Zolgensma is a gene therapy developed by AveXis for the treatment of spinal muscular atrophy. It was approved by the FDA in May of 2019, and was later cleared for use in Japan. It is now working towards approval in Europe. The EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval, which marks a large step towards bringing Zolgensma to Europe.
About Spinal Muscular Atrophy (SMA)
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 has also recently been approved.
Zolgensma in Europe
The CHMP has released a positive opinion of Zolgensma, which is heavily influenced by the results of the third phase of the STR1VE-US trial and the first phase of the START trial. The former of these two studies proved that 91% of participants met the co-primary endpoints after 14 months. The other endpoint, sitting up for at least half a minute without assistance at 18 months, was met by 59% of participants.
This positive opinion marks a large step towards approval for Zolgensma in Europe. Researchers hope that their gene therapy is given approval, as it will better the lives of those with SMA. Besides AveXis, other pharmaceutical companies are working towards treatments of SMA, all with the hope of improving the world of healthcare and the lives of people with spinal muscular atrophy.
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