Rare Daily Staff
Cerecor, a company focused on developing medicines for rare and orphan diseases in pediatrics and neurology, said it has been awarded Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for CERC-801.
CERC-801 is a genetically-targeted, substrate replacement therapy to treat an ultra-rare inherited metabolic disorder known as an Inborn Error of Metabolism (IEM). CERC-801 was originally developed by Ichorion, which Cerecor acquired in September 2018.
Inborn Errors of Metabolism form a large class of genetic diseases involving congenital disorders of metabolism. The majority are due to defects of single genes that code for enzymes that facilitate conversion of substrates into products and/or intermediates. In most cases, issues arise due to accumulation of substances that are toxic or interfere with normal function, or due to the effects of reduced ability to synthesize essential compounds.
“We are excited by the rapid response from the FDA and its recognition of the need for therapies for patients suffering from these serious and life-threatening disorders with no approved treatment,” said Peter Greenleaf, CEO of Cerecor.
The FDA grants RPDD to programs addressing rare diseases or conditions that in which serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years. If a new drug application for CERC-801 is approved, Cerecor is eligible to receive a priority review voucher for another compound, which it could use itself, or sell to another company.
Cerecor is also developing three pre-clinical stage compounds, CERC-611, CERC-406, and CERC-913. Its R&D efforts are supported by revenue from a franchise of commercial multivitamin medications.
October 31, 2018
Photo: Peter Greenleaf, CEO of Cerecor.