UK Expands Referral Criteria for Zolgensma to Heavier Children

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by Steve Bryson, PhD |

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The U.K. has expanded the referral criteria for the approved gene therapy Zolgensma to include children with spinal muscular atrophy (SMA) who weigh up to 18 kilograms (40 lbs), according to a press release from SMA UK.

The expansion to a higher weight category — 15 to 18 kgs (33 to 40 lbs) — was advanced by the National Multidisciplinary Team (NMDT) which, along with infusion centers in England, provide services for children in England and Wales. The NMDT brings together infusion center representatives and others to ensure joint and equitable decision-making about the delivery of medicines such as Zolgensma.

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Initially, the NMDT invited referrals for children, 12 months or younger, who had never been treated (treatment naïve) or were untreated. This included those 6 months and younger who were pre-symptomatic, or newly diagnosed and untreated. Children 7 to 12 months who were newly diagnosed and untreated were also eligible for referrals.

Since then, the NMDT has been reviewing all potentially eligible children and expanded its inclusion criteria to children up to 13 months old who have been previously treated with either Spinraza (nusinersen) or Evrysdi (risdiplam) as a priority.

Other eligible patients include: untreated children 13 months or older, who weigh less than 13.5 kg (30 lbs) and are not receiving non-invasive breathing support, and those between 13 and 18 months weighing less than 13.5 kg. This is followed, in order, by children 18 months up to 24 months weighing less than 13.5 kg; those younger than 30 months and weighing less than 13.5 kg; children who weigh less than 13.5 kg; and finally, those under 15 kg (33 lbs).

Now, the NMDT is inviting referrals of children who are 15 kgs up to 18 kgs (33 to 40 lbs) for Zolgensma assessment. Currently, about 30 potentially eligible children fall into this category. Because few children in this weight group have been treated with the therapy, the needs of each child will be carefully considered.

First, following a referral to the NMDT from a Regional Neuromuscular Centre, the NMDT determines if gene therapy is appropriate. If deemed suitable, SMA children and their families are invited to an infusion center for a full assessment, followed by further discussion by the NMDT to decide eligibility.

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Infused directly into the bloodstream in a single dose based on the patient’s body weight, Zolgensma uses a modified and harmless adeno-associated virus to deliver a working copy of the SMN1 gene to cells. Nearly all cases of SMA are caused by a faulty SMN1 gene.

Zolgensma is approved in the U.S. for children up to 2 years of age with any SMA type. In Europe, the therapy was granted conditional approval for children weighing up to 21 kg (about 46 lbs) with SMA type 1 or those without symptoms but carrying up to three copies of the “backup” SMN2 gene.

The gene therapy is available in more than 40 countries, including Canada, Brazil, Switzerland, Australia, Japan, and South Korea, according to the therapy’s marketer Novartis.