On April 22, 2024, the U.S. Food and Drug Administration (FDA) approved nogapendekin alfa inbakicept-pmln (Anktiva®) with Bacillus Calmette-Guérin (BCG) for adult patients with BCG-unresponsive non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.
Efficacy was evaluated in QUILT-3.032 (NCT0302285), a single-arm, multicenter trial of 77 patients with BCG-unresponsive, high-risk NMIBC with CIS with or without Ta/T1 papillary disease following transurethral resection. Patients received nogapendekin alfa inbakicept-pmln induction via intravesical instillation with BCG followed by maintenance therapy for up to 37 months.
Researchers assessed patients’ tumor status with cystoscopy and urine cytology every three months for up to two years, and a biopsy (random or cystoscopy-directed) was required within the first six months after treatment initiation. They followed local community standards for subsequent assessments.
The major efficacy outcome measures were complete response (CR) at any time and duration of response (DOR). CR was defined by a negative cystoscopy (with TURBT/biopsies as applicable) and urine cytology.
The CR rate was 62% (95% CI = 51, 73). Fifty-eight percent of patients with CR had a DOR of at least 12 months, and 40% had a DOR of at least 24 months.
The most common adverse reactions reported in at least 15% of patients in the clinical trial, including laboratory test abnormalities, were increased creatinine, dysuria, hematuria, increased urinary frequency, micturition urgency, urinary tract infection, increased potassium, musculoskeletal pain, chills, and pyrexia.
The recommended nogapendekin alfa inbakicept-pmln dose is 400 mcg administered intravesically with BCG once a week for six weeks as induction therapy. Patients may receive a second induction course if they do not achieve CR at month 3. For maintenance after induction therapy, the recommended dose is 400 mcg administered intravesically with BCG once a week for three weeks at months 4, 7, 10, 13, and 19 (for a total of 15 doses). Patients with an ongoing CR at month 25 or later may receive maintenance instillations with BCG once a week for three weeks at months 25, 31, and 37 for a maximum of 9 additional instillations. Discontinue treatment for disease persistence after second induction, disease recurrence or progression, or unacceptable toxicity. The maximum treatment duration is 37 months.
View the full prescribing information for nogapendekin alfa inbakicept-pmln.
For its review, FDA used the Assessment Aid, a voluntary submission from the applicant to facilitate the assessment. FDA granted the application breakthrough designation. FDA-expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions—Drugs and Biologics.
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