Three terminated appraisals in a single month is an unusually high number, and includes the CAR-T cell therapy Breyanzi in follicular lymphoma, a setting in which the treatment had previously received a positive recommendation in diffuse large B-cell lymphoma.

Positive Recommendations

Kerendia recommended for heart failure

Finerenone (Kerendia), a non-steroidal mineralocorticoid receptor antagonist developed by Bayer HealthCare, received a positive NICE recommendation on 5 August 2026 for heart failure. Finerenone is already approved in the EU and UK for chronic kidney disease associated with type 2 diabetes, and this recommendation extends its application to heart failure, a condition in which fluid overload and neurohormonal activation play a central role in disease progression. Unlike older steroidal mineralocorticoid receptor antagonists such as spironolactone and eplerenone, finerenone has been designed to offer greater receptor selectivity, potentially reducing the risk of hyperkalaemia and other off-target effects while maintaining cardiovascular and renal protection.

Dawnzera recommended for hereditary angio-oedema

Donidalorsen (Dawnzera), an oral plasma kallikrein inhibitor developed by Ionis Pharmaceuticals and Otsuka, received a positive NICE recommendation on 19 August 2026 for the prevention of attacks in hereditary angio-oedema (HAE). HAE is a rare and potentially life-threatening genetic condition characterised by episodes of severe swelling affecting the skin, gastrointestinal tract, and upper airways; laryngeal attacks in particular can be fatal without prompt treatment. Donidalorsen is administered orally on a prophylactic basis, offering a significant practical advantage over existing parenteral prophylactic therapies. The recommendation represents a meaningful step forward for patients seeking a convenient, long-term preventive option.

Calquence combinations recommended for mantle cell lymphoma and CLL

Two separate NICE recommendations were issued on 19 August 2026 for acalabrutinib (Calquence)-based combination regimens in haematological malignancies, both submitted by AstraZeneca.

For mantle cell lymphoma, acalabrutinib in combination with bendamustine and rituximab (Calquence, Treanda, and Rituxan) was recommended for previously untreated patients. Mantle cell lymphoma is an aggressive B-cell non-Hodgkin lymphoma with a tendency to relapse; adding BTK inhibition to the established bendamustine-rituximab backbone offers a chemotherapy-intensified and targeted approach that has demonstrated improved outcomes in clinical trials.

For chronic lymphocytic leukaemia and small lymphocytic lymphoma (CLL/SLL), acalabrutinib in combination with venetoclax, with or without obinutuzumab (Calquence, Venclexta, and optionally Gazyva), was recommended. This all-oral, chemotherapy-free triplet targets three distinct mechanisms: BTK inhibition, BCL-2 inhibition, and CD20 targeting. CLL is the most common adult leukaemia in the Western world, and the combination approach represents a shift towards deeper, potentially time-limited treatment that may reduce the likelihood of resistance compared with continuous monotherapy.

Odactra recommended for dust mite allergic rhinitis

The sublingual allergy immunotherapy tablet for house dust mite allergy (Odactra), developed by ALK-Abello, received a positive NICE recommendation on 26 August 2026 for allergic rhinitis caused by house dust mite sensitisation. Allergen immunotherapy is the only treatment modality that addresses the underlying allergic mechanism rather than simply controlling symptoms, and sublingual tablet formulations offer a more convenient alternative to subcutaneous immunotherapy injections administered in a clinical setting. The recommendation is significant for the large number of patients with persistent, poorly controlled dust mite allergic rhinitis who do not achieve adequate symptom relief with antihistamines and intranasal corticosteroids.

Updated Recommendation

Itulazax receives updated recommendation for birch pollen allergy

Betula verrucosa sublingual tablet (Itulazax), a birch pollen allergen immunotherapy developed by ALK-Abello and partnered with Torii Pharma and Shionogi, received a positive second appraisal recommendation on 25 August 2026 for allergic rhinoconjunctivitis caused by birch pollen. Itulazax is administered daily as a sublingual tablet and works by gradually desensitising the immune system to birch pollen allergens over a course of treatment typically lasting several years. The updated recommendation follows the initial positive appraisal and likely reflects revised evidence or access arrangements, and sits alongside the same cycle’s first recommendation for Odactra, underscoring growing NICE support for sublingual immunotherapy as a treatment modality across different allergen types.

