Biotech Biogen’s Spinraza follow-up helps children with SMA who didn’t benefit from Zolgensma By James Waldron Jun 25, 2025 8:04am ...

Zolgensma is a prescription drug that’s used to treat spinal muscular atrophy (SMA) in young children. Zolgensma’s cost may depend on factors such as your child’s treatment plan and whether ...

The only other treatment for SMA is a drug called Spinraza (made by Biogen). But unlike Zolgensma, which is administered just once, Spinraza must be taken for life. The first year of Spinraza costs ...

New data showing Zolgensma’s substantial benefits for presymptomatic children made the drug cost-effective at prices up to $1.9 million by one benchmark and up to $2.1 million by another, it said.

It’s not the first time RegenXBio has used this type of deal to generate some much-needed cash. Back in 2020, the biopharma sold $200 million worth of Zolgensma royalties to HealthCare Royalty.

Novartis said Zolgensma has been "transformative," but each child's journey is unique and outcomes can vary. It says there is no evidence additional therapies can help. Notably, it has dropped the ...

Since an initial FDA nod in 2019, Zolgensma has been cleared in over 40 countries and has become a blockbuster therapy. It reeled in $379 million in sales in the second quarter, a 26% increase ...

Zolgensma may cause elevated enzymes, which can lead to serious liver damage. If your child has a history of liver problems, they may be at a higher risk of developing this side effect.

Zolgensma is prescribed to treat SMA in certain children ages 2 years and younger. The dosage is based on the child’s body weight in kilograms (kg). For reference, 1 kg equals about 2.2 pounds (lb).