The company said that oral pirfenidone (Esbriet) has shown promise to slow IPF disease progression and a very large oral dose is required to achieve efficacious lung levels.
Genoa president and chief executive officer Mark Surber said acquiring orphan status marks an important regulatory milestone in GP-101’s life cycle to treat people with this devastating disease.
"We are pleased to continue the development of inhaled GP-101, with clinical trials beginning in early 2015," Surber said.
Despite being established at the upper safety threshold (801mg TID), the resulting oral-delivered lung dose is too low for optimal effect.
In order to address oral shortcomings and maximize IPF efficacy, the company has reformulated pirfenidone for aerosol formation and inhaled, direct-lung delivery (GP-101).