Pulse Newsletter | Volume 7 - Issue 19

FDA Approves Inlexzo™ for Treatment of Bladder Cancer

On September 9, 2025, the U.S. Food and Drug Administration (FDA) approved Inlexzo™ (gemcitabine intravesical system) for the treatment of adult patients with a specific type of bladder cancer, called Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), which is found in about 10% of patients with NMIBC. BCG is an immunotherapy standard of care treatment for NMIBC. If patients become unresponsive to or fail BCG treatment, bladder removal surgery may be recommended, which is invasive and may be high risk for patients with bladder cancer, who tend to be older adults. Treatment with Inlexzo™ is an alternative to bladder removal surgery for patients meeting the clinical criteria. Inlexzo™ was granted priority review by the FDA in July, allowing the drug to quickly come to market. Priority review is given to drugs that are considered to be a significant breakthrough in therapy for a specific disease that may have limited treatment options.

Inlexzo™ was approved based on the results of the SunRISe-1 study, a single-arm, open label, phase 2b clinical study including 85 adult patients. The study evaluated the safety and efficacy of Inlexzo™ based on complete response (no signs of cancer after receiving therapy) to the drug and duration of response. In the study, 82% of patients had a complete response to therapy and 51% maintained their response to therapy for at least one year.

Inlexzo™ is an intravesical drug releasing system (iDRS) which works by delivering the drug (225mg of gemcitabine) directly into the bladder. The drug stays in the bladder for three weeks at a time per treatment cycle for up to six months (eight treatment cycles), then may be administered once every 12 weeks for six additional treatment cycles, for a total of up to 14 treatment cycles. Inlexzo™ is inserted and removed by a doctor in an outpatient setting, such as a provider’s office. The most common adverse reactions associated with treatment were increased urinary frequency, urinary tract infection, pain or discomfort during urination, bladder irritation, and abnormal lab findings, including decreased hemoglobin and increases in liver enzymes. Warnings and precautions for use of Inlexzo™ include avoiding use during pregnancy due to risk of harm to an unborn baby and magnetic resonance imaging (MRI) safety. The package labeling advises patients to notify their health care provider that they are being treated with Inlexzo™ before having an MRI scan and to carry an MRI safety information card. Inlexzo™ is contraindicated in patients with perforation of the bladder. 

FDA To Close Loophole on Direct-to-Consumer Drug Advertising

On September 9, 2025, the FDA, along with the U.S. Department of Health and Human Services, issued notifications to the public regarding the implementation of stricter enforcement of how pharmaceutical companies can advertise directly to consumers (DTC) to help to improve patient education and consumer safety. In 1997, an FDA rule created the “adequate provision” loophole which allows pharmaceutical companies to include only some of the major safety concerns or adverse effects associated with a drug in their DTC ads if they also provide consumers with contact information to receive the drug’s complete safety profile. This has created concern regarding public confusion over the risks and benefits associated with prescription medications as well as concerns over the impact of DTC advertising on prescribing practices if drugs are specifically requested by patients after seeing advertisements on television and social media.

The federal government is closing the loophole and will mandate that pharmaceutical companies include the complete safety profile in their advertisements to consumers to help improve transparency and provide more detailed clinical information for consumers to make well-informed treatment decisions. According to their website, the FDA has recently issued thousands of warning letters to pharmaceutical companies to adjust or remove what the FDA deems deceptive drug advertisements. The current laws require pharmaceutical companies to not exaggerate a drug’s clinical benefits and to provide accurate information to consumers regarding a drug’s safety and efficacy profile, including potential side effects. However, according to the FDA, enforcement over the years has steadily declined, with zero warning letters being sent to pharmaceutical companies in 2024. In addition, with the rise of DTC advertising on social media platforms, there are concerns regarding paid spokespersons that may lead consumers to believe a drug is safe and effective without substantial clinical evidence to verify the information. According to the FDA, there will also be an attempt to expand their oversight of DTC advertising to discourage misleading information on social media platforms. According to the Congressional Budget Office, the billions of dollars spent by pharmaceutical companies on DTC advertising has led to increases in market demand for advertised drugs and has had a significant financial impact on drug spending.

FDA Approves New Extended-Release Dosage Form of Otezla

On August 29, 2025, the FDA approved Otezla XR™ (apremilast) tablets for the treatment of adult patients and pediatric patients ages 6 and older who weigh at least 110 pounds with active psoriatic arthritis (a type of arthritis linked to psoriasis that causes joint pain and swelling) or plaque psoriasis (an autoimmune disease causing thick patches of itchy, scaly skin). Otezla XR™ is also FDA-approved for treatment of adult patients with ulcers associated with Behçet’s disease (a rare autoimmune condition causing inflammation in the blood vessels and painful ulcers). Otezla XR™ is an extended-release tablet that is taken once daily.

Otezla® twice-daily tablets were first FDA approved in 2014 for the treatment of adult patients with active psoriatic arthritis and plaque psoriasis. In 2019, Otezla® twice-daily tablets received FDA approval for treatment of adult patients with Behçet’s disease. Last year, Otezla® twice-daily tablets received FDA approval for the treatment of pediatric patients 6 years and older with plaque psoriasis. After an initial 5-day dose titration (starting with a low dose and gradually increasing the dose) with Otezla® twice-daily tablets, the recommended maintenance dose of Otezla XR™ tablets is 75mg taken once daily. Dose titration is recommended to minimize the more common gastrointestinal side effects, including diarrhea, nausea, and vomiting. Other side effects associated with Otezla® and Otezla XR™ include depression or mood changes and weight loss, which should be closely monitored throughout the course of therapy. Otezla XR™ is not recommended for use in adult or pediatric patients with severe kidney impairment due to increased drug exposure in the body and lack of safety and efficacy data in this patient population. Otezla XR™ tablets should not be split and may be taken with or without food. Taking a medication once daily versus twice daily may help to simplify a patient’s medication regimen, especially for a patient taking multiple medications throughout the day, and therefore, may help to improve a patient’s medication adherence. However, providers should always consider the risks versus benefits specific to their individual patient’s needs when evaluating any medication regimen. Switching a patient between Otezla® twice-daily tablets and Otezla XR™ tablets is acceptable per the drug’s package labeling. Generic equivalents of Otezla® twice-daily tablets are not expected to be available until at least 2028 due to manufacturer drug patents.

New FDA Approvals

New Drug: Wayrilz™ (rilzabrutinib)

Oral tablets approved for the treatment of adult patients with chronic immune thrombocytopenia (ITP) who have not responded to a previous treatment. ITP is a blood disorder associated with low platelet counts which can prevent the blood from clotting appropriately and lead to bleeding. [8/29/2025 – Genzyme Corp.]

New Generics Entering the Marketplace

Saxenda® (liraglutide)

Indication: For the treatment of obesity in adults and pediatric patients aged 12 and older

Dosage Form/Strength: 18mg/3ml pen solution for injection

Average Wholesale Price (AWP): Generic = $308/pen | Brand = $324/pen