StemCell Therapy

Nearly five months after snagging a conditional approval for its spinal muscular atrophy (SMA) gene therapy in Europe, Novartis is rolling out fresh data to support a launch that has seen its share of challenges.

The company unveiled interim data from a phase 3 trial of the gene therapy in babies born with the genetic disease. Of the 33 patients treated so far in the European trial, 21 had achieved milestones in motor skills during a mean follow-up period of 10.6 months that the disease would normally prevent, the company said. For example, six could sit without assistance for more than 10 seconds and 20 could control their head movements.

Most of the children in the study who entered it without requiring ventilation remained free of ventilatorsupport, and 67% were able to eat without help, Novartis said during the World Muscle Society 2020 Virtual Congress. Most of the children received a higher score on a widely used test of neuromuscular functioning than untreated SMA patients typically do, the company added.

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Shephard Mpofu, M.D., chief medical officer of Novartis Gene Therapies, said in a statement that the data boosts previous clinical trial results supporting the use of Zolgensma in patients with type 1 SMA, the most common form of the disease. With more than 600 patients now treated, including some more than five years post-treatment and more than five years old, these data further reinforce the transformative benefit a one-time dose of Zolgensma has on SMA patients, Mpofu said.

RELATED: New Zolgensma 'inflection point' is here as Novartis snags EU nod for SMA gene therapy

Novartis effort to win approval for Zolgensma in Europe ran into one hurdle back in April 2019, when an infant in the trial died of brain damage and respiratory issues. An autopsy later revealed that the death was unrelated to Zolgensma.

But then, in October of last year, Novartis was hit with questions from regulators in Europe and Japan about the Zolgensma manufacturing process. Resolving the queries from the EU required inspections, and it pushed back thedecision from the EUs Committee for Medicinal Products for Human Use (CHMP).

Now that Zolgensma is finally approved in Europe, though, Novartis has an opportunity to steal market share from Biogens SMA drug Spinraza. Thats because the European approval allows the Zolgensma to be used in children weighing up to 21 kilograms, which basically covers any child under the age of 5.

Thats a significant difference from the FDA approval, which only covers children under 2 years of ageand it could allow Novartis to offer its therapy to older children currently taking Spinraza.

RELATED: Roche touts 2-year Evrysdi data as oral SMA drug drives early interest

Novartis has an ambitious expansion plan for Zolgensma in the U.S., too, though that has hit some obstacles recently. Its working on a new intrathecal formulation of the drug, in the hopes that the FDA will approve that version for children up to 5 years old. But last week, the company said the FDA will require it to run a phase 3 study of the intrathecal formulation, rather than considering it for approval based on a smaller phase 1/2 trial. That could push the filing to 2023.

Analysts deemed the FDAs decision a major setback. Jefferies analysts had estimated the gene therapy would peak at $2.8 billion in worldwide sales, with the intrathecal version claiming $1 billion of that.

Meanwhile, Novartis is facing potential competition from more than just Spinraza. Roche introduced an oral drug for SMA, Evrysdi, last month. And it just revealed new data from a pivotal trial of that drug that intensify Novartis marketing challenge.

In the Evrysdi study, 88% of infants with type 1 SMA were alive without needing full-time ventilation after two years. Their motor functioning also improved in the second year, with more patients able to sit, stand, turn over or maintain head control.

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Novartis cites 'transformative' data on Zolgensma as it rolls out SMA gene therapy in Europe - FiercePharma

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