On May 24, 2019, the gene therapy, Zolgensma, was FDA approved for pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. The initial wholesale acquisition cost (WAC) was $2,125,000, and today the WAC for Zolgensma is $2,322,040. The cost of this therapy was staggering in 2019, and while it opened the door for a new approach to treat these devasting diseases, it also paved the way for the acceptance of multi-million-dollar gene therapies. The most expensive gene therapy to date, FDA approved on March 18, 2024, is Lenmeldy, which was intended for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD). Its WAC is $4,250,000.
Our experience from 2019 to 2024 with Zolgensma has totaled nineteen (19) pediatric patients receiving this life-changing therapy with a drug cost of $41 million dollars. The breakdown of the level of benefit includes eleven (11) Medicaid members, six (6) commercial members, and two (2) self-funded members. With all 50 states proving SMA screening as part of newborn screening, it is allowing for the early detection and administration of Zolgensma. Our experience with the other gene therapies has not been as robust as with Zolgensma, although we did observe one case in 2024 for Elevidys at a drug cost of $3.2 million.
Looking forward into 2025, we are already experiencing an uptick in gene therapy with one (1) Zolgensma case, one (1) Elevidys case, two (2) Zynteglo cases, and one (1) Lyfgenia case. Not all gene therapies have been of interest to patients and doctors, such as the gene therapies for hemophilia for which there are existing effective standard treatments, concerns about side effects and duration of therapy, excessive cost, and limited patient access. Beqvez, which was FDA approved in April 2024, is being discontinued by Pfzier.[1] There are currently 14- FDA approved gene therapies and we expect to see the continuing utilization of these therapies in which there is limited or effective treatment for these rare genetic disorders.
Article by Kathy Clark, RN, BSN, CMCN, Vice President, Director of Managed Care. For more information about how this may affect your plan, please contact your Summit ReSources care specialist. The following sources were used as reference material for this article:
[1] Liu, Angus, “Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio”, FIERCE Pharma, February 21, 2025, https://www.fiercepharma.com/pharma/pfizer-empties-gene-therapy-portfolio-discontinues-hemophilia-treatment-beqvez. Accessed 2/24/2025.