Wednesday, August 30, 2023
Roche (SIX: RO, ROG; OTCQX: RHHBY) has received approval from the European Commission to expand the use of Evrysdi® (risdiplam) within the European Union (EU). This extension includes the treatment of infants diagnosed with spinal muscular atrophy (SMA) Types 1, 2, or 3, as well as those with one to four copies of the SMN2 gene, from birth to under two months old. The decision is based on interim data from the ongoing RAINBOWFISH trial, which focuses on pre-symptomatic babies with Type 1 SMA. This data supported the expansion of Evrysdi's usage.
Dr. Nicole Gusset, President & CEO of SMA Europe, expressed approval for this extension, highlighting the significance of early treatment in preserving motor neurons and improving long-term mobility. Levi Garraway, M.D., Ph.D., Roche's Chief Medical Officer and Head of Global Product Development, noted that this label extension allows for the treatment of babies shortly after birth, enhancing their potential to achieve developmental milestones.
The decision was based on an interim analysis of the RAINBOWFISH trial, involving 18 participants, including infants with 2 or 3 copies of the SMN2 gene. After a year of treatment with Evrysdi, all six babies achieved the ability to sit, four out of six could stand, and three out of six could walk independently. Moreover, all infants were alive after 12 months without requiring permanent ventilation.
The safety profile of Evrysdi in pre-symptomatic babies, as shown by the RAINBOWFISH trial, remained consistent with previous trials involving symptomatic SMA patients. Common adverse reactions included fever, diarrhea, rash, upper and lower respiratory tract infections, constipation, vomiting, and cough.
Evrysdi was initially approved in Europe in March 2021 for patients aged two months and older. It is administered in liquid form through the mouth or feeding tube and works by increasing and sustaining the production of SMN protein, which is crucial for maintaining healthy motor neurons and various bodily functions. Evrysdi is also being evaluated in multiple trials involving individuals with SMA, and Roche is researching its use in combination with an anti-myostatin molecule to enhance muscle growth.
SMA, a severe neuromuscular disease, affects around one in 10,000 babies and is a major genetic cause of infant mortality. Roche's efforts in neuroscience encompass various neurological disorders, striving to develop innovative treatments that address the challenges posed by these conditions.
