Roche announced that the U.S. Food and Drug Administration (FDA) has approved Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children two months of age and older, according to a press release from Roche.
Evrysdi showed clinically-meaningful improvements in motor function across two clinical trials in people with varying ages and levels of disease severity, including Types 1, 2, and 3 SMA. Infants achieved the ability to sit without support for at least 5 seconds, a key motor milestone not normally seen in the natural course of the disease. Evrysdi also improved survival without permanent ventilation at 12 and 23 months, compared to natural history. A liquid medicine, Evrysdi is administered daily at home by mouth or feeding tube.
Evrysdi is being studied in more than 450 people as part of a large clinical trial program in SMA. The program includes people from two months to 60 years of age with varying symptoms and motor function, such as people with scoliosis or joint contractures, and those previously treated for SMA with another medication. The approval is based on data from two clinical studies designed to represent a broad spectrum of people living with SMA: FIREFISH in symptomatic infants aged two to seven months; and SUNFISH in children and adults aged two to 25 years. SUNFISH is the first and only placebo-controlled trial to include adults with Types 2 and 3 SMA.
Evrysdi is designed to treat SMA by increasing production of the survival motor neuron (SMN) protein. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
Evrysdi will be available in the United States within two weeks for direct delivery to patients’ homes through Accredo Health Group, an Express Scripts specialty pharmacy.
