The EMA grants PQR309 Orphan Drug Designation for Diffuse Large B-Cell Lymphoma

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.

  1. 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].