RE:2 weeks: It’s all it took for Merck to get AA This very complete paper of March 2021 clearly shows that the only 2 FDA approved BCG-Unresponsive drugs are not convincing. This opens up the road for Accelerated Approval, no doubt.
Note also that Keytruda has never received EU approval.
BCG-unresponsive high-grade non-muscle invasive bladder cancer: what does the practicing urologist need to know? -
Until the end of 2020, the only FDA-approved treatments for BCG-unresponsive high-risk NMIBC were intravesical valrubicin (VALSTAR®) and pembrolizumab (KEYTRUDA®). However, this has brought little change in everyday practice as valrubicin efficacy has been questioned by many experts as the registration trial was not convincing and Pembrolizumab (Merck's Keytruda), although approved in 2020 by the FDA, remains a costly, difficult to access therapy with a role yet to be determined in Europe.
Conclusion
Until January 2021, the only FDA-approved treatments for BCG-unresponsive high-risk NMIBC were intravesical thiotepa, which is not used anymore due to its lack of efficacy, intravesical valrubicin, whose efficacy is questionable, and pembrolizumab not yet approved by the EMEA. Thus, in routine practice little has changed since the collaborative review published in European Urology in 2012 [10]. However, the definition of BCG-unresponsive disease, that is critical in managing patients, is better defined and has gained a near consensus. An effective alternative to radical cystectomy, that remains the optimal treatment option in this setting, remains an important unmet clinical need. Yet, considering the options that are currently under scrutiny, some of them having already completed phase III trials, one can reasonably expect that clinicians will have at their disposal new agents and treatment options in the next 2 years. As for today, clinical trials represent one of the most valid options if the patient is unwilling or unable to undergo a radical cystectomy.