzole in SCA after 3 years of treatment as measured by the change from baseline in the f-SARA. The study utilized Phase 3 data and an external control of matched, untreated SCA subjects from the US Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) in accordance with FDA's Guidance on Real-World Evidence (RWE) of effectiveness. All endpoints were prespecified, and both the study protocol and statistical analysis plan were submitted to, and reviewed by, FDA prior to topline data analysis. The new analysis doubled the previously available 3 year data with 63 subjects now completing 3 years of treatment with troriluzole and matched to the external control arm. Propensity Score Matching (PSM) was used to ensure that untreated patients from the CRC-SCA study were rigorously matched to treated patients from Study BHV4157-206 on baseline characteristics.  The primary objective was to examine the treatment effects of troriluzole for up to 3 years, by comparing data on the f-SARA from patients treated with troriluzole in Study BHV4157-206 to untreated patients from the natural history study. Troriluzole-treated patients demonstrated statistically significant and sustained benefits at years 1, 2 and 3 on the f-SARA compared to a rigorously matched natural history control.

Additionally, prespecified analyses in the protocol employed a separate, independent natural history control from the European SCA natural history study (EUROSCA) for global regulatory purposes. Results using the EUROSCA patients, in addition to a pooled analysis using both CRC-SCA and EUROSCA patients, as the external controls were also statistically significant and consistent with the primary efficacy analysis at all timepoints (see Figure 2 and Figure 3). The addition of EUROSCA data increased the external control sample size and added to the robustness of the statistically significant treatment differences at years 1, 2, and 3, favoring troriluzole.

Jeremy Schmahmann, M.D., Professor of Neurology at Harvard Medical School and Founding Director of the Ataxia Center at Massachusetts General Hospital commented, 

"The stabilization of SCA symptoms as reflected by the topline data at 3 years along with the previously reported reductions in falls show the therapeutic potential of troriluzole. I cannot underscore enough the impact of a potential treatment that can slow SCA disease progression and the effect on patients and caregivers who have helplessly watched generations of family members deteriorate and die from SCA. These new data provide support for troriluzole as a safe and effective once daily treatment for patients with SCA."

Spinocerebellar ataxia is a group of dominantly inherited neurodegenerative disorders characterized by progressive loss of voluntary motor control and atrophy of the cerebellum, brainstem and spinal cord. Patients experience significant morbidity, including progression to a wheelchair, impaired gait leading to falls, inability to communicate due to speech impairment, difficulty swallowing, and premature death. While signs and symptoms can appear anytime from childhood to late adulthood, SCA typically presents in early adulthood and progresses over a number of years. Currently, there are no FDA-approved treatments and no cure for SCA.

Based upon the topline data from Study BHV4157-206-RWE, and previous safety and efficacy data from the troriluzole development program in SCA, Biohaven plans to submit a New Drug Application (NDA) to the FDA in Q4 2024. The troriluzole development program has generated the largest clinical trial dataset in SCA and now has follow-up in some patients treated with troriluzole for over 5 years.  Biohaven has previously received both Fast-Track and Orphan drug designation (ODD) from the FDA, and ODD from the European Medicines Agency, for troriluzole in SCA. An NDA with ODD is eligible for priority FDA review. Biohaven will be prepared to commercialize SCA in the US in 2025, if ultimately approved, based on potential priority review timelines.

Roche recently announced that the United States Food and Drug Administration (U.S. FDA) has approved OCREVUS ZUNOVO™ (ocrelizumab & hyaluronidase-ocsq) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS). OCREVUS ZUNOVO is the first and only twice-a-year, healthcare professional (HCP)-administered approximately 10-minute subcutaneous (SC) injection approved for both these forms of multiple sclerosis, giving people living with MS more treatment options.

“OCREVUS ZUNOVO gives patients and providers another option for receiving OCREVUS, building on a decade of robust safety and efficacy data for OCREVUS in multiple sclerosis,” said Levi Garraway, M.D., Ph.D., Roche’s chief medical officer and head of Global Product Development. “This approval may offer greater flexibility for healthcare providers and people living with multiple sclerosis, based on their individual treatment needs.”

“People are living longer with chronic illnesses and with fewer disabilities because of the extensive progress that has been made in the development of medicines that can slow their progression,” said Natalie Blake, Executive Director of the MS Foundation. “But still, not everyone has access to these medicines. It’s crucial to acknowledge each experience with MS is as unique as the individual navigating it, so providing choices to address each person’s needs is essential. We are pleased that with a new method of delivery, there is now an additional option for those who need flexibility in the route of administration or treatment time.”

