FDA Grants Orphan Drug Designation for AXL1717 for the Treatment of Glioma
Axelar AB, a pharmaceutical company focused on developing targeted oral anti-cancer therapies for indications where there are significant unmet medical needs, announced today that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation, on a first cycle review, for AXL1717 for the treatment of glioma.
“Being granted an orphan drug designation for AXL1717 for the treatment of glioma by FDA is an important regulatory milestone for Axelar and affirms our strategy of focusing specifically on developing oral therapies for patients with brain tumors and brain metastases,” said Ulrika Wennberg, Chief Executive Officer of Axelar.
A glioma is a type of tumor that starts in the glial cells of the brain or the spine. Gliomas comprise about 30 per cent of all brain tumors and central nervous system tumors, and 80 per cent of all malignant brain tumors. The prognosis for patients with high-grade gliomas is generally poor, and is especially so for older patients. Treatment for brain gliomas depends on the location, the cell type, and the grade of malignancy. Often, treatment is a combined approach, using surgery, radiation therapy, and chemotherapy.
About AXL1717 (picropodophyllin)
AXL1717 is an orally bioavailable small molecule with dual mechanisms of action, which has been shown to pass through the blood-brain barrier. AXL1717 targets the IGF-1R signaling pathway but does not bind the IGF-1R. AXL1717 has also been shown to yield cell cycle arrest in mitosis, due to indirect effects on microtubule dynamics, making it a unique anti-cancer drug.
About FDA Orphan Drug Designation Program
The FDA Orphan Drug Designation program provides orphan status to drugs intended to treat rare diseases or disorders that affect fewer than 200,000 people in the US. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain incentives, including federal grants, tax credits and a waiver of Prescription Drug User Fee Act filing fees.
About Axelar AB
Axelar is a Swedish drug development company developing targeted oral anti-cancer therapies for indications where there are significant unmet medical needs. The company was founded based on research performed at Karolinska Institutet, Stockholm and the discovery of a group of compounds that target the IGF-1 receptor signaling pathway without affecting signaling from the closely related insulin receptor.
For additional information about Axelar, please visit www.axelar.se.