AMO Pharma Receives FDA Orphan Drug Designation For AMO-02 For Treatment Of Congenital Myotonic Dystrophy

LONDON and PHILADELPHIA, July 27, 2017 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for AMO-02, the company's investigational therapy in development for the treatment of congenital myotonic dystrophy. 

"This is an important regulatory milestone for our congenital myotonic dystrophy program as we work to bring an effective treatment to this challenging disorder," said Michael Snape, chief executive officer of AMO Pharma.

AMO has initiated a phase 2 clinical trial in the United Kingdom for AMO-02.  This phase 2 exploratory study includes patients with congenital onset myotonic dystrophy. There are currently no approved treatments for myotonic dystrophy, which affects an estimated 1 out of 8,000 people.

Orphan drug designation is granted by the FDA's Office of Orphan Products Development for drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.

About Myotonic Dystrophy
Myotonic dystrophy is the most common form of muscular dystrophy, affecting approximately 1 in 8,000 people. It is a progressive disease and the predominance of symptoms such as muscle weakness, intellectual/developmental impairment or cognitive/behavioral changes is determined by a number of factors, including age at onset. There are currently no approved therapies indicated for the treatment of myotonic dystrophy.  AMO Pharma is currently conducting clinical stage research in individuals with congenital onset myotonic dystrophy.

About AMO Pharma
AMO Pharma is a biopharmaceutical company incorporated in February of 2015. The co-founder, Dr. Michael Snape, has extensive experience in senior scientific and operational roles in both large pharma and biotech companies spanning more than twenty five years, and has brought together a targeted and experienced senior management team with a proven track record of success in all phases of product development and acquisition. The company is 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 genetic diseases. AMO Pharma is currently advancing investigational therapies for treatment of congenital myotonic dystrophy and similar genetically determined orphan disorders. For more information, please visit the AMO Pharma website at


Mike Snape, PhD
Chief Executive Officer
AMO Pharma Ltd.
+44 1483 898 448

Sara Zelkovic
Berry & Company Public Relations
212 253 8881


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SOURCE AMO Pharma Limited

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