The Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) to silmitasertib for the treatment of patients with medulloblastoma.
Medulloblastoma is a rare, severe brain tumor of the cerebellum caused by gene mutations that typically spreads through the cerebrospinal fluid. The signs and symptoms associated with medulloblastoma include headaches, nausea, vomiting, and difficulty with muscle coordination, balance, and fine motor skills.
Silmitasertib is a first-in-class potent and highly selective inhibitor of casein kinase 2 (CK2). In clinical studies, silmitasertib was shown to hit the CK2 target and modulate the expected pathways without displaying toxicity. The investigational treatment was also shown to act synergistically with DNA damaging agents (e.g., gemcitabine plus cisplatin) to improve efficacy.
The Company is currently evaluating silmitasertib in patients with recurrent sonic hedgehog driven medulloblastoma in a phase 1/2 and surgical study (ClinicalTrials.gov Identifier: NCT03904862).
“We are pleased to receive ODD for Silmitasertib for Medulloblastoma, a rare, severe pediatric disease for which there are no approved targeted therapies. ODD represents an important regulatory milestone that has the potential to expedite the clinical development of Silmitasertib, a potent and selective CK2 inhibitor,” said Dr. John Soong, Chief Medical Officer of Senhwa Biosciences.
Additionally, silmitasertib is being evaluated in two phase 2 studies for the treatment of patients with moderate COVID-19 (ClinicalTrials.gov Identifier: NCT04663737) and severe COVID-19 (ClinicalTrials.gov Identifier: NCT04668209). Silmitasertib is believed to block SARS-CoV-2-induced CK2 overactivation and reduce cytokine storm severity.
- Senhwa’s silmitasertib receives US FDA Orphan Drug designation for the treatment of medulloblastoma. News release. Senhwa Biosciences, Inc. Accessed December 17, 2021. https://www.prnewswire.com/news-releases/senhwas-silmitasertib-receives-us-fda-orphan-drug-designation-for-the-treatment-of-medulloblastoma-301447119.html.
- Medulloblastoma. National Organization for Rare Disorders. Accessed December 17, 2021. https://rarediseases.org/rare-diseases/medulloblastoma/.