Terminated Appraisals

Brinsupri appraisal terminated for non-CF bronchiectasis

The appraisal of brensocatib (Brinsupri), a dipeptidyl peptidase 1 (DPP1) inhibitor developed by Insmed, was terminated on 25 August 2026 for non-cystic fibrosis bronchiectasis. Bronchiectasis is a chronic lung condition characterised by irreversible airway dilation and recurrent infections driven by neutrophilic inflammation; DPP1 inhibition reduces the activation of neutrophil serine proteases that contribute to lung damage and exacerbations. Brensocatib had been considered a significant advance in a condition with very few approved pharmacological therapies, making its termination a notable setback for patients and clinicians in this field. The reasons for termination were not specified in the available data.

Wayrilz appraisal terminated for immune thrombocytopenic purpura

The appraisal of rilzabrutinib (Wayrilz), an oral BTK inhibitor developed by Principia Biopharma and Sanofi, was terminated on 20 August 2026 for immune thrombocytopenic purpura (ITP). ITP is an autoimmune bleeding disorder characterised by low platelet counts resulting from immune-mediated destruction of platelets and suppression of platelet production. Rilzabrutinib targets the B-cell receptor signalling pathway and had been under investigation as an oral option for patients with persistent or chronic ITP who have failed or are intolerant of existing therapies. The termination means this treatment will not be available via the NICE appraisal route at this time.

Breyanzi appraisal terminated for follicular lymphoma

The appraisal of lisocabtagene maraleucel (Breyanzi), a CD19-directed CAR-T cell therapy developed by Bristol Myers Squibb, was terminated on 20 August 2026 for relapsed or refractory follicular lymphoma. This termination is particularly notable given that Breyanzi received a positive NICE recommendation for diffuse large B-cell lymphoma earlier in 2026, and is approved in other markets for follicular lymphoma. Follicular lymphoma is an indolent B-cell malignancy that, despite its relatively slow progression, frequently relapses and ultimately requires multiple lines of therapy. CAR-T cell treatment has shown strong efficacy in this setting, and the termination — likely reflecting cost-effectiveness or access agreement issues — will be disappointing for patients and their clinicians.

Decisions referenced carry NICE Positive 1, NICE Positive 2, or Terminated status, issued between 5–26 August 2026. This article is based on data from the NICE Technology Appraisal Programme for England and Wales.

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Medicare covers GLP-1s for Type 2 diabetes but is prohibited from paying for the drugs to treat obesity. However, in March 2024 Medicare clarified that health plans administering the Part D benefit could cover Wegovy to reduce the risk of heart complications in beneficiaries with obesity and heart disease after the U.S. Food and Drug Administration (FDA) expanded approval of Wegovy for that purpose.

In the six months following this increased access, the researchers found:

• Fills of Wegovy increased 136% compared to the previous six months across all coverage sources—Medicare, Medicaid, private insurance and cash-pay—and grew significantly faster than other GLP-1s sharing the same active ingredient, semaglutide. The new cardiovascular indication drove an estimated 593,000 additional Wegovy prescriptions.

• Medicare fills of Wegovy increased 598%, far outpacing growth in other coverage sources. This increase signals pent-up demand among Medicare beneficiaries and shows how FDA approvals of GLP-1s for indications beyond obesity can expand access.
• However, six months after the Medicare announcement, less than 1% of the estimated 3.6 million beneficiaries thought to be eligible based on their weight and cardiovascular health had filled a Wegovy prescription. Several factors could explain the low uptake: Part D plans were likely hesitant to add the costly drugs to their formularies, patients could have faced barriers like high-cost sharing or other restrictions on use, and providers may have had limited awareness of the changes.

Medicare is now aiming to broadly expand access to GLP-1s for weight loss through a temporary pilot program, known as Medicare GLP-1 Bridge, launched this month. However, the pilot, largely funded by the federal government, is set to expire after 2027, and health insurers have balked at a longer-term program that would shift costs onto them.