After the first dose, the time for treatment with OCREVUS ZUNOVO could be as little as 55 minutes. Patients will be required to take premedication at least 30 minutes prior to each dose. Following the first dose, patients will be monitored by their HCP for at least 60 minutes. Patients will be monitored for at least 15 minutes post-injection for subsequent doses.

The FDA approval is based on pivotal data from the Phase III OCARINA II trial, which showed no clinically significant difference in the levels of OCREVUS in the blood when administered subcutaneously, and a safety and efficacy profile consistent to the IV formulation in people with RMS and PPMS. Out of the exploratory outcomes measured, OCREVUS ZUNOVO was consistent with IV, demonstrating suppression of relapse activity (97%) and MRI lesions (97%) through 48 weeks. Additionally, one of several patient-reported outcomes measured during the study showed more than 92% of trial participants reported being satisfied or very satisfied with the SC administration of OCREVUS ZUNOVO.

In the Phase III OCARINA II trial, the safety profile of OCREVUS ZUNOVO was consistent with the well-established safety profile of OCREVUS® IV, with the exception of injection reactions. The most common adverse events with OCREVUS ZUNOVO were injection reactions. Injection reactions were more frequently reported with the first injection, with 49% of trial participants experiencing an injection reaction after the first injection. All injection reactions were either mild or moderate, and none led to treatment withdrawal.

OCREVUS ZUNOVO™ has the potential to expand treatment options to centers without IV infrastructure or with IV constraints, like at a doctor's office.

INDIANAPOLIS, IN - Eli Lilly and Company announced detailed results from the QWINT-5 phase 3 trial evaluating once-weekly insulin efsitora alfa (efsitora) compared to once-daily insulin degludec in adults with type 1 diabetes who require daily basal and multiple daily mealtime insulin injections. The data were published in The Lancet and simultaneously presented today at the European Association for the Study of Diabetes (EASD) Annual Meeting 2024.

In the trial, efsitora met the primary endpoint of non-inferior A1C reduction at week 26. For the efficacy estimand, efsitora reduced A1C by 0.53% compared to 0.59% for insulin degludec resulting in an A1C of 7.37% and 7.32% respectively³.

The time in range as measured by continuous glucose monitoring (CGM) was similar between efsitora and insulin degludec during the four weeks prior to week 26. In an additional key secondary endpoint, the estimated combined rates of patient-reported clinically significant (blood glucose <54 mg/dL) or severe nocturnal hypoglycemic events per patient-year of exposure were similar between efsitora and insulin degludec over the 52-week study period.

"People with type 1 diabetes need insulin every day. Currently, they can deliver the insulin using an automated insulin delivery system or by taking a daily basal insulin injection and multiple mealtime insulin injections each day," said Richard Bergenstal, M.D., executive director of the International Diabetes Center, HealthPartners Institute. "This new data shows that with one dose a week of basal insulin, efsitora was able to achieve a similar A1C reduction as taking an injection of one of the most used background insulins every day. I look forward to further evaluation of these data, including ways to minimize hypoglycemia, so once-weekly insulin can be one option for personalizing the management of type 1 diabetes."

In the trial, estimated combined rates of patient-reported clinically significant (blood glucose <54 mg/dL) or severe hypoglycemic events per patient-year of exposure through week 52 were 14.03 with efsitora vs. 11.59 with insulin degludec. There was no evidence of increased duration of hypoglycemia with efsitora compared to insulin degludec based on CGM data.

Estimated rates of severe hypoglycemic events per patient-year of exposure through week 52 were 0.14 with efsitora vs. 0.04 with insulin degludec. More than half (64%) of the reported severe hypoglycemic events with efsitora took place during the initial 12 weeks of the trial's treatment period and incidence of severe hypoglycemia in both treatment groups declined after week 12.

Overall incidence of treatment-emergent adverse events were comparable across treatment groups. Serious adverse events were higher in efsitora compared to insulin degludec, driven by severe hypoglycemic events.

"When we commercialized insulin more than 100 years ago, it marked the beginning of our commitment to people living with type 1 diabetes – today's announcement continues that legacy," said Jeff Emmick, M.D., Ph.D., senior vice president, product development, Lilly. "These results underscore the potential of efsitora to help some people living with type 1 diabetes lower their A1C with only one basal insulin injection per week, while also highlighting the complexity of treating this chronic disease. With the data we have seen from our phase 3 program so far, we are confident in efsitora's potential to transform diabetes care and will continue to pursue new treatment options until we can eliminate the disease entirely."

