Precision Medicine in Europe: Policy and Approvals Q4 Update
Highlights and Summary
Q4 2025 European updates were characterized by structural policy changes and targeted therapeutic approvals across the UK and EU. Key policy developments included the UK government's decision to increase NICE's cost-effectiveness threshold by 25% starting April 2026 and the launch of the MHRA/NICE "Aligned Pathway" to reduce access delays, while the EU reached a political agreement on its comprehensive Pharma Package and proposed simplifications to MDR/IVDR regulations. Regulatory bodies cleared several first-in-class therapies, including the world’s first twice-yearly biologic for asthma (depemokimab) in the UK and the first treatment for non-CF bronchiectasis (brensocatib) in the EU. Additional authorizations focused on biomarker-driven precision oncology, targeting EGFR-mutated lung cancer, ESR1-mutated breast cancer, and IL-15 responsive bladder cancer, as well as preventative interventions for Type 1 Diabetes and HIV.
United Kingdom
NICE Increases Cost-Effectiveness Threshold | Policy
The UK government announced a landmark 25% increase to NICE's cost-effectiveness thresholds, effective April 2026. The standard threshold will rise from £20,000-£30,000 to £25,000-£35,000 per quality-adjusted life year (QALY), marking the first major increase in over two decades. This policy change is expected to enable NICE to recommend an additional 3-5 new medicines per year, with particular benefits anticipated for breakthrough cancer treatments, rare disease therapies, and innovative precision medicine approaches. The change also includes updates to NICE's quality of life assessment methodology using a new EQ-5D-5L value set.
MHRA Approves First Twice-Yearly Biologic for Asthma | Approval
The MHRA approved depemokimab (Exdensur) on December 15, 2025, marking the world's first regulatory approval for this ultra-long-acting biologic with twice-yearly dosing. The therapy targets IL-5 for the treatment of severe eosinophilic asthma and severe chronic rhinosinusitis with nasal polyps in patients with type 2 inflammation. This represents a significant advance in precision respiratory medicine, as treatment can be tailored to patients with elevated eosinophil biomarkers, with the extended dosing interval potentially improving adherence and reducing treatment burden.
Up to 6,000 patients to benefit from NICE-approved life-extending prostate cancer drug | Approval
People living with metastatic hormone-sensitive prostate cancer in England will gain access to a new treatment combination from October 2025, including an ADT therapy and Darolutamide (Nubeqa). This combination is as effective as another combination treatment already recommended by NICE, apalutamide plus ADT, but has fewer side effects and potential interactions with other drugs, offering clinicians and patients more flexibility in treatment choice.
NICE and MHRA Launch Accelerated 'Aligned Pathway' | Policy
The MHRA and NICE announced the early launch of the "Aligned Pathway," a joint initiative to streamline regulatory approval and health technology assessment. This process removes the typical 90-day delay between authorisation and NICE guidance, accelerating NHS patient access to qualifying new medicines by 3 to 6 months.
Abiraterone Approved for High-Risk Metastatic Prostate Cancer | Approval
NICE approved abiraterone, including generic versions, in combination with androgen deprivation therapy (ADT) and prednisolone or prednisone, for men with high-risk hormone-sensitive metastatic prostate cancer. This new recommendation, a significant shift from previous guidance, was driven by the availability of lower-cost generic alternatives, making it value for money for the NHS.
MHRA Signals Major Overhaul for Rare Disease Therapies | Policy
The MHRA announced a major reform of the UK’s regulatory framework to accelerate the development and approval of rare disease therapies. By addressing barriers like small patient cohorts and complex evidence generation, the new "rulebook" aims to move the UK toward a global leadership position in precision medicine. Key proposals include a single-step approval for clinical trials and marketing authorisations based on limited but compelling evidence, alongside data-sharing initiatives to support advanced, highly personalised modalities like CRISPR and mRNA.
NICE Approves Cemiplimab Combination for Advanced Lung Cancer | Approval
NICE recommended cemiplimab (Libtayo) in combination with platinum-based chemotherapy for adults with untreated advanced non-small-cell lung cancer (NSCLC). This approval is significant for precision oncology as it targets tumors expressing PD-L1, and adds another immunotherapy + chemotherapy option in first‑line NSCLC.
European Union
EU Pharma Package Political Agreement Reached | Policy
The EU Parliament and Council reached a landmark political agreement on pharmaceutical legislation reform, representing the most significant update in over 20 years. Key provisions include: maintaining eight years of regulatory data exclusivity with potential for up to 11 years total market exclusivity for medicines addressing unmet needs; nine years of orphan market exclusivity with 11 years for "breakthrough" orphan products in therapeutic areas with no existing treatment; shortened EMA review timelines from 210 to 180 days; expanded Bolar exemption to accelerate generic/biosimilar entry; and new supply obligations requiring marketing authorization holders to launch products within three years of Member State requests or face exclusivity reductions. The agreement aims to balance innovation incentives with patient access across the EU.
