Precision Medicine in Europe: Policy and Approvals Q2 Update

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Highlights and Summary

Q2 2026 was a transformative period for precision medicine in Europe, marked by landmark structural reforms in the UK and a continued push toward mutation-guided treatments in the EU. These developments collectively underscore an accelerating reliance on molecular diagnostics to define clinical pathways and improve access to innovative therapies.

The UK implemented major regulatory reforms. NICE raised its cost-effectiveness threshold to £25,000–£35,000 per QALY, projected to support 3–5 additional precision therapies annually. The MHRA initiated consultations on a Rare Disease Therapies Regulatory Framework and modernized gene therapy definitions. Clinically, NICE accelerated targeted oncology adoption, notably with the first folate receptor-alpha ADC for ovarian cancer and IDH1/2 inhibitor for glioma.

The EU accelerated histology-independent and mutation-guided prescribing, prioritizing liquid biopsy and molecular diagnostics. The CHMP issued a landmark positive opinion for the first tumour-agnostic HER2-directed therapy (trastuzumab deruxtecan). Progress also included the first authorized therapy for PIK3CA-related overgrowth spectrum (PROS) and endorsement of ESR1-mutation-guided camizestrant.

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United Kingdom

NICE Cost-Effectiveness Threshold Rises to £25,000–£35,000 per QALY  |  Policy

From April 2026, NICE applied a new cost-effectiveness threshold range of £25,000–£35,000 per quality-adjusted life year, the first increase since NICE was established in 1999. The change, introduced through secondary legislation following the December 2025 UK–US Economic Prosperity Deal, applies immediately to all new and ongoing technology appraisals. NICE estimates the revised threshold will enable 3–5 additional medicines or indications to be recommended per year, with anticipated benefits concentrated in precision oncology, gene and cell therapies, and rare disease treatments where marginal cost-effectiveness had previously constrained access.

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MHRA Publishes Draft Rare Disease Therapies Regulatory Framework  |  Policy | Rare Disease

On 21 May 2026, the MHRA launched a public consultation on a proposed Rare Disease Therapies Regulatory Framework introducing a new Investigational Marketing Authorisation (IMA) pathway. The IMA would combine clinical trial approval with a staged, progressive route to full marketing authorisation for conditions with a UK prevalence of typically 1 in 50,000 or fewer. This will cover over 3.5 million people in the UK of whom fewer than 5% currently have approved treatment. The framework explicitly supports adaptive trial designs, surrogate endpoints, real-world evidence, and computational modelling, and is designed to compress development timelines currently averaging 10–12 years.  

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MHRA Opens Consultation on Updated Gene Therapy Medicinal Product Definitions  |  Policy | Gene / Cell Therapy

The MHRA, acting jointly with the Department of Health in Northern Ireland, launched a consultation on 11 May 2026 to update the legal definition of gene therapy medicinal products (GTMPs) in the UK. The current definitions, established in 2007, require GTMPs to be biological in origin which has left synthetically manufactured products and CRISPR-based genome editing technologies outside the regulatory framework. The proposed revisions would reclassify products based on mechanism of action rather than origin, ensuring synthetic nucleic acids and sequence-specific genome editing technologies are unambiguously captured as GTMPs.  

NICE Recommends Mirvetuximab Soravtansine (Elahere) for Folate Receptor-Alpha-Positive Platinum-Resistant Ovarian Cancer  |  Approval | Companion Diagnostic

NICE recommended mirvetuximab soravtansine (Elahere, AbbVie) on 24 June 2026 for adults with folate receptor-alpha (FRα)-positive, platinum-resistant, high-grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received 1–3 prior lines of systemic treatment. The antibody-drug conjugate targets the biomarker FRα, confirmed by a companion diagnostic test, marking the first new NHS treatment for platinum-resistant ovarian cancer in over 20 years. Approximately 270 patients are expected to be eligible in the first year, rising to 420 by year three as access to the required FRα diagnostic broadens.  

NICE Recommends Vorasidenib (Voranigo) for IDH1/2-Mutant Low-Grade Glioma via Aligned Pathway  |  Approval | Oncology

NICE recommended vorasidenib (Voranigo, Servier) on 29 April 2026 for patients aged 12 and older with grade 2 IDH1- or IDH2-mutant astrocytoma or oligodendroglioma following surgery, where immediate chemotherapy or radiotherapy is not required. Vorasidenib is the first targeted therapy for IDH-mutant low-grade glioma, a primary brain tumour predominantly affecting adults under 50; in the phase 3 INDIGO trial it reduced the risk of progression or death by 61% compared with placebo. Notably, the recommendation was issued ahead of MHRA marketing authorisation via the newly operational MHRA/NICE Aligned Pathway, with NHS access secured through interim Cancer Drugs Fund funding for approximately 300 eligible patients.

NICE Recommends Encorafenib with Binimetinib for BRAF V600E-Mutant Advanced Non-Small-Cell Lung Cancer  |  Approval | Oncology

NICE recommended encorafenib with binimetinib (Braftovi/Mektovi, Pierre Fabre) on 6 May 2026 for adults with advanced non-small-cell lung cancer whose tumours harbour a BRAF V600E mutation. This is the first BRAF/MEK inhibitor combination recommended by NICE for lung cancer, extending a biomarker-stratified treatment approach previously established in BRAF V600E-mutant melanoma to a molecularly defined lung cancer subgroup identified by tumour genetic testing. The approval follows confirmation of BRAF V600E mutation status as a prerequisite for treatment eligibility.

