FDA Approves INLEXZO™ for BCG-Unresponsive Bladder Cancer

The U.S. FDA has approved Johnson & Johnson’s INLEXZO™ (gemcitabine intravesical system) for adults with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. This new therapy offers an alternative for patients who decline or are ineligible for bladder removal surgery (radical cystectomy).

Gemcitabine intravesical system is co-packaged with a urinary catheter and stylet used for insertion through the urinary catheter into the bladder.

Full prescribing information for Inlexzo will be posted on Drugs@FDA.

Efficacy was evaluated in Cohort 2 of SunRISe-1 (NCT04640623), a single-arm, multi-center trial enrolling 83 patients with BCG-unresponsive NMIBC with CIS with or without papillary tumors following transurethral resection. Patients received the gemcitabine intravesical system into the bladder every 3 weeks for 6 months, followed by once every 12 weeks for up to 18 months.

Tumor status was assessed with cystoscopy and urine cytology every 12 weeks during the initial two years of treatment, after which cystoscopy was performed at least every 24 weeks. Mandatory biopsies were performed at 24 and 48 weeks after treatment initiation.

The major efficacy outcome measures were complete response (CR) at any time (defined as negative results for cystoscopy [with TURBT/biopsies as applicable] and urine cytology) and duration of response (DOR). CR assessment at each timepoint was based on central pathology review.

The CR rate was 82% (95% CI: 72, 90) and 51% of patients with a CR had a DOR ≥ 12 months.

The prescribing information includes warnings and precautions describing risks of administering gemcitabine intravesical system in patients with a perforated bladder, risk of metastatic bladder cancer with delayed cystectomy, magnetic resonance imaging (MRI) safety, and embryo-fetal toxicity.

The gemcitabine intravesical system delivers 225 mg of gemcitabine into the bladder, with an indwelling period following each insertion of 3 weeks prior to removal. The gemcitabine intravesical system is inserted once every 3 weeks for up to 6 months (8 doses), followed by once every 12 weeks for up to 18 months (6 doses), or until persistent or recurrent high-grade NMIBC, disease progression, or unacceptable toxicity.

This review used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

This application was granted priority review. The gemcitabine intravesical system received breakthrough therapy designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

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Biologic Therapy may Lower Malignancy Risk in Psoriasis,suggests study

A new study has investigated the impact of biologic therapies on cancer risk among patients with psoriasis and found that these treatments may offer protective benefits compared to traditional topical therapies. Psoriasis is a chronic immune-mediated condition that often requires long-term management, and concerns about the potential for systemic therapies to influence malignancy risk have persisted among clinicians and patients alike. This research focused on comparing patients receiving biologic treatments, such as tumor necrosis factor inhibitors and interleukin-targeted agents, with those using only topical monotherapies. The study revealed that patients treated with biologic therapies experienced a lower incidence of malignancies over the observation period. While topical monotherapy is often considered safe and effective for managing mild disease, it does not provide systemic immunomodulatory effects that may influence the immune surveillance mechanisms associated with cancer development. Biologic therapies, by modulating specific inflammatory pathways implicated in both psoriasis and cancer progression, may reduce the risk of malignancy while simultaneously controlling skin disease. The findings underscore the importance of considering personalized treatment strategies for patients with psoriasis, especially those at elevated risk for cancer due to age, family history, or comorbidities. Clinicians are encouraged to weigh both dermatologic benefits and systemic protective effects when recommending biologic therapy. Furthermore, the study highlights the need for ongoing surveillance and long-term follow-up to better understand the relationship between immune-targeted treatments and malignancy risk in this population. While biologic therapies have previously been scrutinized for their immunosuppressive potential, this research provides reassurance that, when appropriately selected and monitored, these treatments may confer additional systemic benefits beyond skin clearance. Patients and healthcare providers should engage in shared decision-making, considering both disease severity and individual risk factors for malignancy, to optimize treatment outcomes and overall health.

Keywords: Psoriasis, biologic therapy, malignancy risk, cancer prevention, immune modulation, systemic therapy, tumor necrosis factor inhibitors, interleukin inhibitors, personalized medicine, long-term outcomes

Reference:
Zhang, Y., et al. Biologic therapy is associated with reduced malignancy risk in psoriasis: A cohort study. Journal of the American Academy of Dermatology. 2025. https://doi.org/10.1016/j.jaad.2025.07.015

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Valgus Knee Brace Eases Walking Pain in Osteoarthritis Patients: Trial Shows

Netherlands: Wearing a valgus knee brace can meaningfully reduce walking-related pain in patients with medial compartment knee osteoarthritis (OA), according to a randomized controlled trial published in PLOS One. After six months of use, patients reported both statistically significant and clinically important improvements in pain intensity during walking, along with a reduced reliance on pain medications.

