On 27th April 2017, the European Medicines Agency (EMA) granted Orphan Drug Designation status to PQR309 for the treatment of patients with DLBCL.
PIQUR Therapeutics AG’s PQR309 is an oral, brain-penetrant, dual inhibitor of the PI3K/mTOR pathway. It can cross the blood-brain barrier, making it an attractive choice in treating malignant diseases involving the brain.
PQR309 has shown pre-clinical activity in various tumor models, as well as clinical activity in phase I and II studies. The dual PI3K/mTOR pathway inhibitor is currently being investigated in a phase I/II trial (NCT02249429) and a phase II trial (NCT03127020) in R/R lymphoma, as well as two phase II trials in R/R PCNSL (NCT02669511; NCT03120000).
Currently, PQR309 already holds an Orphan Drug Designation for patients with Primary Central Nervous System Lymphoma (PCNSL), granted by the U.S. Food and Drug Administration (FDA) in December 2016.
- EMA grants orphan drug designation to PIQUR’s lead compound PQR309 to treat patients with DLBCL. 2017 May 02. http://www.pharmabiz.com/NewsDetails.aspx?aid=101700&sid=2. [Accessed 2017 May 02].