Swedish neurologists warn ALS patients may have to self-pay for new gene therapy

Swedish doctors fear that ALS patients in Sweden may have to pay out of pocket for a treatment that targets a genetic cause of the neurodegenerative disease ALS. The EU Commission will decide this spring whether the drug can be marketed in Europe.

A  30-year-old Swedish man with the neurodegenerative disease ALS (amyotrophic lateral sclerosis) caused by a mutation on the SOD-1 gene has become the first patient from a Nordic country to see significant improvement while being treated with the new gene therapy tofersen, known as Qalsody.

This was a significant breakthrough, according to Swedish doctors, as there is currently no curative treatment for ALS patients.

However, after the story broke in late April other symptomatic ALS patients having the same targeted mutation, said they had been denied treatment in Sweden.

Tofersen is the world’s first therapy to target a specific gene mutation that causes ALS. It is given once a month by injections into the cerebrospinal fluid in the spinal cord. It is considered a lifelong medication.

He still walks

The 30-year-old Swede was told in 2020 that he had up to two years to live, according to doctors. He was then included in a phase 3 tofersen trial at a university hospital in Denmark. After four years of treatment, he still walks and looks after his children.

The drug was recommended for approval in the EU this year by the European Medicines Agency’s scientific committee under ‘exceptional circumstances’.

The  EMA is expected to decide later this spring whether or not to green light the medicine for the EU market.

The applicant is the pharmaceutical company Biogen, which has a licensing agreement with the original developer, Ionis Pharmaceutical.

Biogen offers hospitals the opportunity to enrol patients in an early access programme (EAP), where the treatment is provided free of charge.

In the US, where the drug was approved in 2023, Biogen is reportedly charging €14,230 per monthly injection.

Estimates of the number of patients who may be eligible for treatment in Sweden vary between 12 and 40.

Even if the drug does not have a market authorisation, Swedish doctors can prescribe it as a licensed product or experimental drug and help patients into the EAP.

New therapies council advised against

However, last summer, the New Therapies Council (NT Council), a joint body from the Swedish regions, decided to advise doctors not to prescribe tofersen as a licensed product on a national basis.

This is a standard procedure, Åsa Rangert Derolf, chair of the NT Council, told Euractiv.

“Until we have negotiated prices with pharmaceutical companies, medicines are often very expensive, and our task is to ensure that we have fair and equitable access to new medicines through the efficient use of our shared resources. Hence the discouragement of licensing,” she said.

Mikko Fernström, Head of External Affairs at Biogen Nordics & Baltics, told Euractiv that the company has provided free access to the drug to more than 500 eligible patients across more than 30 countries, including 18 EU countries, as a licensed prescription since 2021.

But despite the NT Council’s advice, an unknown number of patients in Sweden are participating in the EAP.

”Biogen has successfully provided access to tofersen through the named patient access route. Unfortunately, we cannot comment on specific patient numbers,” Mikko Fernström told Euractiv.

Neuro Sweden: “Cynical”

However, according to Lise Lidbäck, chairwoman of the patient organisation Neuro Sweden, a handful of patients with the targeted form of ALS have told her they are being denied treatment because of future costs. She said she finds this cynical.

“As patient advocates, we look forward to more effective drugs and, of course, to patients benefiting from them as soon as possible. We are now witnessing a frightening scenario where our health authorities seem to be blocking opportunities that are available to patients in other countries,” she told Euractiv.

Swedish neurologists, such as Caroline Ingre, Associate Professor and Senior Consultant in Neurology at Karolinska University Hospital, recently warned that patients may have to pay for the injections themselves if tofersen is approved.

If so, they may be advised to seek help abroad.

“We must try to help them find other ways, such as the possibility of getting the medicine in other countries. If the NT Council advises against it, I assume that the Swedish hospitals are not willing to pay for either the medicine or the trip abroad. It could be that a patient has to pay for everything on their own,” Caroline Ingre said in an interview in Läkartidningen.

One of the Swedish patients whose doctor refuses to give him the drug is Erik, 56 years old.

Muscles weaken

Erik was diagnosed with the targeted form of ALS a month ago and is desperate as the muscles in his legs and arms weaken.

“My neurologist refuses to give me tofersen and has explained that she is afraid that the hospital would have to pay for it in the future,” he told Euractiv.

According to Swedish neurologists, good results from treatment with tofersen have been seen in about 40 Belgian and German patients participating in Biogen’s EAP since 2022.

An earlier randomised clinical trial with tofersen showed no significant benefit for patients.

But a longer study – the phase 3 VALOR trial – showed a 60 per cent reduction in plasma neurofilament light chain (NfL) in participants receiving tofersen compared to a placebo group, suggesting reduced neuronal injury, Mikko Fernström at Biogen told Euractiv.

Mild side effects were reported by Biogen during clinical trials, as well as some serious neurological events such as myelitis, radiculitis and increased intracranial pressure.

[By Monica Kleja, edited by Vasiliki Angouridi, Brian Maguire | Euractiv’s Advocacy Lab]

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