Highlights and Summary

Q3 2026 saw continued expansion of biomarker-gated indications for established precision therapies across the UK and EU, alongside a smaller number of first-in-class approvals for genetically defined rare conditions. Regulators leaned on existing molecular diagnostics, such as HER2, BRCA1/2, PIK3CA and ALK, to extend access to already-authorised medicines into new settings and populations.

In the UK, NICE reversed its earlier rejection of Enhertu for HER2-low advanced breast cancer following methodology changes tied to the UK-US pricing deal, and separately recommended teplizumab as the first disease-modifying therapy for pre-symptomatic type 1 diabetes and donidalorsen and mirdametinib for two genetically-defined rare conditions. The MHRA extended Akeega, an existing BRCA1/2-gated combination, into hormone-sensitive metastatic prostate cancer, and NHS England expanded the National Genomic Test Directory with new liquid-biopsy and haematological-oncology tests.

In the EU, the CHMP broadened biomarker-gated indications for Enhertu twice over; into a new HER2-positive combination and the adjuvant early-breast-cancer setting. The CHMP also broadened the prior-treatment wording of Piqray's indication in PIK3CA-mutated breast cancer, while recommending a new ALK-inhibitor, Gevalka, and an orphan therapy for the rare genetic disorder cerebral adrenoleukodystrophy. The European Commission also approved Datroway for first-line triple-negative breast cancer, and granted conditional authorisation to Vijoice, the first EU-authorised medicine for the rare genetic overgrowth disorders known as PIK3CA-related overgrowth spectrum.

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United Kingdom

NICE Recommends Enhertu for HER2-Low Advanced Breast Cancer  | Approval

NICE issued final draft guidance on 17 September 2026 recommending trastuzumab deruxtecan (Enhertu, AstraZeneca/Daiichi Sankyo) for adults with HER2-low metastatic or unresectable breast cancer whose disease has progressed after chemotherapy, reversing a 2024 rejection. The decision follows the UK-US pricing deal's increase to NICE's cost-effectiveness threshold and a new quality-of-life assessment method published in August 2026, and eligibility is confirmed by HER2 immunohistochemistry testing. Around 1,000 people are expected to be eligible, with interim funding available immediately under the Cancer Drugs Fund ahead of routine commissioning 90 days after the guideline is published.

MHRA Grants Marketing Authorisation for Akeega for BRCA1/2-Mutated Metastatic Prostate Cancer | Approval

The MHRA authorised Akeega (niraparib and abiraterone acetate, Johnson & Johnson) in combination with prednisone or prednisolone and androgen deprivation therapy for adults with metastatic hormone-sensitive prostate cancer (mHSPC) harbouring BRCA1/2 mutations. The company describes it as the UK's first licensed precision-medicine combination for BRCA1/2-mutated mHSPC; the same combination was already licensed in the UK for BRCA1/2-mutated metastatic castration-resistant prostate cancer. The company says it will work with health technology assessment bodies to support potential future NHS access.

NICE Recommends Tzield for Stage 2 Type 1 Diabetes  | Approval

NICE published final guidance on 9 July 2026 recommending teplizumab (Tzield, Sanofi) for people aged 8 and over with stage 2 type 1 diabetes, an early, pre-symptomatic phase identified through islet autoantibody testing. Clinical trial data showed the immunotherapy delayed onset of symptomatic (stage 3) disease by an average of nearly three years, making it the first disease-modifying treatment NICE has recommended for type 1 diabetes and the first in Europe via a health technology appraisal. NICE estimates around 1,100 people will be eligible in the first year, with access dependent on identifying pre-symptomatic patients through autoantibody screening.

NICE Recommends Ezmekly for NF1-Associated Plexiform Neurofibromas  | Approval

NICE recommended mirdametinib (Ezmekly, Merck Serono) for symptomatic, inoperable plexiform neurofibromas in children and young people aged 2 to 17 with neurofibromatosis type 1 (NF1), a genetic condition caused by mutations in the NF1 gene. It is the first drug treatment NICE has recommended for children as young as two. Around 200 patients are expected to benefit.

NHS England Updates the 2026/27 National Genomic Test Directory  | Policy

NHS England published an update to the 2026/27 National Genomic Test Directory on 16 July 2026, adding a circulating tumour DNA (ctDNA) liquid-biopsy test and further three multi-target NGS panels for cancer of unknown primary origin. The directory sets out the genomic tests commissioned by NHS England and delivered through Genomic Laboratory Hubs. Further changes included expanded POLE testing for endometrial cancers and a high-sensitivity JAK2 V617F test for myeloproliferative neoplasms.

