HQ Team
August 7, 2024: Servier Pharmaceuticals, the US arm of a French pharmaceutical company, got approval from the US drug regulator for a once-daily pill to treat specific forms of brain cancer.
The US Food and Drug Administration cleared vorasidenib, branded as Voranigo — an isocitrate dehydrogenase inhibitor (IDH) — for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma.
Isocitrate dehydrogenase inhibitors are a class of drugs designed to target and inhibit the activity of isocitrate dehydrogenase enzymes, which play a critical role in the tricarboxylic acid cycle.
These enzymes catalyze the conversion of isocitrate to alpha-ketoglutarate, a key step in cellular metabolism.
In healthy human cells, a family of isocitrate dehydrogenases helps break down nutrients and generate energy for cells.
Grow out of control
Mutations in IDH1 and IDH2 are associated with a variety of cancers, where they prevent cells from differentiating or specializing, into the kind of cells they are ultimately supposed to become.
When cells cannot differentiate properly, they may begin to grow out of control. In IDH-mutant gliomas, Voranigo works by reducing the activity of the mutant IDH1 and IDH2 enzymes, to help control the disease.
The patient population also includes those with a susceptible IDH1 or IDH2 mutation following surgery including biopsy, sub-total resection, or gross total resection.
The FDA nod was based on efficacy outcomes during the end-stage trial.
A blinded independent review committee found that progression-free survival was “statistically significant and clinically meaningful in favour of the vorasidenib arm” compared to the dummy group.
27.7 months
The median progression-free survival was 27.7 months in the vorasidenib group, compared with 11.1 months in the placebo group, according to the company statement.
“Voranigo is available and offers glioma patients the ability to actively manage their disease with the convenience of a once-daily pill,” according to a company statement.
Gliomas are types of brain cancer that can hinder normal brain function and cause a variety of symptoms.
Diffuse gliomas with IDH mutations represent the most common malignant primary brain tumours diagnosed in adults younger than 50 years of age.
The diseases are not curable with current therapies, and without treatment, they continue to grow and infiltrate normal brain tissue.
First breakthrough in 25 years
“Today’s approval of Voranigo is an enormous leap forward in cancer care and a defining moment for people living with Grade 2 IDH-mutant glioma,” said Arjun H. Prasad, Chief Commercial Officer, Servier Pharmaceuticals.
“Voranigo, which is the first breakthrough in this specific disease area in nearly 25 years, offers patients unprecedented improvement in progression-free survival.”
Patients living with Grade 2 IDH-mutant gliomas have long faced the harsh reality of an incurable disease with very limited post-surgery treatment options, said Ralph DeVitto, President & CEO, of the American Brain Tumor Association.
The results of the trial were published in The New England Journal of Medicine.
Agios to get $1.1 billion
“Glioma is a unique cancer. Many of the patients I’ve met are in their 30s and 40s and in the prime of their lives,” said David K. Lee, CEO, of Servier Pharmaceuticals.
“As we advance more targeted therapies, identifying mutations and understanding how these mutations impact cancer and its progression are key to helping the right patients find the right treatment, at the right time.”
Drugmaker Servier gained the oral dual inhibitor of mutant IDH1 and IDH2 when it acquired Agios Pharmaceuticals’ oncology business for $1.8 billion in 2021.
Agios Pharmaceuticals, Inc. will receive a total of $1.1 billion in milestone payments following the FDA approval of vorasidenib.
These payments include a $905 million payment from Royalty Pharma in connection with the vorasidenib royalty purchase agreement Agios announced in May 2024 and a $200 million payment from Servier in connection with Agios’ divestiture of its oncology business in 2021.
