Novartis Pharmaceuticals Canada is lauding the province of Quebec for its decision to provide public reimbursement for Zolgensma (onasemnogene abeparvovec), an approved gene therapy for spinal muscular atrophy (SMA) in children. Zolgensma is the first gene therapy to receive formal public reimbursement in Canada. The current recommendation…

The U.S. Food and Drug Administration (FDA) has added acute liver failure to the list of safety concerns with use of Zolgensma (onasemnogene abeparvovec-xioi) in children with spinal muscular atrophy (SMA). The therapy’s label has been updated to contain a boxed warning highlighting the risk of higher-than-normal…

Novartis has completed negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) regarding pricing and availability of Zolgensma (onasemnogene abeparvovec), its approved gene therapy for spinal muscular atrophy (SMA), for pediatric patients. With this negotiation completed, individual public drug plans in Canada will now make final decisions about the coverage…

The Canadian Agency for Drugs and Technologies in Health (CADTH) will not recommend reimbursement for Zolgensma in children with spinal muscular atrophy (SMA) who are more than 6 months old, citing insufficient evidence of efficacy and safety in older patients. “Other countries are providing guidance and funding based…

Biogen is continuing to actively recruit patients for its Phase 4 RESPOND trial, which is evaluating the benefits of Spinraza (nusinersen) in infants and children with spinal muscular atrophy (SMA) who responded poorly to the gene therapy Zolgensma. Participants are now being enrolled at 10 sites in the U.S. and one in Italy,…

Despite its high cost, giving the one-time gene therapy Zolgensma to an infant identified through newborn screening with spinal muscular atrophy (SMA) is cheaper in the long run than starting treatment with Spinraza (nusinersen) after symptom onset, according to a cost-effectiveness study in Australia. Combining newborn screening (NBS) and early…

The gene therapy Zolgensma safely improved mobility in young children with spinal muscular atrophy (SMA) living in Qatar, a real-world study shows. The case series study, “Gene therapy for spinal muscular atrophy: the Qatari experience,” was published in the journal Nature Gene Therapy. Zolgensma…

Daily treatment with Evrysdi (risdiplam) following the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and leads to motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. While children were followed for a short time after Evrysdi was…

New data about Zolgensma, the one-time gene therapy for children with spinal muscular atrophy (SMA), demonstrate age-appropriate development when used pre-symptomatically, and rapid, meaningful efficacy in symptomatic children, even those with severe disease before treatment, according to two Phase 3 clinical trials. The therapy, developed by Novartis, uses…

England’s National Institute for Health and Care Excellence (NICE) issued its final Zolgensma’s appraisal, recommending that the gene therapy be covered by the country’s national health service (NHS) and provided at low or no cost to eligible spinal muscular atrophy (SMA) patients. The positive recommendation is consistent with England NHS’…