Back to top

Image: Shutterstock

MRK's sNDA for Welireg in Rare Tumors Gets FDA's Priority Tag

Read MoreHide Full Article

Merck (MRK - Free Report) announced that the FDA has accepted its supplemental new drug application (sNDA) seeking approval of its oral HIF-2α inhibitor, Welireg (belzutifan), for a new indication.

The sNDA is seeking approval of Welireg for treating advanced, unresectable, or metastatic pheochromocytoma and paraganglioma (PPGL) in adult and pediatric patients aged 12 years and above. Currently, there are no approved therapies for treating these rare adrenal tumors.

With the FDA granting a priority review to the sNDA, a decision from the regulatory body is expected on May 26, 2025.

The latest sNDA was based on objective response rate (ORR) and duration of response (DOR) data from the phase II LITESPARK-015 study, which evaluated Welireg for the given indication.

The primary endpoint of the LITESPARK-015 study was ORR per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) as assessed by a blinded independent central review.

Time to response, DOR, disease control, progression-free survival, overall survival and safety were the secondary endpoints of the above study.

MRK's Stock Price Performance

In the past year, shares of Merck have plunged 19.2% compared with the industry’s decrease of 2.2%.

Zacks Investment Research
Image Source: Zacks Investment Research

Welireg is currently approved in the United States for patients with advanced renal cell carcinoma (“RCC”) and some von Hippel-Lindau (“VHL”) disease-associated tumors.

A potential FDA nod for advanced PPGL will make it the third approved indication for Welireg in the United States.

Also, if approved, Welireg would become the only available treatment for eligible patients with advanced PPGL in the United States.

Recent Updates on MRK's Welireg

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) rendered a positive opinion recommending conditional approval to Welireg for two indications in December 2024.

The CHMP recommended approving Welireg for certain previously treated patients with advanced RCC and certain patients with VHL disease-associated tumors.

A final decision from the European Commission for both indications is expected later in the first quarter of 2025. Welireg is not currently approved in Europe for any indication.

Merck is currently evaluating Welireg as monotherapy and in combination with other therapies for treating rare cancer indications, including RCC and other tumor types.

In the first nine months of 2024, Welireg generated sales worth $349 million, driven by increased uptake for the additional indication of previously treated advanced RCC in the United States.

With concerns over its blockbuster anti-PD-1 therapy Keytruda’s potential loss of exclusivity post-2028, the approval of new drugs like Welireg for additional indications should help Merck lower its dependence on Keytruda in future quarters.

MRK's Zacks Rank & Stocks to Consider

Merck currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Harmony Biosciences Holdings, Inc. (HRMY - Free Report) , BioMarin Pharmaceutical Inc. (BMRN - Free Report) and Castle Biosciences, Inc. (CSTL - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Harmony Biosciences’ earnings per share have increased from $2.64 to $3.22 for 2025. In the past year, shares of HRMY have rallied 18.5%.

HRMY’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 147.24%.

In the past 60 days, estimates for BioMarin’s earnings per share have moved up from $3.94 to $4.02 for 2025. In the past year, shares of BMRN have plunged 32.7%.

BMRN’s earnings beat estimates in each of the trailing four quarters, the average surprise being 28.70%.

In the past 60 days, estimates for Castle Biosciences’ loss per share have narrowed from $1.88 to $1.70 for 2025. In the past year, shares of CSTL have increased 7.8%.

CSTL’s earnings beat estimates in each of the trailing four quarters, the average surprise being 172.72%.

Published in