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FDA Grants Rare Pediatric Disease Designation to AMO Pharma for AMO-02 for Treatment of Congenital Myotonic Dystrophy

Designation qualifies AMO-02 for fast track review and AMO Pharma to receive a priority review voucher pending marketing approval.

LONDON, Nov. 9, 2020 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), 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.

About AMO Pharma

AMO Pharma is a biopharmaceutical company working to identify and advance promising therapies for the treatment of serious and debilitating diseases in patient populations with significant areas of unmet need, including rare and severe childhood onset neurogenetic disorders with limited or no treatment options. In addition to developing AMO-02 for congenital myotonic dystrophy, the company is progressing AMO-01 as a clinical stage treatment for Phelan-McDermid Syndrome and AMO-04 as a clinic ready potential medicine for Rett Syndrome and related disorders. AMO-02, AMO-01 and AMO-04 are investigational medicines that have not yet been approved for the treatment of patients anywhere in the world. For more information, please visit the AMO Pharma website at


Bill Berry
Berry & Company Public Relations

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Last Updated: 09-Nov-2020