New York: The US Food and Drug Administration (FDA) has officially approved Modeyso, a drug developed by Jazz Pharmaceuticals, for the treatment of diffuse midline glioma (DMG), a rare and aggressive brain tumor, in adults and children aged one year and older.
The FDA announced the approval on August 7, marking a major advancement in cancer treatment as Modeyso becomes the first FDA-approved systemic therapy for DMG with a specific mutation that has progressed despite prior treatment.
This rare form of cancer primarily affects children and young adults, forming in midline structures of the brain and spinal cord, such as the brainstem, thalamus, and spinal cord. According to NIH data, around 3,940 people in the United States are currently living with this condition.
Modeyso is an oral capsule taken once weekly and is expected to become available in the US in the coming weeks.
The approval is based on data from 50 patients across five clinical trials, where approximately 22 percent of patients experienced tumor shrinkage, with benefits lasting a median of just over 10 months for those who responded.
Jazz Pharmaceuticals acquired the drug in March 2024 as part of its $935 million acquisition of Chimerix. The approval significantly broadens Jazz’s oncology portfolio, which had previously focused on treatments for certain blood and lung cancers.
Rob Iannone, Chief Medical Officer at Jazz Pharmaceuticals, said that the drug meets a critical unmet need and emphasized the company’s commitment to working with healthcare providers and advocacy groups to make the therapy accessible as quickly as possible.
Jazz’s approval of Modeyso is seen as a promising step for patients suffering from a devastating diagnosis that, until now, lacked effective systemic treatment options.
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