Santhera says its experimental cystic fibrosis drug POL6014 is likely to be designated an orphan drug in the next 30 days, putting the firm in line to receive regulatory and financial incentives for its further development.
The Committee for Orphan Medicinal Products of the European Medicines Agency has now issued a positive opinion on orphan drug designation for POL6014, acknowledging “the needs of patients with cystic fibrosis” as well as the need for “a novel treatment approach with POL6014 to support these patients,” according to Kristina Sjöblom Nygren, chief medical officer and head of development at Santhera.
POL6014 is an innovative, potent and selective inhibitor of human neutrophil elastase (hNE) licensed from Polyphor, which is now entering Phase I/II clinical development for the treatment of cystic fibrosis.
A first-in-man Phase I study in healthy volunteers and a safety and tolerability Phase I study in cystic fibrosis patients have already successfully been completed, the firm said, noting that the drug was “well tolerated and showed evidence of strong elastase inhibition as previously demonstrated in animal models.”
High levels of elastase causes damage to structural, cellular and soluble components of the pulmonary microenvironment, and so correlates with disease severity, as measured by functional lung parameters.
Cystic fibrosis is a rare pulmonary disease affecting around 35,000 people in the EU.