Detailed results for QWINT-2 are also being presented at EASD and simultaneously published in The New England Journal of Medicine.

Food and Drug Administration approved atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza, Genentech, Inc.) for subcutaneous injection for all the adult indications as the intravenous formulation of atezolizumab, including non-small cell lung cancer (NSCLC), small cell lung cancer (SCLC), hepatocellular carcinoma (HCC), melanoma, and alveolar soft part sarcoma (ASPS). See the prescribing information for the specific indications.

The subcutaneous injection of atezolizumab and hyaluronidase-tqjs was evaluated in IMscin001 (NCT03735121), an open-label, multi-center, international, randomized trial in adult patients with locally advanced or metastatic NSCLC who were not previously exposed to cancer immunotherapy and who had disease progression following treatment with platinum-based chemotherapy. A total of 371 patients were randomized (2:1) to receive subcutaneous atezolizumab and hyaluronidase-tqjs or intravenous atezolizumab until disease progression or unacceptable toxicity.

The primary outcome measure was atezolizumab exposure, with coprimary pharmacokinetic (PK) endpoints of Cycle 1 and AUC. Additional descriptive efficacy outcome measures were overall response rate (ORR), progression-free survival (PFS), and overall survival (OS). The geometric mean ratio (GMR) (90% CI) of subcutaneous atezolizumab and intravenous atezolizumab for Cycle 1 was 1.05 (0.88, 1.24) and AUC was 0.87 (0.83, 0.92), which met the lower limit of the GMR (90% CI) above the pre-specified threshold of 0.8 for comparability. No notable differences in ORR, PFS or OS were observed between the different formulations. The confirmed ORR was 9% (95% CI: 5, 13) in the subcutaneous atezolizumab and hyaluronidase-tqjs arm and 8% (95% CI: 4, 14) in the intravenous atezolizumab arm.

The most common adverse reactions of any grade (≥ 10%) were fatigue, musculoskeletal pain, cough, dyspnea, and decreased appetite.

The recommended dosage is one 15 mL injection (containing 1,875 mg of atezolizumab and 30,000 units of hyaluronidase) administered subcutaneously in the thigh over approximately 7 minutes every 3 weeks.

HORSHAM, PA - Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has approved TREMFYA^® (guselkumab) for the treatment of adults with moderately to severely active ulcerative colitis (UC), a chronic disease of the large intestine in which the lining of the colon becomes inflamed. TREMFYA^® is the first and only approved fully-human, dual-acting monoclonal antibody that blocks IL-23 while also binding to CD64, a receptor on cells that produce IL-23. IL-23 is a cytokine secreted by activated monocyte/macrophages and dendritic cells that is known to be a driver of immune-mediated diseases including UC.

"Treatment with TREMFYA resulted in significant improvement in the chronic symptoms of ulcerative colitis, and importantly, normalization in the endoscopic appearance of the intestinal lining," said David T. Rubin, MD, Director, Inflammatory Bowel Disease Center, University of Chicago Medicine, and lead investigator for the QUASAR program. "Today's approval of TREMFYA builds on the clinical and well-established safety profile of this IL-23 inhibitor and marks a significant step forward in the treatment of this chronic inflammatory disease."

The UC approval is supported by data from the ongoing Phase 2b/3 QUASAR study evaluating the efficacy and safety of TREMFYA^® in adult patients with moderately to severely active UC who experienced an inadequate response or who demonstrate intolerance to conventional therapy, other biologics and/or JAK inhibitors. 

"There is a significant need for new UC therapies that offer meaningful improvements in symptoms and the promise of remission, both overall clinical remission as well as delivering visible healing of the colon through endoscopic remission," said Christopher Gasink, MD, Vice President, Medical Affairs, Gastroenterology & Autoantibody, Johnson & Johnson Innovative Medicine. "In the QUASAR clinical program, TREMFYA demonstrated high reported rates of endoscopic remission at one year of treatment, continuing to raise the bar for efficacy in the treatment of this inflammatory bowel disease."

For the treatment of UC, TREMFYA^® is administered as a 200 mg induction dose intravenously at weeks zero, four and eight by a healthcare professional. The recommended maintenance dosage is 100 mg administered by SC injection at week 16, and every 8 weeks thereafter, or 200 mg administered by SC injection at week 12, and every 4 weeks thereafter. The SC maintenance dose can be self-administered by the patient or administered by a caregiver using TREMFYA^® after proper training. Use the lowest effective recommended dosage to maintain therapeutic response.