Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20-23 July 2026

CHMP Recommends Twelve New Medicines for EU Approval

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) concluded its July 2026 meeting, recommending twelve new medicines for marketing authorization in the EU. Additionally, the committee issued positive opinions for eight extensions of therapeutic indications for already authorized medicines. Three new medicines received negative opinions, and several applications for new medicines and indication extensions were withdrawn. Key recommendations for new medicines included treatments for high cholesterol (Lyrokaul, Evlarco, Ubeslo), wet age-related macular degeneration (Susvimo), plaque psoriasis (Icotyde), cholestatic pruritus (Lynavoy), cerebral adrenoleukodystrophy (Nezglyal), and COVID-19 prophylaxis (Zokovea). A new biosimilar, Cavoley, and three generics also received positive opinions.

• The CHMP recommended marketing authorisations for twelve new medicines, comprising nine non-orphan medicines, two orphan medicines, and one biosimilar. Notable recommendations include Lyrokaul, Evlarco, and Ubeslo for hypercholesterolaemia; Susvimo for wet age-related macular degeneration; Icotyde for plaque psoriasis; Lynavoy for cholestatic pruritus; Nezglyal for cerebral adrenoleukodystrophy; and Zokovea for COVID-19 prophylaxis. These approvals aim to address significant unmet medical needs across various therapeutic areas.
• Beyond new medicines, the CHMP also issued positive opinions for eight extensions of therapeutic indications for existing EU-authorised medicines, such as Enhertu, Repatha, and Rinvoq. Furthermore, a new biosimilar, Cavoley (pegfilgrastim), for neutropenia, and three generic medicines—Riociguat Accord, Ruxolitinib Viatris, and Tafamidis Accord—received positive recommendations, expanding treatment options and potentially increasing access for patients.
• The committee adopted negative opinions for three new medicines: KemSu for acute pain in children, Meplyffa for Niemann-Pick disease type C, and Qezzaqar for soft tissue sarcomas. Additionally, applications for Yartemlea and Xervyteg were requested for re-examination after initial negative opinions. Several applications, including Azacitidine Adienne for a new medicine and Dupixent and Imfinzi for indication extensions, were withdrawn, reflecting the rigorous evaluation process.

Addressing Unmet Needs in Primary Hypercholesterolaemia Treatment

Despite the availability of highly effective lipid-lowering therapies, a substantial proportion of patients with primary hypercholesterolaemia fail to achieve guideline-recommended cholesterol targets. This treatment gap stems from several persistent challenges, including issues with patient adherence, variable drug response, and the difficulty of treating severe, genetically-driven forms of the disease.

• Suboptimal Goal Attainment: A significant percentage of patients do not reach their target LDL-C levels, even when on therapy. Data have shown that goal attainment rates can be as low as 30% in treated dyslipidemic patients, with other analyses indicating that approximately 50% to 60% of individuals remain above their recommended cholesterol goals.

• Poor Patient Compliance: Patient adherence to lipid-lowering regimens is a major barrier to effective treatment. For example, one study in a primary care setting found that only 54.1% of patients using rosuvastatin showed adequate compliance. Furthermore, evidence suggests compliance may be even lower among patients with very high baseline LDL-C levels (OR, 0.20; 95% CI, 0.16 to 0.26).

• Limited Efficacy in Severe Disease: Current agents, including high-intensity statins, ezetimibe, and PCSK9 inhibitors, show limited efficacy in patients with homozygous familial hypercholesterolemia (HoFH). The response in these patients is often modest and dependent on their residual LDL receptor function, resulting in markedly elevated LDL-C that necessitates additional therapies such as lipoprotein apheresis.

• Inter-individual Treatment Response: The efficacy of lipid-lowering drugs can vary significantly between individuals due to genetic variations. This pharmacogenomic diversity complicates treatment optimisation and contributes to the variable success rates observed in clinical practice.

• Disparities in Cardiovascular Risk Management: Treatment rates for hypercholesterolemia have been observed to lag behind those for other major cardiovascular risk factors. In some cohorts, treatment rates for a prior diagnosis of hypercholesterolemia (24-39%) were substantially lower than for hypertension (59-90%) or diabetes (52-99%), indicating a need for strategies to reduce this disparity.

