There’s positive news for Evrysdi, a treatment for spinal muscular atrophy (SMA). The EMA Committee for Medicinal Products for Human Use (CHMP) has recommended that the drug be approved, and they did so under the accelerated assessment pathway. Roche, the developer of Evrysdi, expects to hear a final decision on approval within the next two months.
About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is rare genetic order that is characterized by muscle weakness and degeneration. It affects one in every 10,000 people but can vary in severity from person to person. A mutation in the SMN1 causes this condition, and it causes the loss of motor neurons in the spinal cord and brain stem. Due to this loss, muscles weaken and atrophy, specifically the muscles used for walking, breathing, swallowing, crawling, sitting up, and moving the head.
The symptoms of this condition depend on the severity and type of SMA. Type I is the most severe, and is diagnosed after birth. The symptoms of this type include developmental delay, the inability to sit up or move the head, and trouble with breathing and swallowing. Type II is diagnosed in children between six and twelve months old, and they experience trouble with standing and walking. Those who have type III are diagnosed between early childhood and adolescence and cannot walk or climb stairs independently. Type IV occurs in adults over 30 and is the least severe type. People with this form of SMA usually have mild muscle weakness, problems with breathing, twitches, or tremors.
Once these symptoms are noticed, a diagnosis is confirmed through neurological examination and genetic testing. While there is no cure for SMA, there are treatments available. Physical therapy, braces, and surgery can all help to improve movement. Noninvasive respiratory support and tracheostomies can address issues with breathing while gastrostomy can help with eating. Spinraza is the first FDA approved treatment for SMA, and Zolgensma and Evrysdi have also recently been approved.
Positive Opinion for Evrysdi
Looking at data from Roche’s two trials of Evrysdi, SUNFISH and FIREFISH, CHMP has recommended that the treatment is approved by the European Commission. According to CHMP’s statement, they are suggesting approval for this drug for patients two months old and older who have been diagnosed with one to four SMN2 copies or Type 1, 2, or 3 of SMA.
The European Commission will take this recommendation and make a final decision within the next two months. This decision will impact all 27 countries who make up the European Union, as well as Norway, Liechtenstein, and Iceland. As Evrysdi has been placed in the accelerated assessment process, the decision could be made by the end of March at the earliest.
Evrysdi in the UK
Because the United Kingdom left the EU, Roche must submit separate applications to the MHRA under the EC Decision reliance procedure. This transitional approval process is new, and Evrysdi will be one of the first drugs to go through it. Despite this concern, Roche expects marketing authorization in the UK within the next few months.
Looking Forward
Roche is very hopeful that this positive opinion will lead to EMA approval for Evrysdi. As patients throughout the EU will benefit from this drug, they are looking forward to approval in the near future.
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