Euro Roundup: EMA seeks feedback on plans to draft myasthenia gravis clinical trial guideline

The European Medicines Agency (EMA) has set out plans to create guidance on myasthenia gravis (MG) clinical trials in a draft concept paper.

MG is a chronic neuromuscular junction disorder driven by an autoimmune response. Patients with the rare condition have fluctuating weakness and fatigability of skeletal muscles. Amgen, Argenx, Johnson & Johnson, and UCB have won approval for generalized MG drugs in the European Union in recent years. Yet with R&D continuing and subpopulations still untreated, EMA has identified a need for guidance.

EMA said MG is a rapidly evolving field and “the dynamic landscape justifies the need for an up-to-date and comprehensive regulatory guidance.” The agency also noted challenges to developing treatments for children with MG and patients who are seronegative for AChR antibodies.

That thinking has informed EMA’s plans to provide a general strategy covering the main goals of MG care, symptomatic treatments, and disease-modifying therapies. The agency plans to address the choice of patient population, outlining the characteristics of subpopulations such as people with ocular MG and groups who are positive or negative for certain antibodies implicated in the disease.

Another section of the guidance will cover the design of exploratory and confirmatory trials. EMA plans to discuss outcome assessment tools, explaining the use of methods of assessing clinical severity, the functional impact of interventions, and quality of life. For confirmatory trials, the agency intends to look at efficacy endpoints, duration, and alignment to the estimand framework.

Studies in children, seniors, and people with refractory MG are also on EMA’s agenda. While the agency plans to address trials in underrepresented populations, other neuromuscular junction disorders such as Lambert-Eaton myasthenic syndrome and congenital myasthenic syndrome are outside of the scope of the planned guidance.

EMA is seeking feedback on the draft concept paper until 30 August. The agency aims to release draft guidance by the fourth quarter of 2026.

Concept Paper

Notified bodies set evidence expectations for reagent devices used in combinations

Notified body association Team-NB has provided harmonized expectations for showing the safety and performance of reagent devices used in combination with other devices or equipment.

Team-NB published the position paper to clarify key considerations for ensuring compliance with the In Vitro Diagnostic Regulations (IVDR), which defines general safety and performance requirements and technical documentation for combinations using reagent devices. The paper covers reagents used with one specific instrument, with different but specified instruments, and with general instruments.

“While the regulation does not define explicit rules for validating combinatorial claims, notified bodies expect clear, robust, and reproducible evidence supporting these claims,” Team-NB said. “Manufacturers must prioritize comprehensive validation, risk mitigation, and transparency in labeling and [instructions for use] to align with these expectations.”

Team-NB has provided guidance on how manufacturers should show the compatibility, safety, and performance of combined devices, including the connection system where applicable. Citing IVDR, the association said manufacturers applying for unrestricted combinatorial use must identify and mitigate risks and show device combinations do not introduce new hazards.

The paper also addresses the information provided to users of combination devices. Notified bodies expect applicants to include details of products that reagent devices can be used with, information about known restrictions, and enough detail to ensure correct user integration of the combined system.

Position Paper

EDQM reiterates CEP submission rules as 25% of applicants fail to provide reports

The European Directorate for the Quality of Medicines and HealthCare (EDQM) has reminded companies about recent changes to the Certification of Suitability (CEP) submission process.

EDQM introduced an automated process on 1 November. To fully benefit from automation, applicants must submit an electronic common technical document (eCTD) validation report. EDQM has established requirements for eCTD validation reports that companies must adhere to for the new system to work.

However, EDQM said around 25% of CEP submissions continue to lack the requested eCTD validation report, leading to processing delays. Starting 1 April, the directorate will reject applications that lack the required report.

EDQM shared the warning alongside the requirements for eCTD validation reports and its updated guide for using the Common European Submission Portal. Applicants must use an appropriate eCTD validation tool to check reports against currently applicable EU criteria and correct any fail errors before submitting the document to EDQM.

The title of the report and location of the document in the submission’s file structure are covered by requirements that companies must meet. EDQM also dictates the report’s format, with only PDF, RTF, HTML, or MHTML submissions being accepted.

EDQM Notice

Swissmedic tracks 12% reduction in turnaround time for human drug approvals

The Swiss Agency for Therapeutic Products (Swissmedic) approved human medicinal products with new active substances in a median of 392 calendar days in 2025, down 12% on the prior year.

Swissmedic concluded 45 new authorization applications for human medicinal products with new active substances in 2025, authorizing 40 of the products. The applicants withdrew the other five submissions. Fast-track procedures applied to 50% of the approvals, compared to 39% of the 46 drugs authorized in 2024.

Median fast-track review times were longer in 2025, 338 calendar days, than 2024, 327 calendar days. However, the median review time for all filings fell as Swissmedic cut turnaround times for its standard procedure from 477 to 458 calendar days and the proportion of fast-track assessments increased.

One-quarter of the new drugs were approved as part of Swissmedic’s work with the Access Consortium and Project Orbis, procedures through which the regulator collaborates with other regulatory agencies. The number of approvals via the Access Consortium’s work-sharing initiative increased from five to eight.

Swissmedic authorized 109 additional indications for human medicinal products in 2025, an increase of 54% over the previous year. The agency processed 19% of the additional indications through the Access Consortium and Project Orbis.

Swissmedic Notice

EMA recommends withdrawal of anti-worm medicine over brain disease risk

EMA has recommended the anti-worm medicine levamisole is pulled from the European Union market because the benefits no longer outweigh the risks.

The agency’s Pharmacovigilance Risk Assessment Committee reached the conclusion after assessing links between levamisole and leukoencephalopathy. EMA confirmed leukoencephalopathy is a rare side effect of levamisole. Leukoencephalopathy is a debilitating and life-threatening condition driven by damage to the brain’s white matter, which allows efficient communication between different parts of the organ.

EMA found leukoencephalopathy can occur after one levamisole dose and that symptoms may develop up to several months after treatment. The condition is particularly harmful if untreated. EMA found no ways to reduce the risk or groups of people who may be at higher or lower risk.

Other medicines are authorized in the EU for treating parasitic worm infections. Levamisole is used to treat mild parasitic worm infections. Considering the medical need and severity of leukoencephalopathy, EMA ruled medicines containing levamisole should be withdrawn from the market.

EMA Notice, More

Other News:

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has restricted use of Valneva’s Chikungunya vaccine Ixchiq in response to very rare fatal reactions and other adverse events. Ixchiq is no longer indicated for people aged over 60 years and is contraindicated in anyone with hypertension, cardiovascular disease, diabetes mellitus, or chronic kidney disease. MHRA Notice

Euro Roundup: EMA seeks feedback on plans to draft myasthenia gravis clinical trial guideline

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