Servier Pharmaceuticals announced that its brain tumor treatment, branded as Voranigo, has been approved by the U.S. Food and Drug Administration (FDA). This marks a significant milestone as Voranigo becomes the first drug to receive FDA approval for treating Grade 2 IDH-mutant glioma, a specific type of brain cancer that has traditionally only been managed through surgical removal of the tumor.
Grade 2 IDH-mutant gliomas are caused by mutations in the isocitrate dehydrogenase (IDH) gene family, which can disrupt normal brain function. Until now, there have been no approved drug treatments for this condition, making Voranigo a groundbreaking development in the field of oncology.
The FDA’s approval of Voranigo was based on promising results from a late-stage clinical trial, where patients treated with the drug experienced a progression-free survival period of 27.7 months, significantly longer than the 11.1 months observed in the placebo group. This extended survival period highlights Voranigo’s potential to offer new hope to patients battling this challenging form of brain cancer.
The incidence of IDH-mutant glioma in the U.S. is relatively rare, affecting approximately 0.7 out of every 100,000 people. However, the impact of this FDA approval is profound, as it provides a new treatment option for those affected by this aggressive cancer.
The approval also has significant financial implications. Agios Pharmaceuticals, which initially developed the oncology treatment before selling its business to Servier in 2021 for $1.8 billion in upfront cash, stands to receive up to $1.1 billion in milestone payments from Servier and Royalty Pharma as part of the deal. Specifically, Agios is set to receive a $200 million milestone payment from Servier following the FDA approval and 15% royalties on the potential U.S. net sales of Voranigo.
Earlier this year, Agios sold a portion of its royalty rights to Royalty Pharma. Under this agreement, the FDA approval triggered a payment of $905 million to Agios, further highlighting the significant economic stakes tied to this groundbreaking treatment.