FDA Grants PPI-1040 Orphan Drug Designation

Med-Life Discoveries LP (MLD) is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted PPI-1040, a proprietary synthetic plasmalogen replacement, Orphan Drug Designation (ODD) for the treatment of Rhizomelic Chondrodysplasia Punctata (RCDP). 

RCDP is an ultra-rare pediatric disease with an estimated prevalence of less than 1 in 100,000. The disease is the result of a plasmalogen lipid deficiency caused by mutations in genes involved in plasmalogen biosynthesis. PPI-1040 is a proprietary synthetic vinyl-ether compound designed to replace deficient plasmalogens for the treatment of RCDP. MLD has recently begun enrolling participants into an RCDP Natural History Study at the Alfred I. DuPont Hospital for Children in Wilmington, DE, as part of the clinical development program for PPI-1040.

The FDA’s ODD program is designed to support drugs in development for diseases which affect fewer than 200,000 individuals in the U.S. Drug candidates with ODD status off the sponsor a number of potential incentives to promote drug development, including a seven-year period of U.S. marketing exclusivity, waiver of the FDA user fees, tax credits for clinical research, and additional FDA support in clinical trial design. It is important to stress however that the ODD program does not change the FDA’s requirements for demonstrating the safety and efficacy of a drug before it receives marketing approval.