Pharma BD Deal Intelligence
Novartis's $8.7B all-cash acquisition of AveXis delivered Zolgensma, the gene therapy for spinal muscular atrophy that became a landmark approval and a scalable AAV manufacturing platform. The bet on gene therapy paid off commercially, though the broader pipeline—Rett Syndrome and ALS programs built on the same platform—has yet to reach the same milestone.
Novartis acquired AveXis for approximately $8.7 billion ($218 per share in cash), gaining the gene therapy AVXS-101 (later approved as Zolgensma/onasemnogene abeparvovec) for spinal muscular atrophy, along with a scalable gene therapy manufacturing platform and pipeline programs in Rett Syndrome and ALS.
500 US cases/yr
SMA Type 2 (intermediate) presents between 6-18 months. Patients achieve sitting but not independent walking. All three SMA therapies show benefit, though earlier treatment yields better outcomes.
Same three approved therapies: Zolgensma (primarily for younger patients), Spinraza, Evrysdi. Ongoing debate on optimal sequencing and combination approaches.
400 US cases/yr · $5.0B Global SMA therapeutics
SMA Type 1 is the most severe form, presenting in infancy with progressive motor neuron loss. Three transformative therapies exist: Zolgensma (gene therapy), Spinraza (antisense), and Evrysdi (SMN2 splicing modifier).
Zolgensma: one-time IV gene therapy ($2.1M). Spinraza: intrathecal, ongoing. Evrysdi: oral, daily. Newborn screening expanding early treatment. Key players: Novartis (Zolgensma), Biogen (Spinraza), Roche (Evrysdi).
350 US cases/yr · $1.0B ALS therapeutics (all causes)
SOD1-mutated ALS accounts for ~2% of all ALS cases. Tofersen (Qalsody) received accelerated FDA approval in 2023 as the first therapy targeting a genetic cause of ALS.
Tofersen (Qalsody, Biogen): intrathecal antisense targeting SOD1 mRNA. Gene therapy approaches in preclinical development. Key player: Biogen (Qalsody).
300 US cases/yr · $500M Emerging Rett syndrome therapeutics
Rett syndrome is a rare X-linked neurodevelopmental disorder caused by MECP2 mutations, almost exclusively affecting girls. Trofinetide (Daybue) became the first FDA-approved treatment in 2023. Gene therapy approaches in development.
Daybue (trofinetide) approved 2023 for ages 2+. Gene therapy programs (AveXis/Novartis AVXS-201) in early development. Supportive care (seizure management, PT/OT). Key player: Acadia (Daybue).
Novartis acquired AveXis to gain Zolgensma gene therapy for SMA, the leading genetic cause of infant death.
Zolgensma approved as first gene therapy for SMA at $2.125M per dose, becoming the most expensive drug in the world.
FDA accused AveXis of manipulating mice testing data in Zolgensma application but ultimately declined to punish the company.
AveXis rebranded to Novartis Gene Therapies, signaling gene therapy strategic importance to the company.
Solid Return Despite Plateauing Sales. Assessment of Novartis AG's $8.7B acquisition of AveXis Inc. and its strategic outcomes.
| Deal | Year | Value | Outcome |
|---|---|---|---|
| Novartis AG / AveXis Inc. (this deal) | 2018 | $8.7B | 68 |
| Novartis AG / Advanced Accelerator Applications S.A. | 2017 | $3.9B | 98 |
| Novartis AG / Endocyte, Inc. | 2018 | $2.1B | 96 |
| Novartis AG / Lek Pharmaceuticals d.d. | 2002 | $876M | 88 |
| Novartis AG / GlaxoSmithKline plc | 2014 | $16.0B | 81 |
| Novartis AG / PTC Therapeutics Inc. | 2024 | $2.9B | 80 |
| Novartis AG / Chinook Therapeutics Inc. | 2023 | $3.5B | 78 |
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More: 2018 deals · Novartis AG deals · Gene Therapy deals