AMO Pharma Limited , a privately held biopharmaceutical company focusing on rare, childhood onset neurogenetic disorders with limited or no treatment options, today announced that the U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease (RPD) designation to AMO-Pharma for AMO-02, the company's investigational therapy in development for the treatment of congenital myotonic dystrophy.

The FDA grants RPD designation for serious and life-threatening diseases that primarily affect children aged 18 years or younger and fewer than 200,000 people in the United States. The designation qualifies AMO Pharma to receive fast track review for AMO-02 and a priority review voucher (PRV) at time of marketing approval. PRVs are transferable and can be used by drug developers to earn an expedited six-month review of a new drug application by the FDA. In recent years, sale prices for PRVs have ranged from $67 million to $350 million.

"This Rare Pediatric Disease designation highlights the significant need for a treatment for congenital myotonic dystrophy and adds additional momentum to our development program," said Ibraheem Mahmood, CEO of AMO Pharma. "The option of monetizing a PRV voucher also represents a significant opportunity to support our mission to develop novel medicines for children with developmental disorders."

The AMO-02 clinical trial is a double-blind, placebo controlled, randomized study in children and adolescents with congenital onset myotonic dystrophy intended to support a future submission for marketing authorization in congenital myotonic dystrophy. The trial is being conducted at eleven treatment centers globally and plans to enroll a total of 56 patients who will be assessed on a range of measures of CNS features and muscle function associated with congenital myotonic dystrophy.