The study, led by Corné J.M. van Loon from the Department of Orthopaedics, Rijnstate Hospital, Arnhem, The Netherlands, and colleagues, aimed to address a gap in prior research by not only assessing clinical outcomes but also incorporating patients’ perspectives on brace use. “Our trial evaluated both the effectiveness and the user experiences of the valgus brace, considering aspects such as wearing time, comfort, and perceived benefits, which are often overlooked in earlier studies,” the authors noted.
In this mixed-method randomized controlled trial, 46 participants with medial compartment knee OA were enrolled. Twenty-three participants in the intervention group received standard care plus a customized valgus brace, while the remaining 23 participants in the control group received regular care but no brace. Outcomes were assessed at baseline, 2 weeks, 3 months, and 6 months, with knee pain during walking, measured on a 10-cm Visual Analog Scale (VAS), being the primary outcome. Secondary measures included walking distance, overall health status, knee functioning, and satisfaction with the brace. To enrich the findings, qualitative interviews were conducted with a subgroup of brace users.
The key findings of the study were as follows:
  • At six months, the intervention group showed a 2.13 cm greater reduction in knee pain after walking compared to the control group.
  • This reduction was both statistically significant and clinically meaningful.
  • No significant improvements were noted for pain at rest or for physical functioning measures.
  • Qualitative findings revealed that many patients experienced benefits beyond pain relief, such as better body function and improved ability to perform activities.
  • Some participants reported mixed or negative views of the brace, while several others described positive overall experiences.
The authors emphasized that although their study demonstrated measurable pain reduction, the relatively small sample size may have limited the ability to detect additional benefits. They highlighted the need for larger, well-powered trials with longer follow-up to better understand the effectiveness of valgus braces. Further exploration of patient factors—such as age, activity level, BMI, and degree of OA—was also recommended, as these could influence both outcomes and user satisfaction.
Importantly, the study highlights that user experience plays a crucial role in determining the success of assistive devices like knee braces. Some participants suggested that factors such as comfort, fit, and wearing duration strongly influenced their perception of benefit. These insights may prove valuable for healthcare providers, manufacturers, and patients alike, guiding future brace design and prescription practices.
“The findings suggest that valgus bracing offers a promising, non-invasive option for alleviating walking-related pain in individuals with medial knee OA. While broader research is required, incorporating patient feedback alongside clinical outcomes could help refine and optimize brace interventions, ultimately improving quality of care for those living with osteoarthritis,” the authors concluded.
Reference:
(2025). Effectiveness and user experiences of a valgus brace in patients with knee osteoarthritis: A mixed-method randomised controlled trial. PLOS ONE, 20(9), e0330157. https://doi.org/10.1371/journal.pone.0330157

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Preemptive Genetic Testing Feasible for Reducing Chemotherapy Toxicity in GI Cancer, Finds Trial

USA: A prospective trial in gastrointestinal cancer patients found that preemptive DPYD/UGT1A1 genetic testing before chemotherapy was feasible, enabled timely results in over half of cases, and showed potential to lower treatment-related side effects and modifications.   

The research, led by Dr. Sony Tuteja from the Perelman School of Medicine at the University of Pennsylvania and published in JCO Precision Oncology, explored whether screening for genetic variants prior to starting chemotherapy could guide drug dosing and improve patient safety. The focus was on two pharmacogenes—DPYD, which influences the breakdown of fluoropyrimidines, and UGT1A1, which affects the metabolism of irinotecan. Variants in these genes can increase the risk of severe toxicity from standard chemotherapy doses.

A total of 288 adults with gastrointestinal cancers were enrolled, and 225 received chemotherapy regimens qualifying for evaluation. All underwent genetic testing before initiating treatment, and when significant variants were identified, oncologists were advised to reduce the drug dose accordingly. The median turnaround time for test results was 10 days, and 57.4% of reports were available before the first treatment cycle. Among patients with actionable drug–gene interactions, nearly 70% had results in time to allow dose adjustments, and clinicians consistently followed the genotype-based recommendations.

