PPGL is a group of rare neuroendocrine tumours originating from adrenal gland tissue or paraganglia. Credit: Witthaya Prasongsin via Getty Images.

The US Food and Drug Administration (FDA) will soon decide on whether to expand the label of MSD’s Welireg (belzutifan) to include its use for the treatment of the rare cancer pheochromocytoma and paraganglioma (PPGL). 

The regulatory agency will review MSD’s new drug application (NDA) for Welireg under a Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of 26 May 2025. The new label is intended to cover adult and paediatric patients aged 12 years and older with advanced, unresectable, or metastatic PPGL. 

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MSD’s submission is based on efficacy and safety data from the Phase II LITESPARK-015 trial (NCT04924075), which evaluated Welireg in patients with advanced PPGL. The study measured the objective response rate (ORR) and duration of response (DOR) as primary endpoints and included other secondary endpoints such as disease control, progression-free survival (PFS), and safety. Results from the trial are expected to be presented at an upcoming medical meeting.  

MSD acquired Welireg when it bought Peloton Therapeutics for an upfront payment of $1.05bn in 2019. In August 2021, the drug was first approved by the FDA for the treatment of a rare disorder called von Hippel-Lindau (VHL) disease. Welireg is also approved for treating some patients with advanced renal cell carcinoma (RCC). 

PPGL is a group of rare neuroendocrine tumours originating from adrenal gland tissue or paraganglia. These tumours are often challenging to treat in advanced stages, and current therapeutic options are limited.  

A gap in the PPGL treatment market was left following the withdrawal of Lantheus’s Azedra (iobenguane I-131), a radiopharmaceutical therapy approved by the FDA for treating unresectable, locally advanced, or metastatic PPGL requiring systemic anticancer therapy in 2018. Azedra was the first FDA-approved therapy specifically for this rare indication. However, the drug was voluntarily withdrawn from the market due to manufacturing challenges that affected its commercial availability in 2023.  

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Welireg inhibits hypoxia-inducible factor-2 alpha (HIF-2α), thereby targeting pathways involved in tumour growth and survival under low-oxygen conditions. Welireg generated $218m in global sales in 2023, as per MSD’s financials, with expectations to reach up to $1.5bn in annual sales in 2030, according to GlobalData’s Pharma Intelligence Center. 

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Welireg comes with a boxed warning about its potential to cause embryo-foetal harm if used during pregnancy. It can also render hormonal contraceptives ineffective and cause severe anaemia and severe hypoxia.