On 22 April 2024, the US Food and Drug Administration (FDA) approved nogapendekin alfa inbakicept-pmln (Anktiva, Altor BioScience, LLC) with Bacillus Calmette-Gu\u00e9rin (BCG) for adult patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours.<\/p>\n
Efficacy was evaluated in QUILT-3.032 (NCT0302285), a single-arm, multicentre study 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.<\/p>\n
Tumour status was assessed with cystoscopy and urine cytology every 3 months for up to 2 years and biopsy (random or cystoscopy-directed) was required within the first 6 months after treatment initiation. Subsequent assessment was performed per local community standards. <\/p>\n
The major efficacy outcome measures were complete response (CR) at any time and duration of complete response (DoR). CR was defined by a negative cystoscopy (with TURBT\/biopsies as applicable) and urine cytology. <\/p>\n
The CR rate was 62% (95% confidence interval 51, 73); 58% of patients with CR had a DoR \u2265 12 months and 40% had a DoR \u2265 24 months.<\/p>\n
The most common adverse reactions (\u226515%), including laboratory test abnormalities, were increased creatinine, dysuria, haematuria, urinary frequency, micturition urgency, urinary tract infection, increased potassium, musculoskeletal pain, chills, and pyrexia.<\/p>\n
The recommended nogapendekin alfa inbakicept-pmln dose is 400 mcg administered intravesically with BCG once a week for 6 weeks as induction therapy. A second induction course may be administered if CR is not achieved at month 3. For maintenance after induction therapy, the recommended dose is 400 mcg administered intravesically with BCG once a week for 3 weeks at months 4, 7, 10, 13, and 19 (for a total of 15 doses). For patients with an ongoing CR at month 25 or later, maintenance instillations with BCG may be administered once a week for 3 weeks at months 25, 31, and 37 for a maximum of 9 additional instillations. Treatment should be discontinued for disease persistence after second induction, disease recurrence or progression, or unacceptable toxicity. The maximum treatment duration is 37 months.<\/p>\n
This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA\u2019s assessment.<\/p>\n
This application was granted breakthrough designation.<\/p>\n
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA\u2019s MedWatch Reporting System.<\/p>\n
For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact FDA\u2019s Oncology Center of Excellence<\/a> Project Facilitate<\/a>.<\/p>\n