Bridget Hoyt

The FDA has approved Inlexzo for adults with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ.

The U.S. Food and Drug Administration (FDA) has approved Inlexzo (gemcitabine intravesical system) for adults with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors, the agency announced, noting that the system is co-packaged with a urinary catheter and stylet used for insertion through the urinary catheter into the bladder.

The treatment’s effectiveness was determined in cohort 2 of the SunRISe-1 trial, which enrolled 83 patients with BCG-unresponsive NMIBC with CIS with or without papillary tumors following transurethral resection, with patients receiving Inlexzo into the bladder every three weeks for six months, followed by once every 12 weeks for up to 18 months.

The system delivers 225 mg of gemcitabine chemotherapy into the bladder, with an indwelling period following each insertion of three weeks prior to removal, and the system is inserted once every three weeks for up to six months, or eight doses, and then once every 12 weeks for up to 18 months, or six doses), or until persistent or recurrent high-grade NMIBC, disease progression or unacceptable toxicity.

The treatment, previously known as TAR-200, was granted priority review by the 

Tumor status, as stated in the FDA’s announcement, was assessed via cystoscopy and urine cytology every 12 weeks during the initial two years of treatment, after which cystoscopy was performed at least every 24 weeks, and biopsies were performed at 24 and 48 weeks after treatment initiation.

 and 51% of patients with a complete response had a duration of response of at least one year.

The treatment’s prescribing information, the FDA noted, includes warnings and precautions describing the 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.

“I see many patients that ultimately become BCG-unresponsive and often face life-altering bladder removal. These patients now may be ideal candidates for newly approved Inlexzo,” said Dr. Sia Daneshmand, principal investigator of the clinical trial and professor of urology and director of urologic oncology at the Norris Comprehensive Cancer Center, Keck School of Medicine of University of Southern California, in a statement included in a news release from Johnson & Johnson. “In my experience, Inlexzo is well-tolerated and delivers clinically meaningful results. This will change the way we treat appropriate patients that haven’t responded to traditional therapy.”

“We are proud of the science that has brought us to this historic moment,” said Dr. John Reed, executive vice president of R&D, Innovative Medicineat Johnson & Johnson, in the news release. “Inlexzo is a novel therapy with powerful efficacy and demonstrated safety profile. As the only major healthcare company that hosts both pharmaceuticals and medical devices, we leveraged the speed and scale of Johnson & Johnson to accelerate innovation and deliver this important therapy to patients."

“At BCAN, our mission has always been to advocate for better todays and more tomorrows for everyone impacted by bladder cancer. This approval represents the kind of progress that brings new options to a community that urgently needs them,” said Meri-Margaret Deoudes, CEO, Bladder Cancer Advocacy Network (BCAN), in the news release. “Patients with bladder cancer need guidance and collaboration with providers to navigate bladder-sparing treatment options, including newly approved treatments like Inlexzo, so they can move forward feeling well-informed and confident.”

For more news on cancer updates, research and education, 

don’t forget to subscribe to CURE®’s newsletters here.

“FDA approves gemcitabine intravesical system for non-muscle invasive bladder cancer,” FDA; https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-gemcitabine-intravesical-system-non-muscle-invasive-bladder-cancer?check_logged_in=1

“U.S. FDA approval of INLEXZO™ (gemcitabine intravesical system) set to transform how certain bladder cancers are treated,” news release, Sept. 9, 2025; https://www.multivu.com/johnson-and-johnson/9342851-en-johnson-and-johnson-fda-approval-inlexzo-gemcitabine-intravesical-system

Articles

The FDA has approved 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. The approval is based on the SunRISe-1 trial, which demonstrated an 82% complete response rate. Inlexzo is administered intravesically every three weeks for six months, then every 12 weeks for up to 18 months. The treatment is well-tolerated and offers a new option for patients who have not responded to traditional therapies, potentially avoiding bladder removal.