NICE Recommends Zanidatamab (Ziihera) for HER2-Positive Advanced Biliary Tract Cancer  |  Approval | Companion Diagnostic

NICE recommended zanidatamab (Ziihera, Jazz Pharmaceuticals) on 7 May 2026 for adults with unresectable locally advanced or metastatic HER2-positive (IHC 3+) biliary tract cancer previously treated with at least one prior line of systemic therapy. Zanidatamab is a dual HER2-targeted bispecific antibody that simultaneously binds two distinct extracellular domains of HER2, and its use requires confirmation of HER2 IHC 3+ status by diagnostic testing. This is the first HER2-targeted therapy recommended by NICE for biliary tract cancer, a rare and aggressive cancer group that includes cholangiocarcinoma and gallbladder cancer, embedding molecular diagnostics into a chemo-only treatment pathway.

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European Union 

CHMP Recommends Etcamah (Camizestrant) for ESR1-Mutant ER-Positive HER2-Negative Metastatic Breast Cancer  |  Approval | Companion Diagnostic

The CHMP adopted a positive opinion for camizestrant (Etcamah, AstraZeneca) in May 2026 for the treatment of adults with locally advanced or metastatic ER-positive, HER2-negative breast cancer harbouring an ESR1 mutation, in combination with a CDK4/6 inhibitor, in patients without disease progression during first-line endocrine-based therapy. Camizestrant is a next-generation oral selective oestrogen receptor degrader (SERD) specifically indicated for the ESR1-mutated subpopulation, whose mutation status is typically identified by liquid biopsy circulating tumour DNA testing which marks a significant step toward ctDNA-guided treatment selection in advanced breast cancer in the EU.

CHMP Recommends Enhertu (Trastuzumab Deruxtecan) for HER2-Positive Solid Tumours — First Tumour-Agnostic HER2 Indication in the EU  |  Approval | Companion Diagnostic

The CHMP adopted a positive opinion in May 2026 for trastuzumab deruxtecan (Enhertu, AstraZeneca/Daiichi Sankyo) as monotherapy for adults with unresectable or metastatic HER2-positive (IHC 3+) solid tumours who have received prior systemic treatment and have no satisfactory alternative options. If converted to a full European Commission authorisation, this would be the first HER2-directed therapy and first antibody-drug conjugate to hold a tumour-agnostic indication in the EU, requiring only IHC 3+ HER2 confirmation regardless of primary tumour site.  

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CHMP Recommends Vijoice (Alpelisib) for PIK3CA-Related Overgrowth Spectrum  |  Approval | Rare Disease

The CHMP recommended conditional marketing authorisation for alpelisib (Vijoice, Novartis) in May 2026 for adult and paediatric patients aged 2 years and older with severe or life-threatening manifestations of PIK3CA-related overgrowth spectrum (PROS) requiring systemic therapy. PROS is a group of rare genetic conditions caused by somatic gain-of-function PIK3CA mutations, characterised by uncontrolled tissue overgrowth affecting skin, bone, blood vessels, and brain. This is the first authorised therapy for PROS in the EU; diagnosis requires molecular confirmation of a PIK3CA variant, and the conditional authorisation reflects approval in a rare setting where conventional randomised trial evidence is not feasible.

CHMP Recommends Itvisma (Onasemnogene Abeparvovec) for Spinal Muscular Atrophy  |  Approval | Gene / Cell Therapy

The CHMP recommended marketing authorization for onasemnogene abeparvovec (Itvisma, Novartis) in April 2026 for the treatment of 5q spinal muscular atrophy, the second authorisation for this AAV9-based gene therapy in the EU, increasing the available supply base for a treatment indicated for patients with SMN1 gene deletions or mutations. SMA is caused by biallelic loss-of-function of SMN1, and onasemnogene abeparvovec delivers a functional copy of the SMN1 gene via a single intravenous infusion; increasing the number of authorized products is expected to strengthen supply resilience and potentially improve pricing competition for this high-cost genetic therapy.

CHMP Recommends Redemplo (Plozasiran) for Familial Chylomicronaemia Syndrome  |  Approval | Rare Disease

The CHMP adopted a positive opinion for plozasiran (Redemplo, Arrowhead Pharmaceuticals) in April 2026 as an orphan medicine for adults with familial chylomicronaemia syndrome (FCS). FCS is a rare autosomal recessive disorder caused by mutations in LPL, APOC2, APOA5, LMF1, or GPIHBP1, resulting in severe hypertriglyceridaemia and recurrent pancreatitis; no previously authorized medicine existed in the EU for this condition. Plozasiran is a GalNAc-conjugated siRNA that silences APOC3, a key negative regulator of triglyceride clearance, and is indicated exclusively for patients with genetically confirmed FCS. It represents the first EU approval for an RNA interference therapy in a genetically defined lipid disorder.

CHMP Extends Braftovi (Encorafenib) to Additional BRAF-Mutant Solid Tumour Indication  |  Approval | Oncology

The CHMP adopted a positive opinion in May 2026 recommending an extension of indication for encorafenib (Braftovi, Pierre Fabre) to cover an additional BRAF V600-mutant solid tumour type. The extension broadens application of BRAF inhibition, which is already authorised in the EU for BRAF V600-mutant melanoma and colorectal cancer, into a further molecularly defined patient population, reinforcing the tumour-agnostic utility of BRAF mutation testing as a prerequisite for treatment selection across cancer types.

CHMP Extends Venclyxto (Venetoclax) Indication in AML and Additional Haematological Setting  |  Approval | Oncology

The CHMP adopted two positive opinions in April 2026 recommending extensions of indication for venetoclax (Venclyxto, AbbVie) in acute myeloid leukaemia and an additional haematological malignancy setting. Venetoclax is a BCL-2 inhibitor that targets anti-apoptotic protein overexpression characteristic of certain haematological cancers; expanded authorisation broadens access to this molecularly-targeted agent across additional patient populations where BCL-2 pathway dependence drives disease biology.

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