n 24 May, Novartis announced it had received approval for Zolgensma (AVXS-101), the only pediatric gene therapy to treat Spinal Muscular Atrophy Pipeline Market.
Although this drug has the potential to significantly change the lives of individuals with type 1 SMA, it also carries a hefty price tag of $2.125 million for a one-time treatment.
It is currently unclear how insurers will adapt to this paradigm of high prices for gene therapy cures, although Novartis is working with insurers to create new pay-over-time agreements.
Gene therapy drugs that can cure debilitating diseases are an incredible step forward in human healthcare, but questions remain regarding patient access to these treatment options.
Novartis appears to be placing a high degree of emphasis on innovation through the development of Zolgensma, but it is still focused on relatively rare diseases, as is evident through its pipeline, which has therapies for other types of SMA and Rett syndrome.
Zolgensma is currently the most expensive drug in the world, driven largely by the fact that it treats a disease with a relatively small patient pool, but an independent value-based review conducted by the Institute for Clinical and Economic Review (ICER) determined that its price was at the upper end of the estimated cost-effectiveness bracket.