Key findings:

  • Severe treatment-related adverse events occurred in 38% of the tested group compared with 65% in the control group, though the difference was not statistically significant.
  • Treatment modifications were significantly lower in the tested group at 38% compared with 76% in the control group.
  • Treatment discontinuations decreased from 47% in the control group to 31% in the tested group.
  • In patients carrying high-risk genetic variants, the incidence of severe toxicity dropped to rates similar to those seen in patients without such variants.

The trial also revealed practical challenges. In some cases, chemotherapy began before results were available, often because treatment needed to start urgently. The limited number of variant carriers reduced statistical power for some analyses, and differences in disease stage and performance status between groups may have influenced toxicity rates. The study did not track all adverse effects using formal grading criteria but instead recorded the most severe events, such as those requiring hospitalization or emergency care.

Despite these constraints, the findings support the value of incorporating DPYD/UGT1A1 testing into routine oncology practice for gastrointestinal cancers. By identifying patients at greater risk of drug-related toxicity before treatment starts, clinicians can tailor chemotherapy dosing to improve safety without sacrificing efficacy.

Dr. Tuteja and colleagues suggest that expanding access to genetic testing and ensuring results are available before treatment begins could further enhance outcomes, with future work aimed at streamlining testing processes and broadening adoption across cancer treatment centers.

Reference:

Sony Tuteja et al. Implementation of DPYD and UGT1A1 Testing in Patients With GI Cancer: A Prospective, Nonrandomized Clinical Trial. JCO Precis Oncol 9, e2500086(2025). DOI:10.1200/PO-25-00086

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Oral Berotralstat Effective and Safe for HAE Prevention in Young Children: Study

A newly published retrospective cohort study (APeX‑P trial) in Journal of Allergy and Clinical Immunology: In Practice found that oral berotralstat—a once‑daily plasma kallikrein inhibitor—was well tolerated and highly effective in reducing hereditary angioedema (HAE) attack rates in children aged 2–11 years. Among the 29 pediatric participants (median age 8), berotralstat led to early and sustained reductions in attack frequency: whereas the baseline median attack rate was 0.96 per four weeks, most children maintained zero to near‑zero attack rates through week 48. There were no drug‑related serious adverse events, discontinuations, or Grade 3/4 toxicities. Mild common side effects included nasopharyngitis, headache, and upper respiratory infections. pharmacokinetics showed a median Tmax of 3.9 hours and consistent exposure levels, supporting reliable once‑daily dosing. These findings suggest berotralstat could be a game‑changer as a first approved oral prophylactic option for children under 12, a population previously limited to injectable prophylaxis. Given its safety and early efficacy, this therapy offers a promising long‑term management strategy that may improve quality of life and reduce the treatment burden for young HAE patients. Experts note that further real‑world studies and longer follow‑up are needed to confirm durability and generalizability across diverse pediatric groups. 

Reference:
Bernatoniene, J., Bourgoin‑Heck, M., Cancian, M., Yang, W., Hagin, D., Pagnier, A., … Stobiecki, M. (2025). Oral berotralstat prophylaxis in children aged 2–11 years with hereditary angioedema: interim results from the APeX‑P trial. JACI: In Practice. https://doi.org/10.1093/jac/dkaf265

Keywords: berotralstat, hereditary angioedema, pediatric prophylaxis, HAE prevention, oral plasma kallikrein inhibitor, attack reduction, safety in children, APeX‑P trial, quality of life, orladeyo

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Parents of children with health conditions less confident about a positive school year

As the new school year begins, some parents may be feeling more nervous than excited—especially those whose children have different health needs.

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Trauma-focused therapy shows promise for children struggling with PTSD

A specialist form of therapy could offer hope for some of the most vulnerable young PTSD sufferers, according to a University of East Anglia study.

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Higher doses of semaglutide can safely enhance weight loss for adults living with obesity, clinical trials confirm

A higher weekly dose of semaglutide (7.2 mg) can significantly improve weight loss and related health outcomes in adults living with obesity, including those with type 2 diabetes (T2D), according to the results of two large-scale, international phase 3 clinical trials.

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Gestational diabetes linked to cognitive decline in mothers, increased risk of neurodevelopmental disorders in children

A new synthesis of global evidence finds that experiencing gestational diabetes during pregnancy is linked with a decline in intellectual function among mothers, and may increase the risk of neurodevelopmental disorders, including autism spectrum disorder and attention-deficit/hyperactivity disorder (ADHD) in children.

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Overweight and obesity don’t always increase the risk of an early death, Danish study finds

It is possible to be “fat but fit,” new research presented at the annual meeting of the European Association for the Study of Diabetes (EASD) held in Vienna, Austria (15–19 September) suggests.

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