FDA Approves Inlexzo for Some With Non–Muscle Invasive Bladder Cancer

For some with previously untreated locally advanced or metastatic non-small cell lung cancer Rybrevant plus Lazcluze improved overall survival.

Among patients with previously untreated locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions or L858R substitution mutations, treatment with Rybrevant (amivantamab) plus Lazcluze (lazertinib) was found to result in a statistically significant and clinically meaningful improvement in overall survival versus treatment with Tagrisso (osimertinib) monotherapy, study results have shown.

Results from the phase 3 MARIPOSA study were published in 

 and announced in a news release from Janssen-Cilag International NV, a Johnson & Johnson company.

At a median follow-up time of 37.8 months, the treatment combination of Rybrevant and Lazcluze was associated with a 25% lower risk of death than treatment with Tagrisso. The news release noted that the median overall survival time for the combination is expected to exceed four years versus the median overall survival time of 36.7 months observed with Tagrisso monotherapy.

Overall survival: the time a patient lives, regardless of disease status.

Progression-free survival: the time a patient lives without their disease spreading or worsening.

Paronychia: skin infection around the nail.

“This is a turning point in how we treat EGFR-mutated lung cancer,” said Dr. James Chih-Hsin Yang of the Department of Medical Oncology at the National Taiwan University Cancer Center in Taipei, Taiwanin the news release. “We’re now seeing the potential for patients to live significantly longer than we thought possible. Starting with [Rybrevant] plus [Lazcluze] may prevent common types of resistance and reserves chemotherapy for later lines of therapy, which can help achieve better outcomes.”

Yang was the lead author of the MARIPOSA study.

“These published data further reinforce the potential of the chemotherapy-free, dual-targeted [Rybrevant] plus [Lazcluze] regimen to deliver meaningful, long-term clinical outcomes and experiences for patients and their families versus the current standard of care,” said Henar Hevia, senior director of and head of EMEA Therapy Area for oncology at Johnson & Johnson Innovative Medicine.

Among patients who received the treatment combination, the median time to subsequent therapy was 30.3 months versus 24 months with Tagrisso. The combination was also associated with improved progression-free survival after subsequent therapy, with times of 42.9 months versus 32.8 months in the Rybrevant and Tagrisso treatment groups, respectively.

“With these results, we have the potential to change the trajectory of this disease,” said Dr. Joshua Bauml, vice president and disease area leader of lung cancer at Johnson & Johnson Innovative Medicine. “Unmet needs remain high for people living with EGFR-mutated lung cancer. Now, we’re delivering survival outcomes that have the ability to redefine what first-line treatment can achieve.”

The combination’s safety profile was reportedly consistent with the primary analysis, with the most common treatment-related side effects of any grade being paronychia (69%), infusion-related reaction (65%) and rash (64%). The most common grade 3 (severe) or worse side effects were rash (17%), paronychia (12%), dermatitis acneiform (9%), pulmonary embolism (9%) and alanine transaminase increase (7%).

Overall survival findings from MARIPOSA were 

 earlier this year in a presentation at the 2025 European Lung Cancer Congress, as well as published in a news release from Johnson & Johnson.

“Rybrevant plus Lazcluze helps patients live longer, and the benefit keeps growing over time,” trial investigator, Dr. Nicolas Girard, said in a news release issued at the time. “We see the gap between the survival curves continue to widen, which is exactly what we want to see in lung cancer treatment to improve outcomes for patients. These results reinforce that we are entering a new era for EGFR-mutated non-small cell lung cancer; with this evidence in hand, we need to ensure every patient gets the most effective treatment in the first line for the best possible chance at longer survival.”

Girard, who also presented these data at the meeting, is the head of Medical Oncology at the Institut Curie and a professor of Thoracic Oncology and Respiratory Medicine at the Paris-Saclay University, in France.

