The FDA approval of Evrysdi is based on data from two clinical studies, dubbed FIREFISH in symptomatic infants, and SUNFISH in children and adults
Evrysdi product photo. (Credit: Business Wire.)
Genentech, a subsidiary of the Roche Group, has received the US Food and Drug Administration (FDA) approval for Evrysdi (risdiplam) to treat spinal muscular atrophy (SMA) in adult and paediatric patients, aged two months and older.
Evrysdi is survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. The drug is in liquid form and can be administered daily through the mouth or a feeding tube at home.
The drug works by boosting production of the survival of motor neuron (SMN) protein, which is responsible for maintaining healthy motor neurons and movement, to treat SMA.
Evrysdi has been granted European Medicines Agency (EMA) PRIME designation in 2018, FDA Orphan Drug Designation in 2017, followed by similar designation from EMA in 2019.
Roche chief medical officer and global product development head Levi Garraway said: “Given the majority of people with SMA in the US remain untreated, we believe Evrysdi, with its favourable clinical profile and oral administration, may offer meaningful benefits for many living with this rare neurological disease.
“The strength and resolve of the SMA community has continually inspired us as we developed this first-of-its-kind medicine for SMA, so today we celebrate our collective accomplishment together with them.”
The FDA approval of Evrysdi is based on data from FIREFISH and SUNFISH clinical trials
Genentech has been conducting a clinical development programme for Evrysdi, under collaboration with the SMA Foundation and PTC Therapeutics.
The FDA approval is based on data from two clinical studies, dubbed FIREFISH in symptomatic infants aged 2 to 7 months, and SUNFISH in children and adults aged 2 to 25 years.
The biotechnology company said that its Evrysdi has demonstrated clinically meaningful improvements in motor function, in the clinical trials that evaluated 450 people with different ages and disease severity levels, including types 1, 2, and 3 SMA.
In the clinical trials, treatment using Evrysdi enabled children to sit without support for at least 5 seconds, which marks an important motor milestone and is not naturally observed in the course of the disease.
Also, the drug has shown a positive efficacy and safety profile, consistent with the safety profile established across the FIREFISH and SUNFISH trials, said the company.
Cure SMA president Kenneth Hobby said: “Throughout their lives, many people with SMA may lose their ability to perform critical movements, which can impact the ability to independently participate in aspects of daily life and even be life-altering.
“The approval of Evrysdi is an eagerly awaited milestone for our community. We appreciate Genentech’s commitment to reflecting the full scope of the real-world SMA population in their clinical trial programme and developing a treatment that can be administered at home.”