Accredo Specialty Pharmacy Delivers Hawaii’s First Treatment of ZOLGENSMA Gene Therapy
Shipment of the first SMA gene therapy in Hawaii
Accredo by Evernorth® specialty pharmacy has achieved a major milestone by delivering the first shipment and administering the first treatment of ZOLGENSMA® in the state of Hawaii. ZOLGENSMA, U.S. Food and Drug Administration (FDA) approved in 2019, is manufactured by Novartis Gene Therapies.
ZOLGENSMA is a one-time gene therapy treatment for spinal muscular atrophy (SMA) in children under 2 years old. SMA is a rare and life-threatening genetic disease that affects motor nerve cells in the spinal cord usually discovered during a newborn screening. It impacts muscles used for breathing, eating and walking.1
Standard SMA screening for newborns in all 50 states
As of January 1, 2024, all 50 states added SMA to their newborn screening panels. In Hawaii’s Newborn Metabolic Screening Program, every newborn receives screening services regardless of income or insurance coverage. This was a crucial development in the fight against this devastating disease. Newborn screening allows for early detection of SMA, enabling prompt intervention, which can dramatically improve outcomes if administered in the early stages of the disease.2
The only FDA-approved gene therapy to treat SMA
Although ZOLGENSMA (onasemnogene abeparvovec-xioi), was approved by the FDA in 2019, it is still the only FDA-approved gene therapy to treat SMA.3 It works by addressing the underlying genetic cause of SMA and delivers a functional copy of the missing or defective SMN1 gene. ZOLGENSMA helps slow disease progression to potentially improve motor function, offering hope for patients, their families, and caregivers.
ZOLGENSMA’s first treatment in Hawaii opens the door for other families to access this therapy, giving babies diagnosed with SMA a better chance at a healthier future. This milestone also underscores the importance of a specialty pharmacy in delivering critical therapies and managing the complexities of treatment for rare diseases such as SMA.
Role of a specialty pharmacy
A specialty pharmacy plays a vital role in delivering and managing the treatment of a gene therapy such as ZOLGENSMA. Due to the complexity of gene therapies, Accredo specialty pharmacy helps ensure that patients receive the proper dose and care by handling intricate logistics, coordinating with health care providers, and managing cold-chain delivery of the therapy. Accredo’s expertise helps ensure that the therapy reaches the patient safely and on time, which is critical for ZOLGENSMA because it requires precise handling and quick shipping.
In addition to logistics, Accredo specialty pharmacy plays a pivotal role in the successful administration of ZOLGENSMA by providing personalized patient support, including counseling and education on the gene therapy before deciding to move forward with the treatment, assistance with insurance authorizations, and ongoing high-touch support for the patient and caregivers in the sensitive times before and after treatment.
Unique model goes beyond the prescription
With its unique blend of clinical and complex distribution expertise, Accredo’s Rare Disease Therapeutic Resource Center® (TRC) sets a benchmark for excellence in rare disease care. Accredo does everything to make living with complex condition and taking specialty medication as simple as possible. Learn more about how the Rare Disease TRC supports rare and gene therapies by downloading the Rare Disease TRC white paper here.
