EMA Approves Gene Therapy for Spinal Muscular Atrophy and New Drug for MS
The European Medicines Agency (EMA) has recommended approval of the gene therapy Itvisma (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy. It also approved Cenrifki (tolebrutinib) for patients with secondary progressive multiple sclerosis.
Two hearts in one Friday: How EMA changed the game for SMA and MS patients
Introduction
On April 23, 2026, the European Medicines Agency (EMA) made a double breakthrough by recommending approval of two drugs, each marking a paradigm shift in its field.
The first — Itvisma (onasemnogene abeparvovec) from Novartis — is a gene therapy for patients with spinal muscular atrophy (SMA) aged two years and older. This expands treatment horizons beyond infancy, where the main successes of gene therapy have been concentrated so far.
The second — Cenrifki (tolebrutinib) from Sanofi — is the first oral drug specifically approved for treating non-relapsing secondary progressive multiple sclerosis (SPMS). This disease has long been considered a "dark zone" of MS therapy, where existing drugs proved ineffective.
Both recommendations were adopted by the Committee for Medicinal Products for Human Use (CHMP) during its April meeting. The European Commission is expected to issue the final approval decision within two months.
This article analyzes the significance of these two decisions — for patients, for medical science, and for the pharmaceutical industry as a whole.
Event Details and Timeline
Itvisma: Gene Therapy for the "Forgotten" Age Group
SMA is a rare inherited neuromuscular disease caused by a mutation in the SMN1 gene, which is responsible for producing a protein essential for motor neuron survival. Without this protein, muscles weaken and atrophy, leading to progressive loss of the ability to move, swallow, and, in severe cases, breathe.
What is Itvisma and how does it work:
Itvisma is an adeno-associated viral vector (AAV9) that delivers a functional copy of the SMN1 gene directly into the cerebrospinal fluid via a single intrathecal injection. Unlike intravenous administration (as with Zolgensma for infants), this method delivers therapy directly to the central nervous system.
The key innovation of Itvisma lies in its age coverage. Zolgensma (the intravenous form of the same gene therapy) is only approved for children under two years old. Older patients — school-age children, adolescents, and adults — have been left without gene therapy options for years.
Clinical trial data:
The EMA recommendation is based on results from the pivotal STEER study (phase 3), which included 126 patients aged 2 to 17 years with 2–4 copies of the SMN2 gene.
| Endpoint | Itvisma (n=75) | Placebo/Sham (n=51) |
|------------|----------------|---------------------|
| Improvement on HFMSE scale | 2.39 points | 0.51 points |
| Statistical significance | p = 0.0074 | — |
The difference of 2.39 points on the Hammersmith Functional Motor Scale Expanded (HFMSE) may seem modest, but as Professor Tim Hagenacker from University Hospital Essen notes, "even a 1-point difference on the HFMSE scale can mean real functional improvements for SMA patients, such as the ability to hold a pen."
Supporting studies STRENGTH (phase 3b) and STRONG (phase 1/2) confirmed clinically meaningful benefit both in treatment-naïve patients and those previously treated with other drugs (nusinersen or risdiplam).
Importantly, Itvisma already received FDA approval in 2025 for patients aged two years and older. The EMA recommendation opens the European Union market for this drug.
Cenrifki: First Drug for Non-Relapsing SPMS
Secondary progressive multiple sclerosis (SPMS) is a stage of the disease that follows relapsing-remitting MS. Unlike the relapsing form, where periods of exacerbations alternate with remissions, SPMS is characterized by steady accumulation of disability without clear relapses.
Patients with SPMS experience progressive fatigue, cognitive impairment, difficulty walking, and other symptoms that significantly reduce quality of life. Until now, no approved drugs specifically targeted this form of the disease.
What is tolebrutinib and how does it work:
Cenrifki (tolebrutinib) is an oral Bruton's tyrosine kinase (BTK) inhibitor that crosses the blood-brain barrier. BTK plays a key role in activating B cells, macrophages, and microglia — immune system cells involved in the inflammatory process in multiple sclerosis.
By targeting these cells both peripherally and directly in the central nervous system, tolebrutinib may slow neuroinflammation and, consequently, disability progression.
Key data from the HERCULES study:
The EMA recommendation is based on results from the HERCULES study (phase 3), which included patients with non-relapsing SPMS (no relapses in the last two years).
