The founding of PTC Therapeutics, Inc. (NASDAQ:PTCT), Inc over 20 years ago was driven by the need to create: a patient-centric approach to treating rare diseases. According to its CEO Stuart W. Peltz there has been enormous growth and progression of this vision, and today, the company boasts a wide portfolio of clinically differentiated medicines for rare disorders.
PTC has had the first commercial sale of Evrysdi™ (risdiplam) in the U.S. This is the first at-home treatment for spinal muscular atrophy (SMA) in adults to be administered at home. The sale birthed a $20 million milestone payment from Roche, a License and Collaboration Agreement with PTC.
The Making of and the Approval of Evrysdi™ (risdiplam)
Evrysdi has been taken through a robust clinical trial with over 450 patients and subjects participating. The subjects included infants aged 2 months and adults aged 60. They had to be suffering from a range of symptoms and motor functions, such as joint contractures or scoliosis. Results from two clinical studies demonstrated favorable efficacy and safety profile, which let to its approval by the FDA.
With the current COVID-19 global pandemic situation, the least patients want is to struggle with access to medication. Hence, there is nothing better than having a treatment delivered to patients directly at home. Evrysdi™ is administered in liquid form by mouth or feeding tube right from home.
The Search for Spinal Muscular Atrophy (SMA) Treatment Began Decades Ago
Spinal muscular atrophy (SMA) is a progressive neuromuscular disease and can also be severe. It is approximated that in every 11,000 babies, one is suffering from the devastating complication. SMA is also a rare disease. More often than not, it leads to the advanced loss of nerve cells. Some of its severe effects on a person include diminished or loss of the ability to walk, eat or breathe.
According to the President of the SMA Foundation, Loren Eng, a suitable cure for SMA began a long time ago. Hence, its collaboration with PTC is worth acknowledging. “PTC was one of the companies that embraced and remained committed to the idea of developing truly innovative therapies for this disease,” Eng Emphasized.