Referral Criteria for Zolgensma Has Been Expanded in the UK

According to a recent article in SMA News Today, more spinal muscular atrophy (SMA) patients in the United Kingdom (UK) will have access to Zolgensma. This change comes after the UK’s National Multidisciplinary Team (NMDT) expanded its referral criteria for the gene therapy to include children weighing up to 18 kg (~40 lbs).

About Expanded Referral Criteria

The referral criteria was altered by the NMDT, which brings together providers of care, such as infusion centers, and other important individuals within the distribution and delivery of medications. Specifically, the change in criteria impacted the weight requirements for Zolgensma; the maximum weight rose from 15 kg to 18 kg.

This is not the first time that the referral criteria has been updated. Going back to the beginning, the following groups were eligible for referral:

  • Treatment naïve or untreated children up to 12 months old
    • The group is separated into various categories
      • Recently diagnosed and untreated children 6 months and younger
      • Pre-symptomatic children 6 months and younger
      • Newly diagnosed and untreated children 7 to 12 months old

The list has only grown from there, as the NMDT continuously reviews and expands its criteria as time goes on. After various updates, the following groups were also included:

  • Previously treated (with either Evrsydi or Spinraza) children up to 13 months old
    • This expansion was a priority
  • Untreated children 13 months or older who are not on non-invasive breathing support and weigh less than 13.5 kg
  • Children 13 to 18 months old who weigh less than 13.5 kg

After a number of continuous expansions to the criteria, the NMDT ended at children weighing up to 15 kgs.  Now, the criteria has been updated again, raising the maximum weight to 18 kg.

Because of this update, 30 children with SMA are now eligible for referral. It’s important to note that their needs will be considered carefully and on an individual basis, as there is little history of treating children in this category.

What Happens After a Referral?

The first step is to visit a Regional Neuromuscular Centre, where an SMA patient can be referred to the NMDT. They then decide if gene therapy is the right step, and if it is, the patient and their family visit an infusion center. Here, they undergo a full assessment. As this happens, conversation continues with the NMDT to decide if the patient is eligible. If they are, the next step is treatment with Zolgensma.

About Zolgensma

Zolgensma delivers a functional copy of the SMN1 gene through an adeno-associated virus (AAV). It is administered in a single dose directly into the bloodstream via infusion. Dosage is based on the patient’s body weight.

About Spinal Muscular Atrophy (SMA)

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. A mutated SMN1 gene 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.

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