Regardless of the Bridge program’s future, Medicare will continue to allow coverage of weight-loss drugs for other FDA-approved indications beyond obesity. In addition to cardiovascular disease, Wegovy has also been approved for the serious liver disease MASH, while other GLP-1s have been approved for conditions including obstructive sleep apnea and chronic kidney disease. Researchers continue to study GLP-1s, which were first approved for Type 2 diabetes, for a range of other conditions.

“Although our study shows that federal policy change can help increase access to GLP-1s for heart disease, we still have a long way to go to ensure that patients can access and afford these medications as their clinical indications keep expanding,” said first author Christopher Scannell, a nonresident scholar at the USC Schaeffer Institute and senior research associate at the USC Mann School of Pharmacy and Pharmaceutical Sciences.

About the study

The study was published July 9 in the American Journal of Managed Care. Other authors are John Romley, Rebecca Myerson, Dana Goldman and Dima Mazen Qato.

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Oncology and dermatology feature prominently in this cycle, with notable recommendations for novel antibody-drug conjugates, a new JAK inhibitor indication, and an innovative combination approach in multiple myeloma. The cycle also includes three vaccine recommendations addressing chikungunya, meningococcal disease, influenza, and orthopoxvirus infections.

Oncology

Datroway recommended for triple-negative breast cancer

Datopotamab deruxtecan (Datroway), an antibody-drug conjugate developed by Daiichi Sankyo and AstraZeneca, has received a positive CHMP recommendation for triple-negative breast cancer (TNBC). TNBC remains one of the most challenging breast cancer subtypes to treat, given its lack of hormone receptor and HER2 expression. Datroway targets TROP2, a protein overexpressed in many epithelial tumours, and delivers a cytotoxic payload selectively to cancer cells, offering a more targeted approach than conventional chemotherapy.

Tecvayli and Darzalex Faspro combination recommended for multiple myeloma

Johnson & Johnson has received a CHMP recommendation for the combination of teclistamab and daratumumab (Tecvayli and Darzalex Faspro) for multiple myeloma. Teclistamab is a bispecific T-cell engager targeting BCMA and CD3, while daratumumab is a well-established anti-CD38 monoclonal antibody. Combining the two mechanisms in a chemotherapy-free regimen represents a significant advance in the treatment of relapsed or refractory multiple myeloma, a condition that typically requires repeated lines of therapy over time.

Jaypirca recommended for chronic lymphocytic leukaemia

Pirtobrutinib (Jaypirca), a non-covalent BTK inhibitor developed by Loxo Oncology at Lilly and Eli Lilly, has been recommended for chronic lymphocytic leukaemia and small lymphocytic lymphoma (CLL/SLL). Unlike covalent BTK inhibitors such as ibrutinib, pirtobrutinib is designed to remain active in patients who have developed resistance mutations, offering a treatment option for a population with limited alternatives following prior BTK inhibitor therapy.

Denosumab biosimilar recommended for bone complications in cancer

Denosumab Ascend, a biosimilar of denosumab developed by Ascend GmbH, has received a CHMP recommendation for the prevention of skeletal-related events in patients with bone metastases and for giant cell tumour of bone. The availability of a biosimilar version of this widely used bone-targeted agent is expected to improve patient access and reduce costs for health systems across the EU.

Pegfilgrastim biosimilar recommended for neutropenia

Nylaspeg, a biosimilar of pegfilgrastim submitted by Qilu Pharmaceuticals, has received a positive recommendation for chemotherapy-induced neutropenia. Pegfilgrastim is routinely used to stimulate white blood cell production and reduce the risk of febrile neutropenia in cancer patients receiving myelosuppressive chemotherapy. The biosimilar recommendation supports continued access to this supportive care treatment at competitive cost.

Dermatology & Immunology

Rinvoq recommended for vitiligo and alopecia areata

AbbVie’s upadacitinib (Rinvoq), a selective JAK1 inhibitor, has received two separate CHMP recommendations expanding its indications into dermatology: one for vitiligo and one for alopecia areata. Vitiligo is a chronic autoimmune condition causing depigmentation of the skin, while alopecia areata results in patchy or total hair loss driven by immune-mediated attack on hair follicles. Both conditions have historically lacked effective systemic treatments, and these recommendations follow growing clinical evidence supporting JAK inhibition as a mechanism of action in autoimmune skin diseases. Rinvoq is already approved for several inflammatory conditions including rheumatoid arthritis, psoriatic arthritis, and atopic dermatitis.

