Also known as: Zolgensma
Onasemnogene abeparvovec is an AAV vector-based SMN1 gene replacement therapy used in spinal muscular atrophy treatment. In the U.S., FDA approved the intrathecal formulation onasemnogene abeparvovec-brve (Itvisma) in November 2025 for patients aged 2 years and older with confirmed SMN1 mutation. FDA also notes the active ingredient is identical to intravenous Zolgensma formulation used in younger pediatric SMA populations.
India is expanding rare disease support under the 2021 policy, with aid up to Rs 50 lakh and 15 Centres of Excellence. Officials also called for local innovation as treatment costs can reach ₹16 crore.
Novartis received a positive CHMP opinion for Itvisma in spinal muscular atrophy patients aged 2 years and older. A European Commission decision is expected within about two months.
Novartis is emphasizing innovative medicines across oncology, immunology, neuroscience and gene therapy. The company targets mid-single-digit sales growth through 2027 and core operating income margins above 35%.
International Rare Disease Day on February 28 underscores challenges in diagnosing and treating rare genetic conditions, with patients facing years-long diagnostic delays and geographic barriers to accessing gene therapies.
Former Novartis executive John Tsai has been appointed global head of R&D at Daiichi Sankyo, replacing Ken Takeshita effective April 1. Tsai brings over 25 years of leadership experience in drug development.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT05335876 |
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials |
RECRUITING | PHASE3 |
