Specialty pharmacy Orsini selected to provide SMA treatment Itvisma
It's the first gene therapy approved for older children and adults in US
- Itvisma is the first gene therapy for SMA patients ages 2 and older in the U.S.
- Administered intrathecally, Itvisma restores SMN protein production, addressing SMA's root cause.
- Specialty pharmacy Orsini was selected to provide this newly approved SMA gene therapy.
The specialty pharmacy Orsini was selected as a partner to provide Itvisma (onasemnogene abeparvovec-brve), the newly approved gene therapy for people with spinal muscular atrophy (SMA), ages 2 and older.
Itvisma, marketed by Novartis, is the first gene therapy approved in the U.S. for older children and adults with SMA. Zolgensma (onasemnogene abeparvovec-xioi), also sold by Novartis, is approved for use in children up to age 2. Both Itvisma and Zolgensma are part of Orsini’s Cell and Gene Therapy Center of Excellence.
“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 in a company press release. “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.”
Itvisma differs from Zolgensma in administration method, dosage
SMA is typically caused by mutations in the SMN1 gene that lead to progressive loss of motor neurons, the specialized nerve cells controlling movement, and disease symptoms that include muscle weakness and wasting.
Itvisma contains the same gene therapy construct as Zolgensma, packaged in a modified virus and designed to deliver a healthy version of the SMN1 gene to nerve cells. Both treatments address the root cause of SMA by restoring SMN protein production.
The key difference is that Zolgensma is delivered via intravenous infusion (infusion into the bloodstream) at a dose adjusted to the patient’s body weight, while Itvisma is given by injection into the spinal canal (intrathecal injection) at a recommended dose of 1.2×1014 vector genomes that is used in everyone from young children to adults.
Itvisma’s approval was supported primarily by two Phase 3 clinical trials, STEER (NCT05089656) and STRENGTH (NCT05386680), which tested the gene therapy in children and adolescents, ages 2 to 17, with SMA.
STEER demonstrated that Itvisma significantly improved motor function in patients who’d never previously been treated, while STRENGTH showed that patients’ motor function stabilized after switching from Spinraza (nusinersen) or Evrysdi (risdiplam).
The most common adverse reactions reported with the therapy across the two trials were upper respiratory tract infection, upper digestive symptoms, fever, and headache. Similar to Zolgensma, Itvisma may also cause serious liver injury, and corticosteroids should be given before and after injection to help prevent an immune response against the gene therapy.
“Today we support many neurology programs across multiple rare and complex conditions,” Orsini states on its website. “Our dedicated Therapy Care Teams work with the physician’s office and insurance on a patient’s behalf so that our patients and their family have more time to focus on their treatment and quality of life.”
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