Health Care Professionals


Santhera is passionate about developing new treatments for patients with rare diseases. Our focus is on the development of treatments for neuromuscular and pulmonary diseases that currently lack treatment options.

Our pipeline: lonodelestat

Lonodelestat (POL6014), a clinical stage selective inhibitor of human neutrophil elastase, has the potential to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases such as non-cystic fibrosis bronchiectasis (NCFB), alpha-1 antitrypsin deficiency (AATD) and primary ciliary dyskinesia (PCD).

Lonodelestat 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 1 study in healthy volunteers and a Single Ascending Dose (SAD) safety and tolerability Phase 1 study in CF patients have successfully been completed with lonodelestat. The drug candidate was well tolerated and safe with a favorable pharmacokinetic profile and strong elastase inhibition as previously shown in animal models.

Santhera has in-licensed the compound from Polyphor Ltd. and will assume the global development, regulatory filings and commercialization of lonodelestat.

For more information please see press release of February 15, 2018.