Evrysdi Research Updates from Genentech

2021-03-18T14:04:21-05:00March 18th, 2021|Clinical Trials, Front Page News, Research|

Genentech, a member of the Roche Group, logo

New 2-year data show Evrysdi continues to demonstrate improvement or maintenance of motor function in people aged 2-25 years with Type 2 or 3 SMA

Genentech, a member of the Roche Group, recently shared new exploratory 2-year, longer-term data from Part 2 of SUNFISH, a global placebo-controlled study evaluating Evrysdi (risdiplam) in people aged 2-25 years with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). The study suggests that gains in motor function observed with Evrysdi treatment at month 12 continued to improve or were maintained at month 24 across primary and secondary endpoint measures. Based on the natural history of SMA, people with Types 2 and 3 SMA who remain untreated decline in motor function over time.

Patients in SUNFISH Part 2 ranged in age from 2-25 years and were treated with Evrysdi (n=120) or placebo and Evrysdi (n=60; patients in the placebo arm received placebo for 12 months followed by Evrysdi treatment for 12 months). The study evaluated a number of exploratory 24-month endpoints, which provide important insights into motor function and its impact on daily life. Findings demonstrated that Evrysdi:

  • Maintained motor function improvements between months 12-24 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**** 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.

* MFM is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. It assesses 32 different motor functions from standing and walking through to use of hands and fingers. 

** RULM is a scale designed to assess upper limb movement in people with SMA. It can capture progressive muscle weakness across the spectrum of the disease, reflective of the SUNFISH Part 2 study population. 

*** HFMSE is intended to be used in assessing the functional motor abilities of people with SMA who are able to sit and walk.

**** SMAIS was developed to measure self-reported and caregiver-reported independence.

About Evrysdi™ (risdiplam)

Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA by increasing and sustaining production of the survival of motor neuron (SMN) protein. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube. The U.S. Food and Drug Administration (FDA) approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older in August of 2020. More than 2,500 patients have now been treated with Evrysdi in clinical trial, compassionate use, and real-world settings across the globe.

These data were presented during the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific Conference

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