Precision Medicine in Europe: Policy and Approvals Q1 Update

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Highlights and Summary

Q1 2026 followed the structural reforms announced in Q4 2025, with several policy milestones coming into force and multiple regulatory approvals across the UK and EU. In the UK, the MHRA/NICE Aligned Pathway launched on 1 April, with 27 companies enrolled as early adopters and initial guidance expected in June. Updated UK clinical trial regulations will take effect in late April, introducing a fast-track pathway for lower-risk studies and specific guidance for trials involving companion diagnostics. In the EU, the CHMP recommended several targeted therapies during the first quarter. These included an EGFR-targeted lung cancer therapy, an adenoviral gene therapy for high-risk bladder cancer, a pediatric BRAF-targeted brain cancer therapy, and treatments for rare genetic diseases such as thymidine kinase 2 deficiency and APDS. Additionally, the EU's MDR/IVDR simplification proposal entered its formal legislative feedback phase, and the EMA and FDA jointly issued AI principles for medicine development.

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United Kingdom

NICE Approves First Biologic for Targeted COPD Treatment | Approval

NICE recommended dupilumab (Dupixent) as an add-on maintenance treatment for patients with uncontrolled COPD characterized by raised blood eosinophils (Type 2 inflammation). This is the first biologic reimbursed on the NHS for COPD in the UK, introducing a biomarker-stratified approach for a disease typically managed with broad-spectrum inhalers.

MHRA Sets Out New Phase of Reforms for Clinical Trials | Policy

The MHRA published its updated clinical trials framework (entering into force on 28 April 2026), outlining a 14-day assessment route for Phase 1 trials and a dual-track modification system. Alongside this, the MHRA issued a separate, specific guidance document detailing how In Vitro Diagnostic Devices (IVDs) are assessed within the clinical trial framework, which applies to the development of biomarker-driven therapies and companion diagnostics.

NICE Approves Talazoparib for Metastatic Prostate Cancer | Approval

NICE approved talazoparib, a PARP inhibitor, in combination with enzalutamide for men with metastatic castration-resistant prostate cancer. This provides a targeted treatment option for patients ineligible for chemotherapy or other standard treatments. Clinical trial data indicated a median survival increase of nearly nine months for patients receiving the drug with enzalutamide compared to standard care.

MHRA/NICE Aligned Pathway Formally Launches | Policy

The Aligned Pathway officially launched on 1 April 2026, allowing NICE's cost-effectiveness appraisal to run concurrently with the MHRA's licensing review to coordinate the publication of safety, efficacy, and value decisions. Currently, 27 pharmaceutical companies are signed up as early adopters, with initial guidance from the pathway expected in June 2026. The program includes a new Integrated Scientific Advice (ISA) service for coordinated regulatory and HTA guidance.

MHRA Approves Deuruxolitinib for Alopecia Areata | Approval

The MHRA granted approval for Sun Pharma's JAK inhibitor deuruxolitinib (Leqselvi) for the treatment of severe alopecia areata in adults. The therapy will undergo NICE appraisal to assess availability through the NHS. This therapy joins the current pipeline of immunology approvals targeting specific autoimmune pathways.

MHRA Launches Consultation on Indefinite CE-Mark Recognition for Medical Devices | Policy

The MHRA opened a consultation on proposals to indefinitely recognise EU MDR and IVDR-compliant devices in Great Britain. A stated aim of the consultation is to maintain access to in vitro companion diagnostics utilized in precision oncology pathways and reduce the risk of supply interruptions.

NICE Approves Imlunestrant (Inluriyo) for ESR1-Mutated Breast Cancer | Approval

The MHRA granted marketing authorisation for imlunestrant tosylate (Inluriyo, Eli Lilly) for adults with ER-positive, HER2-negative, advanced or metastatic breast cancer harbouring ESR1 gene mutations who have progressed on prior endocrine therapy. As an oral SERD, it targets the acquired resistance mutation prevalent in this setting.

DHSC Publishes National Cancer Plan Integrating Precision Genomic Therapies | Policy

In February 2026, the UK Department of Health and Social Care published the National Cancer Plan for England, which includes an updated framework for oncology clinical trials. Supported by a £32.3 million funding allocation, the policy outlines plans to integrate genomics-driven precision medicine into NHS care, establishes national clinical leads for rare cancers, and notes expansions for programs such as the Minderoo Precision Brain Tumour Programme.

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European Union 

CHMP Recommends Treatment for Rare TK2d | Approval

The CHMP recommended Kygevvi (doxecitine/doxribtimine) for the treatment of genetically confirmed Thymidine Kinase 2 deficiency (TK2d), a mitochondrial disease affecting an estimated one in a million people. The therapy was evaluated under the EMA’s PRIority MEdicines (PRIME) scheme.

European Commission Approves Aumseqa for EGFR-Mutated NSCLC | Approval

February 2026, the European Commission granted marketing authorisation for Aumseqa (following a December 2025 CHMP opinion) as a first-line monotherapy for adults with advanced non-small cell lung cancer (NSCLC) whose tumors harbor specific EGFR exon 19 deletions or exon 21 (L858R) substitution mutations, as well as for EGFR T790M mutation-positive NSCLC.

Conditional Approval for Pediatric Low-Grade Glioma Therapy | Approval

The CHMP recommended a conditional marketing authorisation for Ojemda (tovorafenib) from Ipsen Pharma for pediatric low-grade glioma in patients aged 6 months and older. As a targeted BRAF inhibitor, this once-weekly oral therapy is indicated for a patient population where existing chemotherapy options are associated with adverse effects.

EMA and FDA Issue Joint AI Principles for Medicines Development | Policy

The EMA and the US FDA jointly issued 10 principles for AI practice across the medicine lifecycle. The document provides an internationally harmonised framework for AI use in drug development and regulatory review, detailing guidelines applicable to the development of AI-driven biomarker tools.

CHMP Recommends Mavorixafor (Xolremdi) for WHIM Syndrome | Approval

The CHMP adopted a positive opinion for mavorixafor (Xolremdi) for patients aged 12 and older with WHIM syndrome, an inherited immunodeficiency caused by CXCR4 receptor dysfunction. Mavorixafor is an oral once-daily CXCR4 antagonist directed at the genetic mechanism that traps white blood cells in the bone marrow.

CHMP Recommends Adstiladrin Gene Therapy for High-Risk Bladder Cancer | Approval

In March 2026, the CHMP recommended conditional marketing authorisation for Adstiladrin (nadofaragene firadenovec), an adenoviral vector-based gene therapy. It is indicated for adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC).

CHMP Recommends Tarlatamab (Imdylltra) for Relapsed Small Cell Lung Cancer | Approval

The CHMP recommended Imdylltra (tarlatamab) as monotherapy for adults with extensive-stage small cell lung cancer whose disease relapsed during or after initial platinum-based chemotherapy. In the DeLLphi-304 study, the DLL3-targeted bispecific T-cell engager demonstrated a median overall survival of 13.6 months compared to 8.3 months with standard chemotherapy.

CHMP Recommends Joenja (leniolisib) for Rare Genetic APDS Syndrome | Approval

The CHMP recommended Joenja under exceptional circumstances for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS). APDS is a genetic immunodeficiency disease, and this approval is the first targeted therapy for this specific genetic pathway in the EU.

CHMP Recommends Scemblix for T315I-Mutated Chronic Myeloid Leukaemia | Approval

The CHMP recommended an extension of indication for Scemblix (asciminib) to include the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML) harboring the T315I mutation who are resistant to, intolerant to, or ineligible for ponatinib. This approval highlights the clinical application of mutational testing to guide therapy choices in hematological malignancies.