Opzelura recommended for atopic dermatitis

Ruxolitinib cream (Opzelura), developed by Incyte Corporation and Maruho, has received a CHMP recommendation for atopic dermatitis (eczema). Opzelura is a topical JAK1/JAK2 inhibitor that provides targeted anti-inflammatory activity at the site of application, offering an alternative to topical corticosteroids for patients with mild to moderate disease. The recommendation addresses a highly prevalent condition that places significant quality of life burden on patients, particularly those with disease affecting visible areas of skin.

Neurology & Paediatrics

Daybue recommended for Rett syndrome

Trofinetide (Daybue), developed by Acadia Pharmaceuticals, has received a CHMP recommendation for Rett syndrome, a rare and severe neurodevelopmental disorder caused by mutations in the MECP2 gene, which predominantly affects girls. The condition leads to progressive loss of motor and communication skills and has no approved disease-modifying treatments in the EU. Daybue is a synthetic analogue of the naturally occurring tripeptide GPE (glycine-proline-glutamate) and represents the first pharmacological treatment specifically developed for this condition to receive a positive EU recommendation.

Crexont recommended for Parkinson’s disease

A prolonged-release combination of carbidopa and levodopa (Crexont), submitted by Amneal Pharmaceuticals and Zambon, has been recommended for Parkinson’s disease. Levodopa remains the cornerstone of Parkinson’s pharmacotherapy, but standard formulations are associated with motor fluctuations and wearing-off effects as the disease progresses. The extended-release formulation aims to provide more consistent plasma drug levels throughout the day, potentially reducing the frequency and severity of motor complications in patients with advanced disease.

Stelara recommended for paediatric ulcerative colitis

Ustekinumab (Stelara), developed by Johnson & Johnson, has received a CHMP recommendation for paediatric ulcerative colitis. Ustekinumab is an interleukin-12 and interleukin-23 inhibitor already established in adult inflammatory bowel disease and psoriasis. This paediatric recommendation addresses a significant unmet need in younger patients with moderate to severe ulcerative colitis who have an inadequate response to conventional therapy.

Infectious Diseases & Vaccines

Ixchiq recommended for chikungunya fever

Ixchiq, a live-attenuated chikungunya vaccine developed by Valneva SE and Instituto Butantan, has received a positive CHMP recommendation. Chikungunya is a mosquito-borne viral illness characterised by fever and severe joint pain, with outbreaks increasingly reported in southern Europe due to the northward spread of the Aedes albopictus mosquito. The approval of a preventive vaccine represents an important public health tool as the geographical range of the disease continues to expand.

MenQuadfi recommended for meningococcal disease

MenQuadfi, a conjugate vaccine targeting meningococcal serogroups A, C, W, and Y, developed by Sanofi, has received a CHMP recommendation. Meningococcal disease can cause life-threatening bacterial meningitis and septicaemia, particularly in infants, young children, and adolescents. The conjugate formulation is designed to produce a robust and long-lasting immune response and is suitable for use in paediatric populations.

Imvanex recommended for monkeypox and orthopoxvirus infections

Bavarian Nordic’s Imvanex, a modified vaccinia Ankara-based vaccine, has received a CHMP recommendation for protection against monkeypox (mpox) and other orthopoxvirus infections. Imvanex was already authorised for smallpox prevention in the EU and was used extensively during the 2022 mpox outbreak. This recommendation formalises its indication for mpox and broader orthopoxvirus prophylaxis, reflecting the continued public health relevance of the product.

Aujemflu recommended for influenza

Aujemflu, an adjuvanted trivalent influenza vaccine developed by Seqirus/CSL, has received a CHMP recommendation for the prevention of influenza. The adjuvanted formulation is designed to enhance immunogenicity, particularly in older adults and immunocompromised populations where vaccine responses may be suboptimal. The recommendation adds to the existing portfolio of seasonal influenza vaccines available to EU member states.

