One Dose, One Gene, One Cure? Itvisma as the World’s most expensive drug
Brand name: Itvisma
Generic name: Onasemnogene abeparvovec-brve
Category: Gene replacement therapy
Indication: Spinal muscular atrophy (SMA) in children aged 2 years and above
SMA is an autosomal-recessive neurodegenerative disorder caused by mutations in the SMN1 gene, characterized by irreversible and progressive motor neuron loss, leading to progressive muscle atrophy and weakness, and subsequent paralysis and death in the most severe cases.
Advantages
- It is administered as a single dose intrathecally.
- The fixed dose does not require adjustment based on age or body weight.
- It has a rapid onset of action and directly targets the genetic root cause of SMA.
- By replacing the SMN1gene, Itvisma can improve motor function, and reduce the need for chronic ongoing treatment.
Adverse effects: Upper respiratory tract infection, gastrointestinal symptoms, pyrexia and headache.
Warnings & Precautions: Hepatotoxicity, thrombocytopenia, ganglionopathy, peripheral sensory neuropathy, thrombotic microangiopathy, elevated cardiac troponin I and tumourogenicity.
Available strength: Single dose vial containing 1.2×1014 vg / 3ml suspension.
Cost: $2.59 million
Note: Prophylactic immune suppression with prednisone, tacrolimus, sirolimus or rapamycin is imperative to sustain gene expression without immune reactions.
References
- https://www.rheumatologyadvisor.com/news/one-time-gene-therapy-itvisma-approved-for-spinal-muscular-atrophy/
- https://checkrare.com/fda-approves-intrathecal-gene-therapy-for-patients-with-spinal-muscular-atrophy/
- Kagiava A and Kleopa KA. Facing the challenge of effective dosing, safety, and timing of intrathecal gene therapy for neurological disorders. eBioMedicine 2026;123: 106045.
