Zolgensma, a gene therapy for spinal muscular atrophy (SMA), has recently been granted conditional approval by the European Commission (EC). This is extremely exciting news for those living with SMA in Europe, especially as it is estimated that up to 600 babies are born with this condition every year. Fortunately, affected individuals will not have to worry about an astronomical price (Zolgensma costs $2.1 million), as a Day One access program has been put into place.
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 has also recently been approved.
Approval in Europe
The EC has only granted Zolgensma conditional approval, as AveXis, the creator, is still working with the European Medicines Agency to finalize the timelines for supply. This has allowed for access pathways to be created in France and Germany, with other countries following closely.
Not only will patients be aided by this decision, but the European healthcare systems will be helped as well. Before Zolgensma, it was estimated that it could cost up to $4 million for each child with SMA within the first ten years of their lives. This high price came from repetitive treatments. Zolgensma only needs to be administered once, greatly reducing the cost and time spent on treating SMA patients.
Pricing is something else that has yet to be finalized, as the cost of Zolgensma is decided locally so that it best fits each community. Fortunately, a Day One access program has been created. This program ensures that those who receive the therapy before reimbursement and local price have been put into place will still receive that fair price after it is agreed upon.
EC approval came after the positive results of trials that evaluated Zolgensma. The Phase 3 STR1VE-US has been completed, joined by the first phase of the START trial. Both of these studies examined the safety and efficacy of Zolgensma after a one time infusion. The STR1VE-EU trial is in its third phase and still ongoing, as is the Phase 3 SPR1NT trial.
All of these studies have shown positive results, with improvement of motor function, event-free survival, the ability to walk, crawl, and sit independently, and age appropriate milestone gain.
It is these results and EC approval that bring excitement to SMA patients and their families all across Europe.
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