For more news on cancer updates, research and education,

“Data published in The New England Journal of Medicine demonstrate RYBREVANT®(amivantamab) plus LAZCLUZE® (lazertinib) could re-set survival expectations in first-line EGFR-mutated advanced lung cancer,” news release, Sept.7, 2025; https://www.globenewswire.com/news-release/2025/09/07/3145711/0/en/Data-published-in-The-New-England-Journal-of-Medicine-demonstrate-RYBREVANT-amivantamab-plus-LAZCLUZE-lazertinib-could-re-set-survival-expectations-in-first-line-EGFR-mutated-advan.html

“First-line Rybrevant Combo Extends Survival in Advanced Lung Cancer,” CURE, March 31, 2025; 

https://www.curetoday.com/view/first-line-rybrevant-combo-extends-survival-in-advanced-lung-cancer

A combination therapy of Rybrevant (amivantamab) and Lazcluze (lazertinib) has shown significant improvement in overall survival for patients with untreated locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR mutations, compared to Tagrisso (osimertinib) monotherapy. The phase 3 MARIPOSA study revealed a 25% reduction in mortality risk and extended median overall survival beyond four years. The combination also demonstrated improved progression-free survival and delayed the need for subsequent therapy. The safety profile was consistent, with manageable side effects.

Rybrevant Plus Lazcluze Associated with Survival Improvement in NSCLC

CURE spoke with the patient advocate and author of “Between Two Kingdoms” and “The Book of Alchemy.”

Suleika Jaouad on Why Self-Advocacy Matters for Patients With Cancer

After receiving her first diagnosis of leukemia at age 22, author and patient advocate Suleika Jaouad is now facing cancer for the third time in 15 years.

Jaouad, the author of the New York Times column “Life, Interrupted” and the books “Between Two Kingdoms” and “The Book of Alchemy: A Creative Practice for an Inspired Life,” now works with 

, the organization formerly known as the Leukemia and Lymphoma Society.

 to share her advice for patients facing a diagnosis of cancer.

When I first got diagnosed, I was focused on treating and hopefully curing my leukemia. What I wasn’t as focused on was the impact it would have on my whole self and on my whole life. My very first piece of advice that I give to patients is to seek out support groups, to find therapy, to make sure that you’re attending to the more invisible imprints of illness on your mental health, on your work, and on your whole self.

No matter how brilliant and wonderful your medical team might be, learning to advocate for yourself is a crucial part of becoming a patient. I learned that through the misdiagnoses that led to my actual diagnosis, I learned that in Googling late one night the side effects of the chemotherapy I was about to start — and learning that they were going to make me infertile — and having to push to make time for fertility preservation treatments.

These are all things that may seem like an aside to cancer, but, to me, are an important part of making sure that you are taking into consideration who you are as an individual, not just as your medical record number, and whatever your particular priorities might be.

Every patient is different. At 22, I approached my new diagnosis like a journalist might. I wanted to read every study I could get my hands on. I wanted second and third opinions. I scoured the internet for information, and I lived by the notion of “knowledge is power.” This time around, given that it’s my third time going through this, the internet is not a useful place for me. In fact, it’s a scary place for me to go. So I’ve appointed a friend of mine who serves the role of advocate, who’s doing that research, who’s contacting doctors for second opinions.

However it is that you want to manage it, whether you want to be the collector of the information or you want to appoint someone in your family or friend group to help you manage that, pushing beyond the information that’s immediately presented to you and making sure you have all the information you need to choose the appropriate treatment path and supportive services for yourself is essential.

Transcript has been edited for clarity and conciseness.

For more news on cancer updates, research and education, don’t forget to 

Videos

The FDA has granted orphan drug designation to the investigational therapy BA-101 for the treatment of patients with glioblastoma.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the investigational therapy BA-101 for the treatment of patients with glioblastoma.

The advancement was announced in a news release from NeuroNOS, a biopharmaceutical company that is a subsidiary of Beyond Air and manufactures BA-101.

