The U.S. Food and Drug Administration has officially approved a new brain tumour treatment developed by Jazz Pharmaceuticals. The medication, called Modeyso, has been cleared for use in adults and children aged one year and older who have been diagnosed with diffuse midline glioma—a rare and fast-progressing form of brain tumour. This approval marks an important advancement for Jazz Pharmaceuticals, expanding its focus in the oncology field beyond its existing work in blood and lung cancer treatments.
Modeyso is the first FDA-approved brain tumour treatment developed to precisely target diffuse midline glioma in patients with a confirmed genetic mutation. It is intended for individuals whose illness has progressed even after receiving prior lines of therapy. The drug, formulated as a once-weekly oral capsule, will soon become available in the U.S.
Diffuse midline glioma (DMG) is a rare and aggressive cancer that primarily affects children and young adults. It develops in key midline areas of the spinal cord and brain, including the brainstem, thalamus, and spinal cord. Data from the National Institutes of Health (NIH) indicates that roughly 3,940 people across the United States are currently living with this tumour.
The FDA’s decision to grant approval was based on evidence collected from five clinical trials involving a total of 50 patients. Across the clinical trials, about 22% of cases showed a measurable decrease in tumour size. Among those who responded, the therapeutic benefit lasted for a median duration of slightly more than 10 months.
“We think it fits very well in terms of addressing a very high unmet need,” said Rob Iannone, Chief Medical Officer at Jazz Pharmaceuticals, ahead of the decision.
Jazz gained ownership of Modeyso in March following its $935 million acquisition of Chimerix. The company has indicated it will work closely with healthcare providers and advocates to help ensure the new treatment becomes accessible without much delay.
