Roche’s spinal muscular atrophy (SMA) therapy has been approved by the European Commission (EC) to treat infants aged under two months, the company announced.
Evrysdi (risdiplam), which is already authorised for use in patients aged two months or older, can now be used to treat infants from birth to below two months with a clinical diagnosis of SMA type 1, 2 or 3, or with one to four SMN2 copies.
SMA is a severe and progressive neuromuscular disease affecting approximately one in every 10,000 babies.
Those living with the condition have insufficient levels of the SMN protein, which is essential to the function of nerves that control muscles and other functions such as swallowing, speaking, breathing and movement.
Roche’s Evrysdi, which can be administered at home in liquid form by mouth or by feeding tube, is designed to treat the disease by increasing and sustaining the production of SMN protein in the central nervous system and peripheral tissues.
The EC’s decision to extend the EU marketing authorisation for the therapy follows a recent recommendation from the European Medicines Agency’s human medicines committee and is supported by an interim analysis of the ongoing RAINBOWFISH study, which included six babies with two or three copies of the SMN2 gene.
After one year of treatment with Evrysdi, all six infants were able to sit, four could stand, and three could walk independently.
The interim analysis also showed that the safety profile of Evrysdi in pre-symptomatic babies was consistent with the safety profile seen in previous trials with symptomatic SMA patients, Roche said.
The company’s chief medical officer and head of global product development, Levi Garraway, said: “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.”
Also commenting on the approval, Dr Nicole Gusset, president and chief executive officer of SMA Europe, said: “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.”