Rare disease pharmacy solutions provider Orsini announced on Tuesday that it has been chosen as 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 paediatric patients two years of age and older with confirmed mutation in the SMN1 gene.

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.

ITVISMA is designed to offer a functional copy of the SMN1 gene to improve motor function, via a one-time intrathecal injection. This is the ninth therapy to be added to the company's Cell and Gene Therapy Center of Excellence and the second SMA treatment to join the company's portfolio.

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