A recommendation supporting the designation, which offers financial and regulatory incentives to develop treatments for rare diseases, was made by Committee for Orphan Medicinal Products (COMP), a branch of the European Medicines Agency (EMA).
The positive opinion is now before the European Commission, which normally accepts them. A decision is expected within 30 days.
“Obtaining orphan drug designation for POL6014 in the European Union is an important regulatory milestone for our development program with POL6014,” Kristina Sjöblom Nygren, chief medical officer and head of development for Santhera, said in a company press release.
POL6014 is a highly potent and selective inhibitor of human neutrophil elastase (hNE), an enzyme released by neutrophils and macrophages (a type of white blood cell) that works to kill bacteria during infections.
Studies have found that high hNE levels in CF patients correlate with higher disease severity, reflected in a poorer lung function.
PL6014 is designed to temporarily block hNE function, so as to halt or slow tissue damage and inflammation within the airways of CF patients, alleviating disease symptoms.
POL6014 was seen to reach high concentrations in the lung when inhaled using the eFlow nebulizer (marketed by PARI Pharma). In Phase 1 studies in healthy volunteers and CF patients, POL6014 was seen to be well-tolerated and inhibited hNE activity.
The company is also pursuing POL6014 to possibly treat other pulmonary conditions in which neutrophils play an important part and for which there is a high medical need; these include non-cystic fibrosis bronchiectasis (NCFB), alpha-1 antitrypsin deficiency, and primary ciliary dyskinesia.
POL6014 has been named an orphan drug in the European Union as a possible treatment for alpha-1 antitrypsin deficiency, and primary ciliary dyskinesia.
“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,” Nygren said.