by Clauda Santos Silva, EAN Scientific Committee member
As part of the EAN Scientific Committee’s liaison with the European Medicines Agency (EMA), we reviewed the EMA Annual Report 2025, published in June 2026, to identify developments of particular relevance to neurology. Below, we summarise the key regulatory milestones, pharmacovigilance updates and innovation initiatives highlighted in the report that are most pertinent to the neurological community. Please see the full version of the EMA annual report here: Homepage | EMA annual report 2025
New Medicines and Indications
In 2025, the EMA recommended 104 medicines for marketing authorisation, namely:
- Attrogy (diflunisal): the EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of a marketing authorisation for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy.
- Austedo (deutetrabenazine) for tardive dyskinesia in adults: the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a marketing authorisation for the treatment of moderate-to-severe tardive dyskinesia in adults.
- Duvyzat (givinostat) for Duchenne muscular dystrophy: the CHMP recommended a conditional marketing authorisation for the treatment of ambulant patients aged six years and older with Duchenne muscular dystrophy (DMD) receiving concomitant corticosteroid therapy. Givinostat is a histone deacetylase (HDAC) inhibitor that modulates pathological HDAC activity in dystrophic muscle, thereby reducing inflammation and fibrosis.
- Imaavy (nipocalimab) for generalised myasthenia gravis: the CHMP adopted a positive opinion recommending a marketing authorisation for the treatment of generalised myasthenia gravis (gMG) in adults and adolescents (≥12 years) who are anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) antibody positive. Nipocalimab is a monoclonal antibody targeting the neonatal Fc receptor (FcRn), thereby reducing circulating pathogenic IgG antibodies.
- Kisunla (donanemab) for Alzheimer’s disease: following a re-examination procedure, the CHMP adopted a positive opinion recommending a marketing authorisation for the treatment of early symptomatic Alzheimer’s disease in adults who are apolipoprotein E ε4 (ApoE ε4) non-carriers or heterozygotes.
- Riulvy (tegomil fumarate) for multiple sclerosis: the CHMP adopted a positive opinion recommending a marketing authorisation for the treatment of relapsing-remitting multiple sclerosis (RRMS) in adults and adolescents aged 13 years and older. Tegomil fumarate is an oral immunomodulatory agent whose active metabolite, monomethyl fumarate, exerts anti-inflammatory and immunomodulatory effects.
Negative CHMP Opinions Relevant to Neurology
The CHMP adopted negative opinions for the following medicines relevant to neurology:
- Blarcamesine (Anavex): intended for the treatment of Alzheimer’s disease.
- Elevidys (delandistrogene moxeparvovec): intended for the treatment of Duchenne muscular dystrophy (DMD).
- Nurzigma (pridopidine): intended for the treatment of Huntington’s disease.
Changes in Orphan Designation Status
Orphan designation is reviewed by the EMA’s Committee for Orphan Medicinal Products (COMP) at the time of marketing authorisation to determine whether a medicine remains eligible for orphan status and the associated ten-year market exclusivity. Among the 104 medicines recommended for marketing authorisation in 2025, 16 retained their orphan designation. Five medicines lost orphan status before marketing authorisation, while remaining eligible for marketing authorisation. Among the medicines relevant to neurology, Attrogy (diflunisal) lost its orphan designation prior to approval.
Pharmacovigilance and Safety Updates
- Clozapine: following a review of long-term safety data, the EMA concluded that the risk of severe neutropenia decreases substantially over time. Consequently, routine haematological monitoring requirements were simplified: only the absolute neutrophil count (ANC) is now required, while white blood cell count monitoring is no longer mandatory. These changes are expected to reduce the monitoring burden while maintaining patient safety.
- Semaglutide: product information was updated to include non-arteritic anterior ischaemic optic neuropathy (NAION) as a very rare adverse reaction. Clinicians are advised to discontinue semaglutide if NAION is confirmed, following evidence suggesting a possible association with this sight-threatening optic neuropathy.
- Tegretol (Carbamazepine): the EMA restricted the use of the 100mg/5mL oral suspension in neonates because the formulation contains propylene glycol at concentrations exceeding recommended safety thresholds for this age group.
Scientific and Regulatory Innovation
Beyond individual medicines, several broader regulatory initiatives are expected to influence the future development and evaluation of neurological therapies.
The EMA continued to expand the use of real-world evidence (RWE) through DARWIN EU, supporting regulatory decision-making using healthcare data from approximately 180 million patients across Europe.
Artificial intelligence became increasingly integrated into medicines regulation, marked by the publication of the first EMA AI Observatory Report and the development of joint EMA–FDA guiding principles for the use of AI throughout the medicine lifecycle, including medicine development, clinical trials, manufacturing and pharmacovigilance.
The EMA also continued to modernise the European regulatory framework through accelerated assessment pathways and strengthened support for innovative medicines via the PRIority Medicines (PRIME) scheme, which provides enhanced regulatory support for medicines addressing unmet medical needs.
Finally, the European Shortages Monitoring Platform (ESMP) became fully operational in 2025. This digital platform centralises and automates medicines shortage reporting by National Competent Authorities (NCAs) and Marketing Authorisation Holders (MAHs), enabling earlier detection, improved coordination and more effective management of medicine shortages across the European Union.
Conclusion
The EMA’s activities in 2025 highlight continued progress in the regulation of innovative therapies for neurological diseases. Positive opinions for new treatments in hereditary ATTR amyloidosis, myasthenia gravis, Duchenne muscular dystrophy, Alzheimer’s disease, multiple sclerosis and tardive dyskinesia illustrate the rapidly evolving therapeutic landscape. At the same time, important pharmacovigilance updates, including changes to clozapine monitoring and new safety recommendations for semaglutide and carbamazepine, reinforce the Agency’s commitment to patient safety.
Beyond individual medicines, initiatives such as DARWIN EU, the integration of artificial intelligence into medicines regulation, the PRIority Medicines (PRIME) scheme and the implementation of the European Shortages Monitoring Platform (ESMP) demonstrate the EMA’s ongoing efforts to modernise the European regulatory framework and facilitate timely access to innovative therapies.
The EAN Scientific Committee will continue to monitor these developments and keep the neurological community informed of regulatory advances that may influence clinical practice and future therapeutic innovation across Europe.
