Orsini Selected as Specialty Pharmacy Partner for ITVISMA® (onasemnogene abeparvovec-brve)

ITVISMA is an FDA-approved intrathecal gene therapy for the treatment of spinal muscular atrophy

ELK GROVE VILLAGE, Ill., Dec. 2, 2025 /PRNewswire/ -- Orsini, a leader in rare disease pharmacy solutions, announced today that it is a specialty pharmacy partner for ITVISMA® (onasemnogene abeparvovec-brve), an FDA-approved intrathecal gene therapy for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in SMN1 gene. Read the full Prescribing Information here.

SMA is a genetic disease that affects the central nervous system, peripheral nervous system and voluntary muscle movement, caused by a missing gene that encodes the survival motor neuron (SMN) protein. SMA occurs in nearly 1 in 11,000 births in the United States, and the age at which symptoms begin tends to correlate with the severity to which motor function is affected. ITVISMA is designed to provide a functional copy of the SMN1 gene to improve motor function, through a one-time intrathecal injection. It is the ninth therapy to be added to Orsini's Cell and Gene Therapy Center of Excellence and the second SMA treatment join Orsini's portfolio. The first is ZOLGENSMA® (onasemnogene abeparvovec-xioi), a previously approved intravenous therapy that contains the same active ingredient as ITVISMA, indicated for the treatment of SMA patients less than 2 years of age.

"Since 2019, Orsini has been honored to serve the SMA community by supporting families and patients living with this rare disease," Darin DeCarlo, Chief Commercial Officer at Orsini, said. "With the approval of ITVISMA, we are both excited to expand our care to older pediatric and adult patients and eager to work to ensure that everyone affected by SMA can access these life-changing gene therapies."

About Orsini
Providing patients with comprehensive and compassionate care since 1987, Orsini is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers and payors to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated rare disease pharmacy solutions including pharmacy distribution, patient services, clinical management and convenient home infusion services, Orsini simplifies how patients connect to advanced therapies. Orsini's high-touch care model centers on experienced and trained therapy care teams that provide personalized patient care to ensure that No Patient is Left Behind™.

Orsini holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC and NABP. Orsini has earned URAC's Rare Disease Pharmacy Center of Excellence Designation and ACHC's Distinction in Rare Diseases and Orphan Drugs. For more information, visit www.orsini.com.

SOURCE Orsini