Approvals

This extended authorisation follows interim data from the ongoing RAINBOWFISH trial assessing the drug for the treatment of pre-symptomatic babies from birth to six weeks with Type 1 SMA. The trial included six babies with two or three copies of the SMN2 gene who had completed at least a year of study assessments; all were able to sit after a year on Evrysdi, four could stand and three could walk independently.

The safety profile of the drug remained consistent with previous data, with the most common adverse reactions being fever, diarrhoea, rash, upper respiratory tract infection (including nasopharyngitis rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting and cough.

Levi Garraway, MD PhD, Roche’s chief medical officer and head of Global Product Development, added: “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.”

Dr Nicole Gusset, president and CEO of SMA Europe, commented: “The SMA community welcomes the EC’s decision to extend the use of Evrysdi from birth. Preserving motor neurons from the earliest age possible and preventing their irreversiblelosscanhaveasubstantial 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.”

The drug was approved in the EU in March 2021 for patients over the age of two months.