Main results:
- 31% reduction in risk of confirmed disability progression compared to placebo
- 38% reduction in the number of new and enlarging T2-hyperintense lesions per year (adjusted mean)
The data were presented at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) conference in 2024 and at the American Academy of Neurology (AAN) annual meeting in 2025, and published in the New England Journal of Medicine.
Safety:
The most common side effects were COVID-19 infections and upper respiratory tract infections. However, the most serious identified risk is drug-induced liver injury, requiring strict monitoring of liver enzyme levels.
Impact and Significance
For SMA Patients: New Hope for Adults and Older Children
Until now, gene therapy for SMA was only available for the youngest patients. Zolgensma, approved for children under two, revolutionized treatment of infantile SMA. But what about those who missed this "window of opportunity"? Older patients could only rely on chronic therapy — nusinersen (Spinraza), requiring repeated intrathecal injections every four months, or risdiplam (Evrysdi), an oral daily medication.
Itvisma offers these patients a single gene therapy — theoretically a lifelong solution. Nicole Gusset, CEO of SMA Europe, emphasizes: "Living with SMA affects all stages of life, including education, employment, and independence. Older children, adolescents, and adults face fewer opportunities for innovative therapy. The positive CHMP opinion for Itvisma is an important step toward closing this gap."
For SPMS Patients: First Target in the "Dark Zone"
Perhaps even more significant is the approval of Cenrifki for SPMS. Multiple sclerosis is one of the most common neurological diseases in young adults. But while there are about a dozen approved disease-modifying therapies for relapsing-remitting MS, very few exist for progressive forms.
As noted in Sanofi's analytical materials, SPMS is a stage where patients experience continuous accumulation of disability, including fatigue, cognitive impairment, and difficulty walking. The ability to slow this process with an oral drug taken once daily is not just a new option — it is the first real tool for this patient category.
For Medical Science and the Industry
Both approvals have significance beyond the specific diseases:
For gene therapy:
Itvisma demonstrates that AAV vector gene therapy can be effective not only in infants but also in older patients with established disease. This is an important precedent for other neuromuscular and neurodegenerative diseases where treatment traditionally starts late.
Also notable is the drug's development path: first an intravenous form for infants (Zolgensma), then an intrathecal form for older patients (Itvisma). Novartis is progressively expanding the therapeutic intervention window.
For MS therapy:
Cenrifki is the first drug in many years approved for a specific subgroup of MS patients, and the first BTK inhibitor to reach this stage. Its success paves the way for other BTK inhibitors in development.
Key Stakeholder Reactions
Novartis welcomed the CHMP decision as a strategic step. Patrick Horber, President of the International Division at Novartis, stated: "Today's positive CHMP opinion is a significant step toward potentially reducing the long-term burden of chronic drug administration for SMA patients in Europe. It reflects our ambition to expand treatment options for a broader SMA patient population."
Sanofi is also actively promoting Cenrifki. The company has submitted approval applications to regulatory authorities worldwide. The EMA decision is the first major regulatory success for this drug.
Patient organizations expressed cautious optimism. SMA Europe, representing SMA patients across Europe, called the decision "an important step toward closing the gap" in treating older age groups.
Forecast and Conclusions
Both drugs now await the final decision from the European Commission, expected within two months. After that, the process of inclusion in national healthcare systems of EU countries will begin — which, in the case of expensive gene therapy, is always a separate challenge.
Key points to watch:
- Pricing and accessibility. Itvisma, like Zolgensma, will likely have a high price. How will European healthcare systems assess the cost-benefit of a single administration for older patients, where the effect is more modest than in infants?
- Safety monitoring. For Cenrifki, given the risk of liver injury, adherence to monitoring protocols in real-world clinical practice will be key.
- Long-term data. For Itvisma, longer follow-up is needed to assess durability of effect in older patients.
- Impact on treatment standards. If Cenrifki confirms its effectiveness in real-world practice, it could become the standard of care for non-relapsing SPMS.
Main takeaway:
April 23, 2026, will go down in history as the day when two of the most challenging neurological diseases received new, fundamentally important tools. Itvisma expands the boundaries of gene therapy beyond infancy. Cenrifki gives hope for the first time to those who have watched their condition steadily deteriorate without intervention.
This is not just pharmaceutical industry news. It is real change in the lives of thousands of patients across Europe. And as Nicole Gusset from SMA Europe noted, even if the effect seems modest, the ability to hold a pen or maintain independence for one more year is not just numbers. It is quality of life.
— Editorial Team
