Tofersen's EMA approval: When is it coming (and how to get the medicine in the meantime)

Last updated: 21 February 2024

In 2023, the FDA granted accelerated approval to Biogen's Qalsody (tofersen) for the treatment of ALS. Specifically, ALS associated with an SOD1 gene mutation ¹. While this is positive news, patients based in Europe and the UK are still facing the uncertainty of waiting for local approval.

Here's everything you need to know about tofersen's EMA approval timeline, and your options to get the medicine in the meantime.

What is Qalsody (tofersen) used for?

Qalsody(tofersen) is a prescription medicine for the treatment of ALS patients with the SOD1 gene mutation (SOD1-ALS). Its active ingredient is tofersen - an antisense oligonucleotide, which works by interfering with the production of the faulty SOD1 protein.

Tofersen is not a cure for ALS. Instead, it aims to slow down the disease progression.

Does tofersen really work for SOD1-ALS?

Tofersen's accelerated FDA approval was granted on the basis of the VALOR clinical trial results. For the approval to be continued, the medicine's efficacy needs to be confirmed in a phase 3 trial, which is currently ongoing.

Although the VALOR trial did not meet its primary objective (a significant reduction in average ALSFRS-R scores), it demonstrated a significant decrease in neurofilament light chain (NfL) in the patients' blood. NfL is an indicator of nerve damage and deterioration. That's why this result was taken as an indication of slowing down the progression of the disease ⁴.

These are the key highlights from the VALOR trial:

Patients receiving Qalsody experienced less decline in average ALSFRS-R scores than the placebo group. However, this difference was not statistically significant;
At 28 weeks of treatment, patients receiving Qalsody saw a 55% decrease in NfL blood levels. The placebo group, on the other hand, experienced a 12% increase ⁴.

A recent report on real-life results from a German early access program with tofersen confirmed the VALOR trial results. It reported a significant reduction in NfL serum levels at 6 months and at 9 months of treatment⁶. The report's conclusion was that tofersen is an effective therapeutic approach for SOD1-ALS patients.

Tofersen and life expectancy

12 months after the start of the VALOR study, data was collected about life expectancy of patients. According to the published report, SOD1-ALS patients showed consistent slowing of disease progression with tofersen. Results reportedly favored earlier treatment with tofersen, citing better scores on clinical function, respiratory function, muscle strength and quality of life for patients who started treatment at the start of the VALOR trial versus 6 months later.

The trial couldn't establish a median time to death or permanent ventilation (PV). However, early survival data suggested a lower risk of death or PV within 12 months, with earlier tofersen initiation.

Tofersen's EMA approval: What's the status?

In December 2022, the EMA accepted a marketing authorization application for Qalsody². As of February 2024, the application is still under review.

When will tofersen be available in Europe?

Typically, EMA approval decisions are made within 210 days. However, in tofersen's case the process has taken longer. This may be due to the CHMP waiting for additional data on the medicine's efficacy. For example, the recently published report from the German early access program ³.

As soon as tofersen gets EMA approval, each member state can start their own process for making the medicine available on the local market. For orphan drugs such as Qalsody, this can take on average from 102 days (Germany) to 1,081 days (Estonia) ⁷.

With this in mind, it's technically possible that Qalsody becomes available in some European countries within 2024. However, a more specific timeline is difficult to provide.

Does tofersen have UK approval?

As of February 2024, not yet. According to the post-Brexit European Commission Decision Reliance Procedure, the MHRA may rely on EMA decisions for its local approvals. In other words, the MHRA could approve Qalsody as soon as the EMA does.

However, before Qalsody can become available on the NHS, it must also be assessed by NICE. This assessment considers cost-effectiveness, clinical trial evidence, and input from experts and patients. Tofersen's assessment by NICE is ongoing, expected to conclude by mid-2024. That's positive news, as it means that if the MHRA approves the medicine, it will already be on track to NHS availability.

Assuming a positive assessment from all agencies, tofersen could be available in the UK within 2024.

How to safely get Qalsody (tofersen) before its EMA approval

Does your doctor believe that you could benefit from treatment with Qalsody? Then you're probably frustrated by the delay in getting EMA approval for the medicine. In that case, there's good news. You don't have to wait.

There are multiple options to get tofersen before it's widely available outside the USA. One option is joining a clinical trial. Another is via Biogen's early access program. And a third option is to buy Qalsody immediately as a Named Patient.

Here's a bit more about each option.

Join a tofersen clinical trial

Joining an ongoing clinical trial is a good way to get quick access to Qalsody. Finding trials can be challenging, but it can be done. To participate, you must meet the eligibility criteria and have your doctor's support. Also keep in mind that you might receive a placebo instead of the active ingredient.

Here are some good places to start looking for ongoing clinical trials:

ClinicalTrials.gov: This is a database with all clinical trials in the USA. However, some of the trials are also open to international participants. At the moment, the ATLAS study is recruiting international participants. However, new trials may become available at any time, so it's worth keeping an eye on this list.
EUClinicaltrials.eu: This database contains all clinical trials in the European Union. Currently, it contains limited information on trials launched before 31 January 2022. For those trials, you can refer to the EU Clinical Trials Register.
myTomorrows and FindMeCure: These organizations can help you find other clinical trial options.

Get tofersen via Biogen's early access program (Compassionate use)

Biogen started an early access program (EAP) for tofersen in July 2021. The program is active in countries where that's allowed, offering free access to the medicine before it's locally approved.

To be considered for the program, your treating doctor needs to submit a request to Biogen directly via [email protected]. Note that submitting a request doesn't guarantee access to the program.

Buy tofersen as a Named Patient

If joining a clinical trial or the EAP aren’t an option for you, you can also buy tofersen right away as a Named Patient. This is made possible thanks to the Named Patient Import regulations in place in most countries. To make use of these regulations, you need to have a prescription for Qalsody(tofersen) from your treating doctor.

Already have a prescription? Send it to our team of Medicine Access experts and we'll support you in buying tofersen immediately. Wherever you are based.

Tofersen cost

If you're considering buying a medicine that isn't available on your local market yet, you should take prices as indicative only. The final cost will vary based on your location, the supplier, and any shipping or importation fees that might apply to safely deliver the medicine.

As an indication of tofersen costs per year, a single-dose vial (100 mg/15 mL) currently costs about EUR 30,850. At the start of your treatment, you'd need 3 doses administered at 14-day intervals. After that, you'd need one dose every 28 days. Roughly, this brings the yearly cost of a tofersen treatment to EUR 448,400.

Buy tofersen

Final thoughts

Ultimately, the decision to seek access to Qalsody(tofersen) before its EMA approval is between you and your doctor. It's essential to approach this process with realistic expectations and a thorough understanding of the potential risks and benefits involved. This being said, when you're ready to start your treatment, our team is here to help you skip the wait.