EMA Recommends Three New Therapies for Hypercholesterolaemia

Recent clinical trials continue to refine therapeutic strategies for primary hypercholesterolemia, focusing on novel agents and combination therapies to achieve stringent LDL-C targets. Data from studies evaluating fixed-dose combinations, new PCSK9 inhibitors, and bempedoic acid have demonstrated significant lipid-lowering efficacy alongside favorable safety profiles. The following table summarizes key outcomes from several notable recent clinical investigations.

Shifting the Paradigm: The Evolving Hypercholesterolaemia Treatment Landscape

The treatment landscape for primary hypercholesterolaemia has notably evolved from a primary reliance on statin monotherapy towards a more aggressive combination-based approach. While statin prescriptions have increased overall, recent data indicates a marked decrease in statin monotherapy use, driven by compelling evidence for the superior efficacy of combination regimens. A 2024 meta-analysis involving over 24,000 participants demonstrated that moderate-intensity rosuvastatin combined with ezetimibe achieved significantly greater LDL-C reduction than high-intensity rosuvastatin monotherapy (MD -8.06), with a lower incidence of adverse events. This is corroborated by trial data, such as the ROSE-CH study, where fixed-dose rosuvastatin/ezetimibe combinations resulted in superior LDL-C lowering compared to rosuvastatin alone (e.g., -51.48% vs. -42.47% for the 10/10 mg dose). This evidence confirms that combining agents, particularly for high-risk patients, improves LDL-C target attainment and is becoming the new standard of care.

Despite the proven benefits of intensifying treatment with combination therapies, a substantial treatment gap persists, as a large proportion of high-risk patients still fail to achieve guideline-recommended LDL-C goals. For instance, in the German HYDRA-ACS registry, 50% of very high-risk patients did not reach their lipid targets 12 months post-event, even with 100% on lipid-lowering therapy. Similarly, the DISCOVERY study revealed that only 3.3% of ASCVD patients met their 2019 ESC/EAS guideline-recommended LDL-C goals. To address this persistent unmet need, the therapeutic armamentarium is expanding beyond statins and ezetimibe. This includes novel agents such as bempedoic acid, an ATP citrate lyase inhibitor; PCSK9-targeted therapies like the siRNA molecule inclisiran, which offers a twice-yearly dosing advantage; and the ANGPTL3 inhibitor evinacumab, now approved for familial hypercholesterolaemia. For the most refractory cases, specialized treatments like lipoprotein apheresis continue to play a critical role, with registry data showing a 73% reduction in major adverse cardiovascular events during the first two years of use.

EMA Greenlights Novel Ocular Delivery and Oral Biologic

The EMA's latest recommendations signal a pivotal moment for innovation in chronic disease management, particularly in ophthalmology and dermatology. The positive opinion for Susvimo, a ranibizumab Port Delivery System (PDS), represents a significant leap forward in treating neovascular age-related macular degeneration (nAMD), diabetic retinopathy (DR), and diabetic macular edema (DME). This refillable intraocular implant offers continuous anti-VEGF delivery for up to six months, directly addressing the substantial treatment burden and adherence challenges associated with frequent intravitreal injections. For patients, this could mean fewer clinic visits and a more consistent therapeutic effect, potentially preserving vision more effectively in the long term.

However, the path forward for Susvimo is not without its considerations. While the PDS offers clear benefits in reducing injection frequency, studies indicate that its long-term cost-utility ratio may be less favorable compared to conventional injections, a factor that will undoubtedly influence reimbursement decisions across diverse European healthcare systems. Furthermore, clinical evidence suggests that some patients, particularly those with polypoidal choroidal vasculopathy, may be refractory to ranibizumab-based therapies, including Susvimo, highlighting the need for alternative treatments like faricimab that target additional pathways. The ongoing need for longer-term real-world safety data also remains a critical aspect for its widespread adoption.

In parallel, the EMA's positive opinion for Icotyde, an oral peptide IL-23 receptor antagonist for moderate-to-severe plaque psoriasis, introduces a compelling new option in dermatology. As an oral biologic, Icotyde offers a convenient alternative to injectable therapies, potentially improving patient adherence and quality of life. This move underscores the industry's drive towards developing targeted, patient-friendly formulations that can reshape treatment paradigms. Both Susvimo and Icotyde exemplify a broader trend: leveraging advanced drug delivery systems and novel molecular targets to enhance efficacy, reduce treatment burden, and ultimately improve patient outcomes in chronic conditions.