Symtuza and Prezcobix recommended for HIV

Two HIV antiretroviral regimens have received CHMP recommendations. Symtuza — a single-tablet combination of darunavir, emtricitabine, cobicistat, and tenofovir alafenamide (TAF), developed by Johnson & Johnson and Gilead Sciences — offers a complete one-tablet-once-daily regimen for HIV-1 infection. Separately, Prezcobix — a combination of darunavir and cobicistat developed by Janssen Therapeutics — provides a boosted protease inhibitor backbone for use in combination with other antiretrovirals. Both recommendations likely reflect updated indications or label revisions for these established regimens.

Cardiovascular & Diabetes

Leqvio recommended for hypercholesterolaemia

Inclisiran (Leqvio), a small interfering RNA (siRNA) therapy developed by Novartis, has received a CHMP recommendation for hypercholesterolaemia. Inclisiran works by inhibiting the production of PCSK9, a protein that reduces the liver’s ability to clear LDL cholesterol from the blood. Administered just twice yearly by subcutaneous injection, inclisiran offers a highly differentiated dosing schedule compared with daily oral statins or monthly PCSK9 inhibitor antibodies, which may improve adherence in patients requiring long-term lipid-lowering therapy.

Onswik recommended for type 2 diabetes

Insulin efsitora alfa (Onswik), a once-weekly basal insulin analogue developed by Eli Lilly, has received a positive CHMP recommendation for type 2 diabetes. The treatment represents a significant step forward in insulin therapy, offering a weekly injection as an alternative to the daily or twice-daily basal insulin regimens that many patients find burdensome. A reduced injection frequency is expected to support better adherence and patient quality of life, particularly in the early stages of insulin initiation.

All nineteen decisions listed carry CHMP Recommended status. CHMP recommendations are subject to final marketing authorisation by the European Commission. This article is based on data from the European Medicines Agency CHMP opinion cycle.

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The pathway aligns regulatory review by the Medicines and Healthcare products Regulatory Agency (MHRA) with the National Institute for Health and Care Excellence (NICE) health technology appraisal process, so that decisions are reached simultaneously.

But Huseyin Naci at the London School of Economics and Political Science notes that these two bodies require different forms of evidence. The MHRA focuses on a product’s clinical effectiveness and safety, while NICE assesses its comparative effectiveness and long term value for money against current NHS treatments.

Rather than aligning evidentiary standards, the pathway runs two independent processes in parallel with the aim of synchronising their conclusions, he explains, offering companies earlier revenue and a longer rebate-free window, while benefits for patients and the NHS are less clear.

He acknowledges that earlier access to effective new drugs can matter for patients with significant unmet needs, but says, unlike the patient-focused Innovative Licensing and Access Pathway (ILAP), the new pathway expedites all new medicines regardless of added therapeutic benefit.

The pathway will also impose timelines to ensure that MHRA and NICE decisions are reached simultaneously. Yet Naci points out that fixed drug evaluation deadlines have historically been linked to higher rates of adverse events, and fast-tracked medicines are more prone to post-marketing safety issues than those approved through standard routes.

A further problem is that NICE committees may be asked to assess products on evidence not yet fully vetted by the MHRA, compounding the uncertainty under which they already operate, he writes.

Earlier adoption of expensive, potentially low-value medicines will also extend the period over which they divert NHS resources from more cost-effective interventions, a problem exacerbated by the 2025 US-UK trade agreement committing NICE to a 25% higher cost-effectiveness threshold, he adds.

Naci urges the government to ground medicines access policy in patient need and population health, calling for clarification on the assumptions underpinning the pathway and assessment of its anticipated benefits and harms for all stakeholders, not just industry.

NICE’s focus on synchronisation of decisions risks overlooking whether the NHS will be adequately protected from the earlier adoption of medicines that prove harmful, ineffective, or poor value, he concludes.

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For years, drug companies and industry allies have argued that the U.S. gets faster and wider access because its government moves quicker than foreign regulators, but the new analysis suggests the U.S. advantage in drug access is driven less by faster government review and more by when companies apply for review and the type of drugs they submit.

The study, published in Health Affairs, looked at every new prescription drug approved between 2014 and 2018 in the U.S., and Europe, and then tracked submission delays and review times for these products across regulators in Canada, Japan and Australia through the end of 2022. The analysis assessed the speed of the review process and the timing of submissions for approval, with results broken down by drug characteristics, including therapeutic value of the drugs.

