Zynyz receives orphan drug designation in Korea

The immuno-oncology drug will be introduced to Korea by Handok

First FDA-approved treatment option for anal cancer

Demonstrated improved survival in the POD1UM-303 study

The immuno-oncology drug Zynyz that Handok has decided to market in Korea has been designated as an orphan drug in Korea.

 

The Ministry of Food and Drug Safety (MFDS) recently announced the designation through a public notice.

 

Specifically, the drug is indicated for ▲Merkel cell carcinoma and ▲ squamous cell carcinoma of the anal canal (SCAC).

 

Among these, the Merkel cell carcinoma indication had already been granted orphan drug status earlier in June.

 

Zynyz (retifanlimab), which was developed by the US company Incyte, was also designated in July as a subject for the “Global Innovative Products on Fast Track (GIFT)” program.

 

Since receiving approval from the U.S.

 

FDA in May, the PD-1 inhibitor Zynyz has been gaining attention as the first first-line treatment for adult patients with unresectable locally recurrent or metastatic anal cancer, in combination with platinum-based chemotherapy (carboplatin, paclitaxel).

 

The efficacy of Zynyz in anal cancer was demonstrated in the Phase III POD1UM-303 study.

 

This study evaluated 308 patients with unresectable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC) by comparing outcomes between the Zynyz + carboplatin + paclitaxel combination arm and the standard-of-care arm.

 

Results showed that the median progression-free survival (PFS) was 9.3 months in the Zynyz arm, which is a 37% reduction in the risk of disease progression or death compared with the 7.4 months in the standard-of-care arm.

 

In the interim analysis, the median overall survival (OS) was 29.2 months in the Zynyz arm and 23 months in the standard-of-care arm.

 

The objective response rate (ORR) for the Zynyz arm was 56%, with 22% achieving complete response and 33% partial response—higher than the 44% observed in the standard-of-care arm.

 

The median duration of response (DoR) was 14 months in the Zynyz arm, compared with 7.2 months in the standard-of-care arm.

 

SCAC accounts for about 85% of all anal cancers and is classified as a rare cancer.

 

The majority of cases are caused by human papillomavirus (HPV) infection, and HIV-positive individuals face a 25–35 times higher risk.

 

Early symptoms often resemble hemorrhoids, leading to delayed diagnosis, with many patients visiting hospitals at advanced stages.