Results from the Second Year of Evrysdi™ (risdiplam) Treatment Demonstrated Sustained Improvement of Motor Function in a Broad Range of SMA Patients
"We're encouraged by the long-term results from the SUNFISH trial, which reinforce the sustained clinical benefit that Evrysdi offers to a very broad population of patients with SMA, many of whom experience difficulty performing daily functions," said
Two-year data from SUNFISH Part 2 include efficacy and safety data in the overall population (2-25 years old):
- MFM32 (Motor Function Measure – 32), RULM (Revised Upper Limb Module) and HFMSE (Hammersmith Functional Motor Scale Expanded) scores showed improvement or maintenance of motor function during the second year of treatment with Evrysdi.
- Maintained motor function improvements between months 12 and 24 as measured by Motor Function Measure (MFM-32).
- Increased motor function as measured by Revised Upper Limb Module (RULM) and the Hammersmith Functional Motor Scale-Expanded (HFMSE) between months 12 and 24.
- Stabilized motor function for patients who began treatment with Evrysdi after 12 months of placebo as measured by MFM-32, RULM and HFMSE.
- Increased total score change from baseline, as measured by the caregiver-reported SMAIS (SMA Independence Scale) upper limb module, and the patient-reported SMAIS score stabilized between months 12 and 24.
Decreases in serious adverse events, high-grade adverse events and treatment-related adverse events were observed in the second year versus the first year in both treatment arms. The most common adverse events observed in the Evrysdi arm and the placebo and Evrysdi arm from 12-24 months were upper respiratory tract infection (15.8% and 10%, respectively), nasopharyngitis (21.7% and 16.7%, respectively), pyrexia (13.3% and 10%, respectively), headache (10% and 16.7%, respectively), diarrhea (7.5% and 10%, respectively), vomiting (11.7% and 13.3%, respectively) and cough (10% and 8.3%, respectively). The most common serious adverse events were pneumonia (6.7% and 0%, respectively) and influenza (0.8% and 0%, respectively).
Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement. Roche leads the clinical development of Evrysdi as part of a collaboration with the
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 throughout the central nervous system (CNS). Evrysdi is administered daily at home in liquid form by mouth or feeding tube. In
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.
About the Evrysdi Clinical Studies
FIREFISH (NCT02913482) - 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. Enrollment for Part 2 was completed in November 2018. 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) - 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 total score of Motor Function Measure 32 (MFM-32) 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.
In addition to FIREFISH and SUNFISH, Evrysdi™ is being evaluated in a broad range of people with SMA, including in:
JEWELFISH (NCT03032172) - 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).
RAINBOWFISH (NCT03779334) - 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 of age (at first dose), with genetically diagnosed SMA, who are not yet presenting with symptoms. The study is currently recruiting.
About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and left 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 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.
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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