Specifically, the researchers explored whether different patterns in submission and review times emerged for drugs that offered little added medical benefit over drugs that were already on the market.

“Some commentators have argued that foreign regulators take too long to review drugs and should do more to ensure timely access to new therapies, often pointing to limited availability of new cancer therapies in Europe and other rich markets relative to what’s on the market in the United States, as evidence that regulatory red tape is getting in the way of timely patient access,” said lead author Irene Papanicolas, a professor of health services, policy and practice at the Brown University School of Public Health. “Where we’re coming at this from is saying that broader availability of new medicines is generally a great thing — we want patients to get access to new meds — but not all new medications are equally important from a medical standpoint.”

In fact, what stood out most was how companies handled drugs that provide little therapeutic advantage over existing treatments, which the authors referred to as “low-value” drugs in their analysis. The researchers found these drugs were typically submitted to U.S. regulators months or even years before companies sought approval in other high-income countries, giving Americans earlier and wider access to expensive drugs that may not significantly improve patient outcomes.

The findings likely reflect a mix of business incentives and policy choices, according to the research team which along with Papanicolas and other Brown co-authors Olivier Wouters and Tania Sawaya also includes health policy experts from Vanderbilt University and the London School of Economics and Political Science.

The U.S. is the world’s largest drug market, and manufacturers can generally set prices freely when a drug launches. In contrast, many other countries evaluate how much a new drug improves health compared with existing treatments and use that information to negotiate prices or limit coverage, said Wouters, an associate professor in the Department of Health Services, Policy, and Practice.

“There are many drugs that enter the U.S. market that frankly aren’t much better than what’s already available,” Wouters said. “Companies generally seem to submit these lower-value products for approval  earlier in the United States than in other markets. This may reflect the fact that governments in other countries tend to drive a tougher bargain than U.S. payers, which could influence companies’ decisions about where and when to seek approval.”

The study also showed that drugs offering clear medical benefits over existing treatments tended to reach most high-income countries at roughly the same time. This is because drugmakers typically submit those products for approval simultaneously across the high-income countries the researchers looked at. The Food and Drug Administration was only slightly faster than its counterparts abroad in approving the drugs by a few weeks or a month on average, the researchers said.

“Historically, yes, the U.S. gets more new drugs and gets them faster than other countries but a lot of what is driving this pattern aren’t the drugs that have this meaningful therapeutic gain for patients,” Papanicolas said. “Everybody’s getting those important new drugs quickly.”

Overall, the study helps add nuance to the question of why the U.S. spends far more on prescription drugs than other high-income countries without consistently better health outcomes. It also paints a more complicated picture as policymakers debate proposals such as the “most favored nation” approach, to bring down drug spending, which proposes linking U.S. drug prices to those paid in peer countries.

“It’s not clear how this is going to work,” Papanicolas said. “How will the US authorities handle products that haven’t yet been marketed abroad? Will the policy affect where and when companies decide to submit drugs abroad? No one really knows yet.”

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Researchers found that between 2012 and 2020, 178,000 Medicare beneficiaries received early access to cancer drugs through the FDA’s accelerated approval pathway. While the pathway aims to give patients faster access to promising treatments, fewer than half of these drugs were later shown to help people live longer.

Key Findings:

• Limited Survival Gains: Only 45% of Medicare beneficiaries who received accelerated approval drugs were treated with drugs that ultimately improved survival.
• Life-Years Added: Early access to accelerated approval drugs delivered an estimated 76,000 extra years of life for Medicare beneficiaries.
• Concentrated Benefits: Just three drugs—for melanoma and lung cancer—accounted for over two-thirds of all extra life-years gained.
• High Cost: Medicare spent over $20 billion more on early access to cancer drugs than on alternative treatments, averaging $263,000 per additional year of life. Costs ranged from $26,000 per life-year for melanoma drugs to $4.5 million per life-year for some breast cancer drugs.

