Santhera Receives Positive Opinion for Orphan Drug Designation in the EU for POL6014 in Cystic Fibrosis

Santhera Pharmaceuticals (SIX: SANN) announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has issued a positive opinion on orphan drug designation for POL6014 in the treatment of cystic fibrosis (CF), a rare pulmonary disease affecting around 35,000 people in the European Union.  

"Obtaining orphan drug designation for POL6014 in the European Union is an important regulatory milestone for our development program with POL6014," stated Kristina Sjöblom Nygren, MD, Chief Medical Officer and Head of Development of Santhera. "With this positive opinion the COMP acknowledges the needs of patients with cystic fibrosis, and for a novel treatment approach with POL6014 to support these patients."

POL6014, an innovative, potent and selective inhibitor of human neutrophil elastase (hNE) licensed from Polyphor, is entering Phase I/II clinical development for the treatment of CF.

The positive opinion from the EMA's COMP will be sent to the European Commission (EC), which is expected to grant the orphan drug designation within 30 days. The orphan drug designation will provide Santhera with regulatory and financial incentives to develop POL6014 in the treatment of CF.

About POL6014
POL6014 is a highly potent and selective inhibitor of human neutrophil elastase (hNE) and was shown to reach high concentrations in the lung when administered by inhalation via an optimized eFlow® nebulizer (PARI Pharma GmbH). A first-in-man Phase I study in healthy volunteers and a Single Ascending Dose (SAD) safety and tolerability Phase I study in CF patients have successfully been completed. The drug candidate was well tolerated and showed evidence of strong elastase inhibition as previously demonstrated in animal models. In addition, POL6014 may show therapeutic benefit for a range of neutrophilic pulmonary diseases with high medical need such as non-cystic fibrosis bronchiectasis (NCFB), alpha-1 antitrypsin deficiency (AATD) or primary ciliary dyskinesia (PCD). POL6014 has EU orphan drug designation for the treatment of AATD and PCD.