The European Commission (EC) has conditionally approved MSD’s Welireg, an oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, as a single agent for the treatment of adults with von Hippel-Lindau (VHL) disease and advanced clear cell renal cell carcinoma (RCC).
Welireg is intended for adults with VHL disease who need therapy for associated renal cell carcinoma (RCC), central nervous system hemangioblastomas or pancreatic neuroendocrine tumours, and for those individuals for whom localised procedures are not suitable.
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The second is for adults with advanced clear cell RCC that has advanced after two or more lines of therapy, including a programmed death-ligand 1 inhibitor or a programmed death receptor-1 and a minimum of two vascular endothelial growth factor targeted therapies.
The conditional approval is based on the outcomes from the open-label trials LITESPARK-004 and LITESPARK-005.
It is valid for one year and is subject to annual renewal based on Phase II LITESPARK-004 trial’s additional clinical data from ongoing trials in subjects with certain VHL disease-related tumours.
Welireg is now sanctioned in more than 30 nations for certain adult people with previously treated advanced RCC and in more than 40 countries for those with specific VHL disease-associated tumours.
With this approval, the therapy can be marketed in all 27 EU member states, including Norway, Iceland and Liechtenstein.
The timing for the therapy’s commercial availability in individual EU nations will be contingent on national reimbursement procedures and other factors.
In the European Union (EU), Welireg is the first systemic therapy for treating VHL disease-associated tumours. The approval for this indication rests on the duration of response and objective response rate outcomes from the LITESPARK-004 trial.
MSD recently announced that the US Food and Drug Administration has accepted for priority review a supplemental new drug application for Welireg for the treatment of adult and paediatric patients with advanced pheochromocytoma and paraganglioma.
