The Food and Drug Administration (FDA) has granted Orphan Drug designation to tasquinimod for the treatment of myelofibrosis.
Tasquinimod is an oral small molecule immune modulator with anti-angiogenic properties. The designation was based on preclinical data that showed tasquinimod reduced fibrosis in the bone marrow and normalized spleen size in a myelofibrosis mouse model. A clinical study involving patients with myelofibrosis is expected to begin in early 2023.
“The Orphan Drug designation awarded by the FDA for tasquinimod in myelofibrosis represents an important step forward for Active Biotech”, said Helén Tuvesson, CEO Active Biotech. “It opens an important regulatory pathway and provides us with the potential to rapidly advance the development of tasquinimod in this patient population.”
Tasquinimod is also being evaluated in patients with relapsed or refractory multiple myeloma in a phase 1b/2a study (ClinicalTrials.gov Identifier: NCT04405167).
FDA grants Orphan Drug designation for tasquinimod in myelofibrosis. News release. Active Biotech AB. Accessed May 18, 2022. https://www.globenewswire.com/news-release/2022/05/18/2446074/0/en/FDA-grants-Orphan-Drug-Designation-for-tasquinimod-in-myelofibrosis.html