As the number of U.S. spinal muscular atrophy (SMA) patients Zolgensma treats each quarter stabilizes, Novartis is counting on a set of inflection points for future growth. Now, it has one.

After a manufacturing-related delay, Novartis has won conditional approval in the EU for the one-time gene therapy to treat patients with a clinical diagnosis of SMA type 1 and others with up to three copies of the SMN2 backup gene, the company said Monday.

Zolgensmas EU label is different from the U.S. version. While its approved by the FDA to treat children less than 2 years of age, the EMA allows it in babies and young children who weigh up to 21 kilograms. According to a Pediatric Neuromuscular Clinical Research natural history study of SMA, almost all patients under the age of 5 will be under 21 kg.

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The number of SMN2 genes determines the severity of the disease, with SMA type 1 the most severe form. The U.S. approval is for all SMA types, but the EU nod leaves out a small proportion of patients who could develop mild, late-onset type 3 or type 4 SMA. Patients with type 3 SMA, sometimes called Kugelberg-Welander disease, may have up to four copies of SMN2.

The difference could havemixed effectson Zolgensmas opportunity in the two territories. For existing patients already on Biogens Spinraza, Zolgensma could steal share from older patients in the EU. But for new patients, the FDA label enables the Novartis drug to reach more patients if diagnosis through newborn screening is widely adopted and patients are treated early.

Zolgensma has reached a steady state where it treats about 100 patients per quarter in the U.S., Novartis CEO Vas Narasimhan recently told investors. That translated into $170 million sales in the first quarter, a slight quarter-over-quarterdecline due to COVID-19. The companys expecting approvals in new countriesand new indications to be inflection points that will eventually propel the drug to blockbuster sales.

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It had the first point in March with a Japanese nod, also for patients under 2 years old. Last week, the Japanese government approved Zolgensmas price at 167 million yen ($1.55 million), lower than its U.S. list price of $2.12 million.

In Europe, final pricing and reimbursement decisions will be determined at the local level, a spokesperson at Novartis AveXis gene therapy unit told FiercePharma. Cumulative healthcare costs per SMA patient are estimated at between 2.5 million to 4 million over the first 10 years, the company said.

Novartis said its working with European countries local regulators on an access program called Day One. Its designed to provide fast access to Zolgensma even before national pricing and reimbursement agreements are in place, a process that sometimes takes years. The program offers several options such as deferred payments and installment over years as well as outcomes-based rebates. The drug has been made available in France under the countrys Temporary Authorization for Use pathway, and access is expected shortly in Germany, the Swiss drugmaker said.

Beyond the already-approved intravenous version, Novartis is developing a formulation that delivers Zolgensma through an injection into the spinal canal so that it can reach older patients up to 5 years of age in the U.S. Its working with the FDA to resolve a partial clinical hold slapped on the high dose of the intrathecal formulation before it can file for an approval.

Currently, AveXis makes Zolgensma at its site in Libertyville, Illinois. It also has plants in Durham, North Carolina, and Longmont, Colorado. The latter two are expected to be licensed in 2021, the company spokesperson said, adding that it has no plans for a manufacturing facility in the EU at this point.

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