SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Evrysdi™ (risdiplam) was approved in Japan by the Japanese Ministry of Health, Labor and Welfare for the treatment of spinal muscular atrophy (SMA). The approval has been granted based on data from two pivotal studies evaluating Evrysdi in infants and adults living with type 1, 2 or 3 SMA. The first commercial sale will trigger a $10M milestone payment to PTC from Roche under its License and Collaboration agreement.
“We are thrilled to see the Evrysdi approval in Japan and are excited that SMA patients have an effective and convenient at home, oral treatment option that avoids the need for in-hospital administration,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “The rapid adoption of Evrysdi globally underscores the importance of this drug and the profound benefit this therapy provides to SMA patients.”
Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement. Evrysdi has been approved for the treatment of patients with SMA, aged 2 months and older by the FDA and the EMA. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Evrysdi is marketed in the United States by Genentech, a member of the Roche Group.
About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies and when untreated is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to progressive muscle weakness over time. 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.
About Evrysdi™ (risdiplam)
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 designed to distribute evenly to all parts of the body, including the central nervous system (CNS). Evrysdi™ is administered daily at home in liquid form by mouth or feeding tube. The U.S. Food and Drug Administration approved Evrysdi™ (risdiplam) in August 2020 for the treatment of spinal muscular atrophy for adults and children 2 months and older. Evrysdi™ (risdiplam) is marketed in the United States by Genentech, a member of the Roche Group.
About the Evrysdi™ (risdiplam) Clinical Studies
FIREFISH (NCT02913482) is an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of risdiplam in 41 infants with Type 1 SMA treated for two years followed by an open-label extension. The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed in the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III) (defined as sitting without support for five seconds). The study met its primary endpoint.
SUNFISH (NCT02908685) is a two-part, double-blind, placebo controlled pivotal study in people aged 2 to 25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using the Motor Function Measure 32 (MFM-32) scale at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.
RAINBOWFISH (NCT03779334) is an open-label, single-arm, multi-center study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in babies (~n=25), from birth to 6 weeks old (at first dose), with genetically diagnosed SMA, who are not yet presenting symptoms. The study is currently recruiting.
JEWELFISH (NCT03032172) is an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi™. The study has completed recruitment (n=174).
Clinical Trial Safety Data
The safety profile of Evrysdi™ was established across FIREFISH and SUNFISH pivotal trials. The most common adverse reactions in later-onset SMA (incidence of at least 10 percent of patients treated with Evrysdi™ and more frequently than control) were fever, diarrhea, and rash. The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, the most common adverse reactions (incidence of at least 10 percent) were upper respiratory tract infection, pneumonia, constipation, and vomiting.
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. The Company’s strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. 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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