The Food and Drug Administration has given another approval to a gene therapy, this time for a rare disease that causes paralysis.

Swiss drugmaker Novartis announced the approval of Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy (SMA) in children younger than 2 who have biallelic mutations in the SMN1 gene. The original developer of Zolgensma was AveXis, which Novartis acquired last year for $8.7 billion.

The therapy is an adeno-associated viral vector-based therapy. The first gene therapy approved by the FDA was Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), for biallelic RPE65 mutation-associated retinal dystrophy, a genetic disease that causes blindness, in December 2017.

In a conference call with reporters Friday, AveXis President Dave Lennon noted that the therapy would cost $2.125 million, paid in installments over five years at $425,000 per year, under a partnership with the specialty pharmacy Accredo. AveXis is negotiating with payers to create five-year, outcomes-based agreements for the therapy.

Ahead of the approval, reports had emerged that the therapy would cost as much as $5 million, but Novartis CEO Vas Narasimhan told reporters at an event Wednesday that it would be priced “far lower” than that, according to Reuters. In the Friday conference call, Lennon said the price point would be below the costs of current chronic treatment for SMA and for extremely rare, genetic diseases in children.

The company further noted that the price point would be 50 percent below the Institute for Clinical and Economic Review’s cost-effectiveness threshold for ultra-rare diseases, with a cost of about $250,000 per quality-adjusted life year.

The company is ready to launch the product immediately, having set up a production site in Illinois, with plans for additional sites as well.

Another approved therapy for SMA is Biogen’s Spinraza (nusinersen). However, Lennon pointed out that the two products will not necessarily compete, given the different indications. Spinraza, an antisense oligonucleotide drug, is approved for SMA in children and adults.

At the Veeva Summit in Philadelphia last week, Spark CEO Jeff Marrazzo said installment payments could potentially be used across the gene therapy space, but that policies at the Centers for Medicare and Medicaid Services had been slow to adapt. Novartis has a deal with Spark to market Luxturna outside the US. Another Swiss drugmaker, Roche, agreed to acquire Philadelphia-based Spark for $4.8 billion in February.

Photo: jxfzsy, Getty Images

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