PTC Therapeutics Announces First Commercial Milestone Payment Following U.S. FDA Approval of Evrysdi™ (risdiplam)

SOUTH PLAINFIELD, N.J., Aug. 26, 2020 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company achieved a $20 million milestone payment from Roche under its License and Collaboration Agreement. The milestone payment was triggered by the first commercial sale of Evrysdi™ (risdiplam) in the U.S. Evrysdi™ was approved by the Food and Drug Administration (FDA) on Aug. 7, for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. 

"We are excited that Evrysdi™ is able to reach U.S. SMA patients so quickly following FDA approval,” said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "This is especially relevant during the COVID-19 global pandemic, given that Evrysdi™ can be delivered directly to patients at home.”

Evrysdi™ is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi™ is administered daily at home in liquid form by mouth or feeding tube. Evrysdi™ (risdiplam) is based on PTC science and is commercialized in the United States by Genentech, a member of the Roche Group. Roche led the clinical development of Evrysdi™ as part of a collaboration with the SMA Foundation and PTC Therapeutics.

About Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies. SMA leads to the progressive loss of nerve cells in the spinal cord that control muscle movement. Depending on the type of SMA, an individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene that results in a deficiency of SMN protein. SMN protein is found throughout the body and increasing evidence suggests SMA is a multi-system disorder and the loss of SMN protein may affect many tissues and cells, which can stop the body from functioning.

About PTC Therapeutics, Inc.

PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.

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