Orsini Partners with ITVISMA® to Enhance Treatment Options for SMA Patients

Orsini, a well-respected name in rare disease pharmacy solutions, has announced that it will be providing specialty pharmacy services for ITVISMA® (onasemnogene abeparvovec-brve). This innovative therapy is specifically designed for the treatment of spinal muscular atrophy (SMA), a genetic disorder that significantly impairs motor function.

SMA affects individuals due to the lack of a gene that encodes the survival motor neuron (SMN) protein, pivotal for the proper functioning of motor neurons. Approximately 1 in 11,000 births are affected by SMA, demonstrating its prevalence as a critical healthcare challenge in both adult and pediatric populations. Symptoms of the disease often emerge in early childhood and progress with age, influencing the patient's motor abilities.

ITVISMA® stands out as a one-time intrathecal gene therapy aimed at replacing the missing SMN1 gene, thus providing a functional copy that can improve motor functions. Orsini's commitment to treating rare diseases is evident in its continuous efforts to expand services; ITVISMA marks their ninth addition to the Cell and Gene Therapy Center of Excellence.

Darin DeCarlo, the Chief Commercial Officer at Orsini, stated, "Since 2019, we have been deeply invested in the SMA community by supporting families and patients living with this rare disease. The approval of ITVISMA allows us to further extend our care to older pediatric and adult patients, ensuring that everyone affected by SMA has access to these transformative gene therapies.”

This announcement comes as a beacon of hope to many families grappling with the effects of SMA, who often face significant challenges in accessing suitable therapies. Orsini aims to simplify the process for patients by offering comprehensive solutions that integrate pharmacy distribution, clinical management, and home infusion services—guaranteeing that no patient is left behind.

The introduction of ITVISMA balances the existing portfolio of Orsini, which already includes ZOLGENSMA® (onasemnogene abeparvovec-xioi), a previously approved intravenous option for SMA patients under the age of two. These enhanced options highlight a significant step in the therapeutic landscape of SMA, demonstrating Orsini's dedication to pioneering treatments and supporting families in their healthcare journeys.

Orsini has been recognized for its passionate commitment to providing care since 1987, working tirelessly with biopharma innovators, healthcare providers, and payors to drive patient access to vital treatments for rare diseases. The company has garnered accreditations from various health authorities, which solidify its status as a leader in rare diseases and gene therapies.

Interested parties can learn more about ITVISMA and Orsini’s offerings by visiting www.orsini.com. As the landscape of genetic therapies continues to evolve, patients and families can remain hopeful that advancements like ITVISMA will pave the way for new avenues of treatment and care.