The FDA, as the agency explains on its website, grants orphan drug designation to a drug or biological product used to prevent, diagnose or treat a rare disease or condition, and this designation qualifies sponsors for incentives such as tax credits for clinical trials, exemption from user fees and as many as seven years of market exclusivity after approval.

“Glioblastoma is one of the most common and deadliest brain cancers in adults, however, patients have seen little improvement in treatment options over the past several decades. Emerging industry research shows that [nitric oxide] is an important modulator of biological therapy response in glioblastoma,” said Amir Avniel, CEO of NeuroNOS, in a statement included in the news release. “We believe this data and the urgent unmet medical need have highlighted the opportunity for our groundbreaking science to develop small molecule therapies that balance nitric oxide levels in the brain. We believe [a nitric oxide] inhibition strategy has the potential to transform outcomes for patients.”

Glioblastoma, as defined by the National Cancer Institute, is a fast-growing type of central nervous system tumor that forms in the supportive glial tissue of the brain and spinal cord and has cells that look very different than typical cells. Glioblastoma usually occurs in adults and affects the brain more often than the spinal cord.

“Glioblastoma represents a profound unmet need," said Prof. Haitham Amal, chief science officer of NeuroNOS, in a statement of the news release. “Our published papers and unpublished data showed a strong link between [nitric oxide] and [glioblastoma]”.

Amal continued, “We are committed to working closely with regulators, investigators, patient groups, and foundations to accelerate development of BA-101 toward first-in-human studies.”

Surgery, radiation, and temozolomide chemotherapy are standard of care for patients with glioblastoma and can potentially extend survival, but they do not cure glioblastoma, according to the news release from NeuroNOS. Median survival of the disease is less than 12 months with two- and five-year survival rates lower than 20% and 10%, respectively, according to the news release.

According to the American Cancer Society, in 2025 approximately 24,820 malignant tumors of the brain or spinal cord, including 14,040 in males and 10,780 in females, will be diagnosed. Likewise, about 18,330 people, 10,170 males and 8,160 females, will die from brain and spinal cord tumors.

Study findings in mice published earlier this year in 

 stated that “combining BA-101 with the antitumor drug temozolomide (TMZ) synergistically reduced tumor size and significantly increased survivability in mice bearing TMZ-sensitive cells.”

“NeuroNOS Granted FDA Orphan Drug Designation for Glioblastoma, the Most Common and Deadliest Primary Malignant Brain Cancer in Adults,” news release, Sept. 8, 2025; https://www.globenewswire.com/news-release/2025/09/08/3146057/0/en/NeuroNOS-Granted-FDA-Orphan-Drug-Designation-for-Glioblastoma-the-Most-Common-and-Deadliest-Primary-Malignant-Brain-Cancer-in-Adults.html

“Designating an Orphan Product: Drugs and Biological Products,” FDA; https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products

“Glioblastoma,” National Cancer Institute; https://www.cancer.gov/publications/dictionaries/cancer-terms/def/glioblastoma

“Key Statistics for Brain and Spinal Cord Tumors,” American Cancer Society; https://www.cancer.org/cancer/types/brain-spinal-cord-tumors-adults/about/key-statistics.html

The FDA has granted orphan drug designation to BA-101, an investigational therapy for glioblastoma, developed by NeuroNOS. This designation provides incentives like tax credits and market exclusivity. Glioblastoma, a fast-growing brain cancer, has limited treatment options, with standard care including surgery, radiation, and temozolomide chemotherapy. BA-101, which modulates nitric oxide levels, shows promise in preclinical studies, especially when combined with temozolomide. The median survival for glioblastoma is under 12 months, highlighting the urgent need for new therapies to improve patient outcomes.

Bridget Hoyt

FDA Approves Inlexzo for Some With Non–Muscle Invasive Bladder Cancer

Rybrevant Plus Lazcluze Associated with Survival Improvement in NSCLC

Suleika Jaouad on Why Self-Advocacy Matters for Patients With Cancer

FDA Grants Orphan Drug Designation to BA-101 in Glioblastoma

Latest News