To provide a brighter future for infants battling spinal muscular atrophy (SMA), Roche has unveiled a momentous development. The European Commission has granted its seal of approval for the extension of the Evrysdi (risdiplam) marketing authorization within the European Union (EU). This extension paints a canvas of possibility for infants, encompassing those diagnosed with SMA Type 1, Type 2, or Type 3, along with those bearing one to four SMN2 copies, from birth to just under two months.
A pivotal beacon of this approval shines from the ongoing RAINBOWFISH trial, where pre-symptomatic babies, from birth to six weeks, holding the torch of Type 1 SMA, are under observation. The glow of hope is fostered by interim data, a snapshot of potential, which has added momentum to this authorization extension.
“The SMA community welcomes the European Commission’s decision to extend the use of Evrysdi from birth. Preserving motor neurons from the earliest age possible and preventing their irreversible loss can have a substantial impact on a person’s future ability to move and function. We look forward to continued collaborative efforts to improve diagnosis, including newborn screening, and ensuring all individuals living with SMA have access to medicines.”
– Dr Nicole Gusset, President & CEO of SMA Europe
The crux of this transformation rests upon the RAINBOWFISH interim analysis, which welcomed 18 participants into its embrace. Within this cohort, six infants bore the genetic tapestry of 2 or 3 copies of the SMN2 gene, a mosaic that often defines the trajectory of SMA. A symphony of achievement resonated as 100% of these babies managed to assume a seated posture after a year of Evrysdi treatment. Further notes echoed – 67% could stand, and a triumphant 50% could even take their first steps unaided. Vitality flowed through their journey, for at the 12-month milestone, the gentle rhythm of breath sustained them, unburdened by the shackles of permanent ventilation.
The colors of this newfound hope paint a portrait of consistency, for the safety profile of Evrysdi in these pre-symptomatic infants mirrors the landscape observed in previous trials that navigated the realm of symptomatic SMA patients. Within this canvas, notes of adversity emerged as fever, diarrhea, rash, and a host of respiratory tract infections made an appearance. However, in the grand tapestry of progress, these challenges were met with resilience.
“With this label extension, we can treat babies soon after birth with Evrysdi, allowing them the greatest chance to achieve the milestones of sitting, standing and walking, similar to healthy children. ”
– Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development
The narrative of Evrysdi extends beyond this chapter, for its initial debut on the European stage transpired in March 2021. Its entrance into the arena was underpinned by the intricate threads of clinical data woven from the SUNFISH and FIREFISH studies, which paved the way for its approval.
Roche’s commitment to unraveling the complexities of SMA persists. In the Ph II/III trial MANATEE, Evrysdi finds itself in the company of an anti-myostatin molecule, aiming to unlock the secrets of muscle growth for those ensnared in the clutches of SMA.
In the heart of this development lies a poignant message – a symphony of science and compassion, harmonizing to rewrite the stories of infants who once grappled with the shadows of SMA. The extension of Evrysdi’s embrace is an ode to resilience, a nod to innovation, and a timeless reminder that the pursuit of progress is a symphony worth composing.
