The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for trofolastat (RoTecPSMA), marking a significant advancement in prostate cancer diagnostics. This novel agent is the first prostate-specific membrane antigen (PSMA)-targeting product authorized for use with technetium-99m to detect cancerous lesions in men with prostate cancer.
Prostate cancer affects approximately 1 in 8 men in the UK during their lifetime, with about 55,100 new cases diagnosed annually. Accurate diagnostic imaging is crucial for effective disease management and treatment planning.
How Trofolastat Works
Trofolastat is administered as a single injection after being combined with the radioactive tracer technetium-99m to form Technetium (99mTc) trofolastat. The compound specifically binds to PSMA, a protein highly expressed on prostate cancer cells. This binding allows physicians to visualize cancerous areas during single photon emission computed tomography (SPECT) imaging procedures.
Julian Beach, MHRA Interim Executive Director of Healthcare Quality and Access, emphasized the significance of this approval: "As the first PSMA-targeting diagnostic product approved with Technetium-99m, which is widely available in UK nuclear medicine facilities, this approval has the potential to expand access to prostate cancer imaging and support diagnostic pathways within the NHS."
Beach added, "The approval of this diagnostic product follows a rigorous assessment to ensure that it meets the required regulatory standards. As with all products, we will continue to monitor its safety and effectiveness."
Clinical Applications and Evidence
The MHRA has approved trofolastat for three specific clinical scenarios:
- Identifying the extent of high-risk prostate cancer spread before initial treatment
- Detecting disease recurrence in patients with rising prostate-specific antigen (PSA) levels
- Determining whether targeted therapies might benefit patients with metastatic prostate cancer
The regulatory decision was supported by compelling evidence from a multi-center, prospective study involving 105 prostate cancer patients. In this clinical investigation, Technetium (99mTc) trofolastat demonstrated remarkable efficacy with 94.2% sensitivity in identifying prostate cancer lesions and 83.3% specificity in confirming cancer-free areas.
Safety Profile and Monitoring
The most commonly reported side effect associated with Technetium (99mTc) trofolastat was headache. As with all approved medications, the MHRA will maintain close surveillance of the agent's safety and effectiveness.
Patients experiencing potential side effects are encouraged to discuss them with healthcare providers and report them directly to the Yellow Card scheme through the MHRA website or mobile application.
Implications for UK Healthcare
The approval of trofolastat represents a meaningful advancement in prostate cancer management within the UK healthcare system. By utilizing technetium-99m, which is already widely available in nuclear medicine departments throughout the country, this new diagnostic tool has the potential to significantly expand access to specialized prostate cancer imaging.
Improved imaging capabilities may lead to more precise staging of newly diagnosed prostate cancer, earlier detection of recurrent disease, and better selection of patients for targeted therapies—ultimately contributing to improved outcomes for men with prostate cancer.
The marketing authorization for trofolastat was officially granted on March 27, 2025, following the MHRA's comprehensive evaluation of its safety, efficacy, and quality profiles.
