Pediatric Brain Tumor Companies

Novartis Received FDA approval for KISQALI (tafmidis meglumine) for the treatment of Desmoid Tumor, a rare cancer affecting both adults and children. Ongoing clinical trials of CAR-T cell therapy for medulloblastoma, a high-grade pediatric brain tumor.

AbbVie Launched their Venclexta (venetoclax) in combination with Rituximab for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia (ALL), which can affect the brain in some cases Investing in research on targeted therapies for diffuse intrinsic pontine gliomas (DIPG), an aggressive pediatric brain tumor with limited treatment options.

Bluebird bio Received FDA approval for their gene therapy, Skysona (betibeglogene autotemcel), for the treatment of cerebral adrenoleukodystrophy (CALD), a rare pediatric brain disorder. Developing other gene therapy candidates for different types of pediatric brain tumors.

Orphazyme A/S Received FDA approval for arimoclomol for the treatment of Niemann-Pick disease type C, a rare neurodegenerative disorder that affects children and adults. Partnering with research institutions to explore its potential use in other pediatric brain disorders.

BeiGene, Received EMA approval for their Bruton's tyrosine kinase (BTK) inhibitor, Brukinsa (zanubrutinib), for the treatment of mantle cell lymphoma in pediatric patients.Ongoing clinical trials of Brukinsa for other types of pediatric B-cell malignancies, including some types of brain tumors.

• (US)


• AbbVie Inc. (US)


• Akorn Inc. (US)


• Agios Inc. (US)


• Baxter (US)


• Bayer AG (US)


• Epizyme Inc. (US)


• Novartis AG (Switzerland)


• Mylan N.V. (US)