Detalimogene voraplasmid

Table of contents

• What is detalimogene voraplasmid?
• How does detalimogene voraplasmid work?
• Which conditions is detalimogene voraplasmid studied for?
• How is detalimogene voraplasmid given?
• What do the clinical trials show?
• What side effects are known?

What is detalimogene voraplasmid?

Detalimogene voraplasmid is an investigational gene therapy. It has not been granted a marketing authorisation and is therefore not available as a prescribed medicine. The substance is being developed for use in oncology, specifically for bladder cancer. In the trial registry, it is listed under the drug name DETALIMOGENE VORAPLASMID for intravesical use, meaning it is instilled directly into the bladder.^[1]

How does detalimogene voraplasmid work?

The registry data does not describe the molecular mechanism of action for detalimogene voraplasmid. It is referred to as an "immunoablative" treatment in the trial's brief summary, which suggests it is intended to act on the immune system within the bladder, but the specific target or pathway is not stated in the provided sources.^[1]

Which conditions is detalimogene voraplasmid studied for?

• Non-muscle invasive bladder cancer (NMIBC) — a type of bladder cancer that has not grown into the muscle layer of the bladder wall. The trial includes patients with carcinoma in situ (CIS) and recurrent low-grade Ta tumours.

How is detalimogene voraplasmid given?

Detalimogene voraplasmid is given by intravesical administration, which means it is instilled directly into the bladder through a catheter. In the trial, it is used as a solution for intravesical use. One substudy also involves a bladder rinse with a surfactant before the detalimogene instillation.^[1]

What do the clinical trials show?

There is one registered clinical trial for detalimogene voraplasmid in the EU Clinical Trials Information System. The trial is a Phase 1/2 master protocol with a planned enrolment of 474 participants. It is currently authorised, meaning it has received regulatory approval to proceed.^[1]

What are the trials measuring?

The primary outcome measures include the percentage of patients with a complete response (CR) on cystoscopic examination, urine cytology, and biopsies. The bladder rinse substudy measures the nature, incidence, and severity of adverse events, as well as dose-limiting toxicities. The IR-NMIBC substudy measures the complete response rate at 3 or 6 months, defined as the absence of visible tumour on cystoscopy and negative or atypical urine cytology.^[1]

Who can take part in these trials?

The trial enrols patients with BCG unresponsive non-muscle invasive bladder cancer with CIS, as well as high-risk NMIBC patients who are BCG naïve or received incomplete BCG treatment. One substudy focuses on patients with recurrent low-grade Ta IR-NMIBC.^[1]

What side effects are known?

The provided sources do not contain information on side effects of detalimogene voraplasmid. The trial's primary outcomes include the collection of adverse events and serious adverse events, but no results are yet reported.^[1]