the pan-Canadian Pharmaceutical Alliance (pCPA) on a Letter of Intent (LOI) for Zolgensma® (onasemnogene abeparvovec), which is an adeno-associated virus (AAV) vector-based gene therapy specified for treating pediatric patients who suffer from 5q spinal muscular atrophy (SMA) as well as biallelic mutations in the survival motor neuron 1 (SMN1) gene and 3 or fewer copies of SMN2 gene; or infantile-onset SMA1.
Andrea Marazzi, Country Pharma Organization Head at Novartis Pharmaceuticals Canada, expressed hopes to work with Zolgensma. He said, “We know the SMA community has been waiting for this day and we’re thrilled that children with SMA are one step closer to having provincial access to Zolgensma. We look forward to working with provincial jurisdictions to make Zolgensma available as quickly as possible through public formularies to ensure all children who may benefit from this one-time therapy can access it.”
Susi Vander Wyk, Executive Director at Cure SMA Canada, expressed delight over the partnership. He said, “This is encouraging news for the SMA community across Canada. However, motor neuron damage from SMA…
