Tasquinimod Granted Orphan Drug Designation for Myelofibrosis

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to tasquinimod for the treatment of myelofibrosis (MF).

Tasquinimod is an oral immunomodulatory and antiangiogenic investigational treatment that affects the tumor’s ability to grow and metastasize. Tasquinimod has previously been studied as an anti-cancer agent in patients with solid cancers, including a phase III, randomized trial of patients with metastatic prostate cancer. The tolerability of tasquinimod is well-characterized based on these previous experiences.

Tasquinimod has demonstrated a clear therapeutic potential in preclinical models of multiple myeloma (MM) when used as a single agent and in combination with standard MM therapy. A clinical phase Ib/IIa study is ongoing with tasquinimod in relapsed or refractory MM.

“The Orphan Drug Designation awarded by the FDA for tasquinimod in MF represents an important step forward for Active Biotech,” said Helén Tuvesson, Chief Executive Officer of Active Biotech, the drug’s manufacturer. “It opens an important regulatory pathway and provides us with the potential to rapidly advance the development of tasquinimod in this patient population.”

Active Biotech and Erasmus University Medical Center in Rotterdam, Netherlands, have initiated a research collaboration with tasquinimod in MF that includes preclinical studies as well as a clinical proof of concept study in patients with MF. The clinical study will be financed by Oncode and is planned to start early 2023.

Source: Active Biotech Press Release, May 2022