Janssen Korea’s promising bladder cancer treatment, TAR-200, has faced an early hurdle in its path to the Korean market.

The Central Drug Review Committee (CDRC) under the Ministry of Food and Drug Safety (MFDS) could not reach a clear consensus on whether to grant the drug orphan drug designation -- raising concerns about delays in patient access to the therapy.

According to recently disclosed meeting minutes, the committee held a written deliberation from May 7 to 9 to review the feasibility of granting TAR-200 orphan drug designation while still in development.

However, opinions among committee members were divided.

Supporters argued that TAR-200 met the criteria, citing meaningful efficacy improvements over existing treatments. They pointed to early clinical results showing a complete response (CR) rate of 83.5 percent and a response duration of 13.9 months -- suggesting TAR-200 could serve as an alternative to cystectomy.

Skeptics countered that TAR-200 is “essentially a reformulation of an existing drug” and that it lacked sufficient scientific evidence to prove a “significant improvement in efficacy” -- a key requirement for orphan drug designation. Many urged caution, recommending the application be reconsidered after results from head-to-head clinical trials become available.

Janssen’s orphan drug strategy falters amid regulatory uncertainty

In Korea, orphan drug designation comes with significant regulatory incentives, including priority review, reduced documentation requirements, cost support, and favorable reimbursement status upon health insurance listing. Analysts note that such benefits are especially critical for rare diseases, where limited patient populations make drug development more difficult.

As such, the CDRC’s indecision could impact Janssen’s broader strategy. A delayed or denied orphan drug designation may slow TAR-200’s market entry and limit patient access in Korea.

TAR-200 is a drug delivery system placed directly into the bladder to release gemcitabine over time. It targets high-risk, non-muscle-invasive bladder cancer (HR-NMIBC) patients unresponsive to Bacillus Calmette-Guerin (BCG) therapy, and it is viewed as a novel approach to addressing the shortcomings of existing treatments.

At the recent American Urological Association (AUA 2025) meeting, Janssen presented data from the global phase 2b SunRISe-1 study (Cohort 4), showing disease-free survival (DFS) rates of 85.3 percent at six months and 81.1 percent at nine months. The bladder preservation rate was 94.2 percent -- promising results in a patient group at high risk of disease progression.

In addition, 95.6 percent of patients remained progression-free at nine months, with an overall survival rate of 98 percent -- reinforcing TAR-200’s potential as a viable alternative to cystectomy-centered treatment.

TAR-200 can be inserted without anesthesia during a brief outpatient procedure, offering a non-surgical option for elderly patients or those ineligible for surgery.

Patient tolerance and clinical comparisons remain key concerns

Still, some CDRC members flagged concerns about patient discomfort and adherence due to the prolonged insertion of the device. Clinical data showed that around 48.1 percent of patients skipped an insertion, and 28.2 percent had the device removed early.

Moreover, with no direct comparisons to BCG combinations or other immunotherapy agents, reviewers said more data -- including head-to-head trials -- are needed to confirm TAR-200’s efficacy and safety.

TAR-200 was granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) in December 2023, and Janssen completed a New Drug Application under the FDA’s Real-Time Oncology Review (RTOR) program in January 2025. This puts the drug on track for U.S. approval soon.

In Korea, however, even orphan drug status remains undecided. “Depending on how the MFDS interprets the committee’s recommendation, Janssen’s domestic launch strategy could be significantly affected,” said an industry source.

Another source said orphan drug designation is not just a matter of regulatory benefit but is directly tied to treatment access -- urging the MFDS to show flexibility in its policies to minimize delays compared to global markets.