Tag: Regulatory Affairs

Non-human primate hand via pickpik.com (CC0)

EMA public consultation on non‑human primate use in safety testing

EMA seeks stakeholder input on new reflection paper to reduce and replace non‑human primate use in medicines safety testing through 3Rs approaches.
Molecular therapy

Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation

Outside traditional drug development pathways, clinicians and scientists face the challenge of systematically evaluating whether individual patients with severe ultrarare diseases might be eligible for and potentially benefit from individualized mutation-specific RNA therapies.
European Medicines Agency (EMA)

First Italian-made gene therapy for Wiskott-Aldrich Syndrome

On 13 November in Milan, EMA’s expert committee backs Italy’s first home‑grown gene therapy for people in Europe living with Wiskott–Aldrich Syndrome.
World Orphan Drug Congress USA 2026

World Orphan Drug Congress USA 2026

More than 280 speakers from industry, regulators, patient organisations, investors and healthcare providers, and aims to showcase new science, technologies and policies that can accelerate orphan drug development and improve access to therapies for people living with rare conditions.
European Medicines Agency (EMA)

EMA public consultation on phage therapy medicinal products

EMA invites comments on new quality guideline for bacteriophage‑based medicines to support safe phage therapy development in Europe.
drug discovery today

Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval

With over 300 million people affected by rare diseases, timely access to effective therapies is critical. Despite strong alignment in regulatory expectations, patients in many regions face years of delays highlighting the need for improved international coordination.
Medicine Safety studies visual

EMA publishes new guidelines for non-interventional studies

New EMA ICH M14 guidelines sets common international standards for non‑interventional real‑world data studies to strengthen medicine safety assessment.
EDERA WODC & GAM - Preview- JVDPhotography-1-min

ERDERA at WODC Europe 2025: wrap up and next steps

Sharing patient‑centred methods, early diagnostics, and data‑driven trial innovation to accelerate rare disease research across Europe.
Training online

Information Webinar – ERDERA Joint Transnational Call 2026

This information webinar on the 2026 Joint Transnational Call for Proposals is scheduled for 16 December 2025, 15:00–17:00 CET, focusing on resolving unsolved rare genetic and non‑genetic diseases.
Ai Exhibition

EMA opens public consultations on patient experience data and use of external controls

EMA invites feedback to strengthen how the EU generates and uses evidence that reflects what matters to patients, including in rare diseases
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