Germany first in EU to get Novartis' SMA gene therapy, costing almost 2m euros

Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) in Germany at the start of July priced at 1,945,000 euros, and says it hopes to reach a “smart deal” allowing UK patients access by the end of the year. 

The European Commission granted a conditional marketing authorisation for Zolgensma at the end of last month, covering children and babies weighing up to 21 kg with the most severe form of the muscle wasting disease. 

A company spokesperson told pharmaphorum that patients in Germany are likely to get the drug first, as drug pricing is decided by each member state and negotiations with payers in other major European countries are ongoing. 

Newly-launched drugs enjoy a year of free pricing in Germany, before the country’s AMNOG cost-effectiveness system assesses their benefits compared with standard care and sets a price. 

The company is also engaged in private discussions with payers in other European countries in the build-up to other national launches, the company told pharmaphorum. 

AveXis has priced Zolgensma at around $2.1 million in the US and $1.55 million in Japan. Zolgensma (onasemnogene abeparvovec) is already considered to be the world’s most expensive drug.

But the Swiss company’s AveXis unit argues that its high price is justified considering the lifetime cost of treating the disease of between 2.5 to 4 million euros. 

Mike Fraser, general manager EMEA at AveXis, said: “All gene therapies are required to go through the benefit assessment process (AMNOG) in Germany and this will be ongoing in the coming months, potentially making Germany one of the first countries to make gene therapy available as a treatment option for SMA patients.

Smart deal in UK

Fraser added that in the UK, AveXis already began talks with NICE in 2018 and the drug is being assessed using its Highly Specialised Technology pathway, but said that the review had been “impacted somewhat” by the COVID-19 emergency.

Novartis managed to get the UK to agree to a “smart deal” on pricing for its CAR-T cancer cell therapy Kymriah, meaning patients in the country were the first to get access outside of the US.

“We are confident that we shall reach an agreement so that families can access our gene therapy. We are keen for that to happen this year,” said Fraser. 

“In countries such as France, AveXis is leveraging existing early access funding pathways. Whilst in other countries, AveXis is already in advanced discussions to agree on terms to enable immediate access.” 

Elsewhere in Europe the company hopes to offer flexible pricing options including early access rebates, deferred payments and installment options, and outcomes-based rebates. 

Zolgensma can be used in patients with inherited mutations affecting genes known as SMN1, who have either been diagnosed with SMA type 1 - the most severe type - or have up to 3 copies of another gene known as SMN2. 

Around 550-600 babies are born with the disease in Europe each year, which causes rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. 

Zolgensma will compete against another highly expensive drug - Biogen's Spinraza (nusinersen), while Roche is also developing a potential rival called risdiplam that is slated for a European filing later this year.