Cystic fibrosis: Orphan drug designation to counter lung inflammation

The European Medicines Agency (EMA) has granted Orphan Drug Designation for cystic fibrosis to GY971, a new anti-inflammatory molecule developed by the Universities of Ferrara and Padua and the Azienda Ospedaliera Universitaria di Verona with funding from the Foundation for Cystic Fibrosis Research - ETS (FFC Ricerca).

The molecule was designed by research groups coordinated by Prof. Adriana Chilin of the Department of Pharmaceutical Sciences at the University of Padua, and its anti-inflammatory activity has been confirmed by numerous trials coordinated by Prof. Ilaria Lampronti and Prof. Giulio Cabrini of the Department of Life Sciences and Biotechnology at the University of Ferrara and the Azienda Ospedaliera di Verona, with subsequent collaboration of a large network of Italian researchers.

Ownership of the orphan drug designation for GY971 has been assigned to FFC Ricerca, which as of this year is also supporting a new study on the molecule coordinated by the University of Ferrara in partnership with the University of Padua.

“Daily use of anti-inflammatory drugs in people with cystic fibrosis has been shown to significantly protect respiratory function over time. The only anti-inflammatory drug indicated for cystic fibrosis is ibuprofen, a compound widely used as a pain reliever and antipyretic, but in daily use in cystic fibrosis it produces serious side effects in the gastro-intestinal system, leading to its discontinuation,” clarifies Prof. Giulio Cabrini, Director of the Center for Research on Innovative Therapies for Cystic Fibrosis at the University of Ferrara, Innthera4CF, who coordinated the submission of GY971's dossier to EMA in collaboration with Dr. Marco Prosdocimi of Rare Partners and Dr. Ermanno Rizzi, of the scientific direction of FFC Ricerca.