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Novartis claims EU approval for SMA gene therapy Zolgensma

Company in discussion with EU countries over pricing in the hope of launching it quickly


Novartis has secured conditional approval from the EMA for its spinal muscular atrophy (SMA) gene therapy Zolgensma, and says it is talking with EU countries over pricing in the hope of launching it quickly.

The approval covers the use of Zolgensma (onasemnogene abeparvovec) for patients with a clinical diagnosis of SMA type 1, the most severe form of the disease, as well as for any SMA patient who has three copies of the SMN2 gene.

The approval comes after the EMA asked for more information on manufacturing of the gene therapy, which delayed its review by several months.

Zolgensma was approved in the US a year ago, becoming the world’s most expensive drug with a price of $2.1m per patient, and also got a green light in Japan in March where it has been given a lower price of around $1.55m dollars.

Novartis is offering a ‘day one’ access programme to EU governments and reimbursement agencies, and says access to the drug is already available in France and will be shortly in Germany

That price drove the drug to sales of $361m last year, adding another $170m in the first quarter of this year, which was actually a slight decline on the last three months of 2020, but Novartis is confident that Zolgensma will be another blockbuster product, and GlobalData has tipped iot to make $2.5bn a year at its peak.

The EU approval covers babies and young children with SMA up to 21kg, said Novartis. There are around 550-600 infants born every year in Europe with SMA, which causes rapid and irreversible loss of motor neurons, affecting muscle function and leading to problems with breathing, swallowing and movement.

SMA is a devastating condition which, in the most severe cases, leaves babies with a life expectancy of little more than two years.

Zolgensma will launch in Europe as a rival to Biogen’s existing biologic treatment for SMA, Spinraza (nusinersen), that made its debut in Europe in January 2017 for SMA types 1, 2, 3 and 4 and was the first drug approved to treat the disease.

Spinraza was a $2bn product last year, still growing at a healthy rate (22%) despite the arrival of Zolgensma on the US market, as it is used across a broader spectrum of SMA patients including older patients.

Novartis is developing a version of Zolgensma for injection into the spinal column rather than intravenously, which would expand the use of the gene therapy into older children, but that has been held up by an FDA clinical hold to scrutinise its safety.

The excitement surrounding Zolgensma lies in its role as a one-shot therapy for SMA that over time could both improve the clinical outcome for SMA patients and reduce the overall cost of care, which in Europe is estimated to be between €2.5m and €4m in the first ten years of life.

In the pivotal phase 3 STR1VE and the completed phase 1 START trials used by the FDA to make its decision, all 15 infants treated with Zolgensma were alive and off permanent breathing assistance at two years.

Novartis acquired Zolgensma as part of its $8.7bn takeover of US-based biotech AveXis in 2018.

Article by
Phil Taylor

19th May 2020

From: Research, Regulatory, Healthcare



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