Curatis on Track: Submits Orphan Drug Application for Key Product Candidate in the US

Curatis Holding AG (SIX:CURN) has submitted an application to the FDA for Orphan Drug Designation for its lead product candidate, C-PTBE-01. This product aims to alleviate the severe side effects experienced by children with a specific type of brain tumor.

Curatis focuses its development efforts on C-PTBE-01 for a very rare and aggressive brain tumor known as Diffuse Midline Glioma (DMG). DMG primarily affects children, with most cases diagnosed between ages 5 and 9. Approximately 800 new cases are diagnosed annually in the US, similarly to Europe.

DMG often causes indirect brain damage due to the accumulation of extracellular fluid around the tumor. These peritumoral brain edemas can lead to severe symptoms, including headaches, vomiting, and neurological impairments.

Current standard treatments for PTBE include the use of corticosteroids, which have significant side effects such as severe myopathies and osteoporosis. C-PTBE-01 has shown a strong steroid-sparing effect in two clinical studies, potentially reducing or even replacing the need for steroids.