CHMP Recommends Aumolertinib for EGFR-Mutated NSCLC | Approval
The CHMP adopted a positive opinion in December 2025 for aumolertinib (Aumseqa), a third-generation EGFR tyrosine kinase inhibitor for EGFR-mutated non-small cell lung cancer. This precision oncology therapy targets patients with specific EGFR-sensitizing mutations, representing a personalized approach for advanced lung cancer treatment. Clinical data from the ACROSS 2 trial demonstrated improved progression-free survival when combined with chemotherapy in patients with concomitant tumor suppressor gene mutations.
CHMP Recommends Anktiva for BCG-Unresponsive Bladder Cancer | Approval
The CHMP recommended conditional marketing authorization for Anktiva (nogapendekin alfa inbakicept), an IL-15 receptor agonist, in combination with BCG for adults with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ. This immunotherapy activates NK cells, T cells, and memory T cells, offering a precision immunotherapy approach as an alternative to radical cystectomy. In the pivotal single-arm trial, 71% of patients achieved complete response with responses lasting approximately 27 months on average.
Aficamten Receives Positive CHMP Opinion for Hypertrophic Cardiomyopathy | Approval
The CHMP adopted a positive opinion for Myqorzo (aficamten) in December 2025 for adults with obstructive hypertrophic cardiomyopathy. This oral selective cardiac myosin inhibitor targets the molecular basis of the disease, reducing hypercontractility through direct myosin binding. The SEQUOIA-HCM trial demonstrated significant improvement in peak oxygen uptake (1.8 ml/kg/min increase vs. 0.0 ml/kg/min with placebo) with consistent benefits across all patient subgroups, representing a precision cardiology breakthrough.
EU Proposes Simplification of MDR/IVDR | Policy
On December 16, 2025, the European Commission published proposals to simplify the Medical Devices Regulation and In Vitro Diagnostics Regulation, including streamlined provisions for companion diagnostics. The proposal removes mandatory recertification cycles, harmonizes documentation processes, and eliminates notification requirements for certain IVDR performance studies on companion diagnostics using leftover specimens. These changes aim to reduce regulatory burden while supporting precision medicine through improved access to diagnostic technologies.
Industry Calls for Urgent MDR/IVDR Regulatory Reform | Policy
Major industry groups, including MedTech Europe, urged the European Commission to take urgent action to simplify and streamline the Medical Device Regulation (MDR) and IVDR. The call aims to address current regulatory bottlenecks and administrative burdens, specifically by promoting the elimination of the mandatory five-year recertification cycle for devices and advocating for the harmonization of documentation and processes.
CHMP Recommends Wayrilz for Immune Thrombocytopenia | Approval
CHMP adopted a positive opinion recommending marketing authorisation for Wayrilz (rilzabrutinib) for the treatment of immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. Wayrilz will be the first BTK inhibitor for ITP in the EU, targeting the root cause of the disease through multi-immune modulation.
First Treatment Recommended for Non-Cystic Fibrosis Bronchiectasis | Approval
CHMP recommended granting marketing authorisation for Brinsupri (brensocatib). This marks the first approved treatment for non-cystic fibrosis bronchiectasis, a chronic, progressive lung disease characterized by damaged airways and severe pulmonary dysfunction. The medicine was supported through the EMA’s PRIority MEdicines (PRIME) scheme, which targets areas of high unmet medical need.
CHMP Backs Targeted Treatment for ESR1-Mutated Breast Cancer | Approval
The CHMP recommended Inluriyo (imlunestrant, Eli Lilly) for adults with locally advanced or metastatic breast cancer harboring an ESR1 mutation. As an oral selective estrogen receptor degrader (SERD), it offers a personalized treatment for patients who progressed on prior endocrine therapy. Clinical data showed that Inluriyo monotherapy significantly improves progression-free survival (PFS) compared to standard treatments like fulvestrant or exemestane.
Teplizumab Recommended to Delay the Onset of Type 1 Diabetes | Approval
Teizeild (teplizumab) received a positive recommendation from the CHMP to delay the onset of stage 3 Type 1 diabetes (T1D) in adults and children (8+ years with stage 2 T1D). This immunotherapy targets the T-cells responsible for destroying insulin-producing beta cells, representing a major shift toward "preventative" precision medicine in diabetes.