MHRA Rare Disease Therapies Regulatory Framework Consultation Closes  | Policy

The MHRA's consultation on its draft Rare Disease Therapies Regulatory Framework closed on 30 July 2026. The framework proposes a new Investigational Marketing Authorisation pathway, combining clinical trial approval with a staged route to full marketing authorisation for conditions affecting typically 1 in 50,000 people or fewer in the UK. The MHRA is still analysing results and has not yet published its consultation response or confirmed a date for implementing the proposed framework.

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European Union 

European Commission Grants Conditional Approval to Vijoice for PIK3CA-Related Overgrowth Spectrum  | Approval

Vijoice (alpelisib, Novartis) became the first medicine authorised in the EU for PIK3CA-related overgrowth spectrum (PROS) when the European Commission granted conditional marketing authorisation on 15 July 2026, following a CHMP positive opinion on 21 May. The authorisation covers adults and children aged 2 and over with severe or life-threatening manifestations of these rare genetic conditions who require systemic therapy. Until now, these conditions were managed with supportive care, including surgery. The approval rests on a retrospective review of 57 patients treated through a compassionate-use programme, in which 12 of the 32 patients assessed at 24 weeks (37.5%) had a reduction of at least 20% in the size of 1 to 3 lesions, and the company must run a confirmatory study.

CHMP Recommends Broadened Indication for Piqray in PIK3CA-Mutated Breast Cancer  | Approval

On 23 July 2026, the CHMP adopted a positive opinion extending the indication for alpelisib (Piqray, Novartis) by changing its prior-treatment requirement from disease progression after endocrine therapy alone to progression after an endocrine-based regimen. This may include patients previously treated with endocrine therapy alongside another drug. Piqray remains indicated only in combination with fulvestrant, for postmenopausal women and men with HR-positive, HER2-negative, locally advanced or metastatic breast cancer with a PIK3CA  

CHMP Recommends Nezglyal for Cerebral Adrenoleukodystrophy  | Approval

Leriglitazone (Nezglyal, Minoryx Therapeutics) was recommended for marketing authorisation under exceptional circumstances by the CHMP on 23 July 2026 for the treatment of cerebral adrenoleukodystrophy, in male patients aged 2 to 12 with gadolinium-negative brain lesions. The condition is a rare genetic disorder affecting the brain that typically presents in childhood. The medicine holds orphan designation, reflecting the rarity of the condition and the limited existing treatment options. The European Commission granted marketing authorisation on 21 September 2026, describing it as the first pharmacological treatment approved for the condition in the EU.

European Commission Approves Datroway for First-Line Metastatic Triple-Negative Breast Cancer  | Approval

The European Commission approved Datroway (datopotamab deruxtecan, AstraZeneca/Daiichi Sankyo) on 29 July 2026 as monotherapy for the first-line treatment of adults with unresectable or metastatic triple-negative breast cancer who are not candidates for PD-1/PD-L1 inhibitor therapy. The approval follows a CHMP positive opinion based on the Phase III TROPION-Breast02 trial, which showed an overall survival benefit over chemotherapy, and gives datopotamab deruxtecan its second breast cancer indication in the EU alongside its existing HR-positive, HER2-negative use.

CHMP Recommends Gevalka for ALK-Positive Advanced NSCLC  | Approval

A new ALK inhibitor, ensartinib (Gevalka, SFL Pharmaceuticals), received a CHMP positive opinion on 17 September 2026 recommending marketing authorisation for adults with anaplastic lymphoma kinase (ALK)-positive advanced non-small-cell lung cancer who have not previously received an ALK inhibitor or who have previous received crizotinib.

CHMP Recommends Adjuvant Extension for Enhertu in Resected HER2-Positive Early Breast Cancer  | Approval

On 17 September 2026, the CHMP recommended extending Enhertu’s EU indication to adjuvant monotherapy for adults with resected HER2-positive breast cancer who have residual invasive disease after neoadjuvant taxane-based and HER2-targeted treatment. This followed a separate July CHMP opinion recommending Enhertu plus pertuzumab as first-line treatment for unresectable or metastatic HER2-positive breast cancer.