GB Pharma: Born as a CRO
Becomes a Pharmaceutical Company - Biotech
Adopting an Innovative Business Model Through Drug Repurposing
In 2010, GB Pharma S.r.l. adopted a new business model focused on drug repurposing opportunities. In the same year, the company obtained its own SIS code from AIFA (Italian Medicines Agency) and received Marketing Authorization (MA) for a well-known small molecule, originally registered and approved for its initial indication many years earlier.
Orphan Drug Designation for a Rare Disease
Following this achievement, GB Pharma successfully obtained Orphan Drug Designation (ODD) for the use of this small molecule in the treatment of a rare disease. The mechanism of action is unique, as the drug works through two complementary pathways: a direct effect and a hormone-mediated mechanism related to rare genetic conditions.
Ongoing Clinical Development
The clinical development program of this repurposed drug is currently ongoing. In parallel, in-vitro studies and preclinical experiments are being conducted to further validate its therapeutic potential.