Huseyin Naci, Associate Professor of Health Policy at the London School of Economics, and lead author of the study, said: “Early access to new cancer drugs through FDA’s accelerated approval pathway can save lives, but our findings show that most offered little or no survival benefit while costing Medicare substantial sums. This raises important questions about how to balance rapid access to novel treatments with solid evidence of effectiveness.”

FDA’s accelerated pathway allows new drugs to enter the market based on interim measures rather than waiting for long-term results like overall survival. Pharmaceutical companies are required to run follow-up studies after initial approval to confirm the benefits, but these studies are not always completed. From 1992 to 2020, nearly half of the drugs approved through this pathway were still missing these follow-up studies.

Joseph Ross, Professor of Medicine and Public Health at Yale University and co-author of the study, said: “Treatments which have been approved for use must be followed up with trials measuring overall survival and then appropriate regulatory actions put in place. This will help direct Medicare funds towards treatments with proven clinical benefits.”

Anita Wagner, Associate Professor of Population Medicine at Harvard Medical School, and senior author of the study, added “Importantly, FDA should also clearly communicate the degree of uncertainty about drugs which have had accelerated approvals. This will help clinicians and patients be more informed as they make important health decisions.”

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New marketing approvals were recommended for the following:

• Austedo (deutetrabenazine), for the treatment of adults with moderate-to-severe tardive dyskinesia, a disorder in which predisposed patients experience abnormal involuntary movements resulting from chronic or even episodic exposure to dopamine receptor antagonists.
• Imreplys (sargramostim) for a marketing authorisation under exceptional circumstances for the treatment of people with haematopoietic acute radiation syndrome, when the bone marrow produces fewer blood cells leading to a higher risk of infection and bleeding, following acute exposure to radiation.
• Ogsiveo (nirogacestat) for the treatment of adults with progressing desmoid tumours, a type of soft tissue tumours that form in fibrous tissue, most commonly in the abdomen, arms and legs, that do not spread to other locations.
• Rezdiffra (resmetirom) for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH), a serious disease where fat cells accumulate in the liver causing chronic inflammation.
• Zemcelpro (dorocubicel / unexpanded umbilical cord cells), a stem cell therapy to treat patients with haematological malignancies (blood cancers). This medicine provides an option for patients with blood cancer who need a blood stem cell transplant and have no suitable donor.

The committee adopted positive opinions for six biosimilar medicines:

• Mynzepli (aflibercept), and its duplicate Afiveg (aflibercept), Vgenfli (aflibercept), and its duplicate Eiyzey (aflibercept), for the treatment of age-related macular degeneration and visual impairment.
• Usymro (ustekinumab) for the treatment of plaque psoriasis, arthritis psoriatic and Crohn’s Disease.
• Vivlipeg (pegfilgrastim) to reduce the duration of neutropenia and the incidence of febrile neutropenia after cytotoxic chemotherapy.

Two generic medicines were also recommended for approval:

• Emtricitabine/Rilpivirine/Tenofovir Alafenamide Viatris (emtricitabine / rilpivirine / tenofovir alafenamide), for the treatment of adults and adolescents infected with human immunodeficiency virus type 1 (HIV-1).
• Nintedanib Viatris (nintedanib), for the treatment of idiopathic pulmonary fibrosis, other chronic fibrosing interstitial lung diseases with a progressive phenotype, and systemic sclerosis associated with interstitial lung disease.

The therapeutic indications of six medicines were extended for the following products which had previously been authorised for other indications: Benlysta, Cabometyx, Darzalex, Imbruvica,  Nubeqa, and Sarclisa.

The CHMP recommended an extension of indication for one medicine intended for use outside the EU. Dapivirine Vaginal Ring 25 mg (dapivirine) is a vaginal ring originally approved in July 2020 and used to reduce the risk of women 18 years and older getting infected with human immunodeficiency virus type 1 (HIV-1) through vaginal intercourse. The new opinion extends the indication for this medicine to include its use in women from 16 years of age.

All EMA opinions are subject to formal approval by the European Commission.

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Among these are a number of medicines that stand out due to their contribution to addressing public health needs or the innovation they represent, the Agency said in its Annual Report. Oncology was the area of highest activity, both in the approval of new products and in the extension of therapeutic use of existing drugs.

On the veterinary side, EMA recommended 25 veterinary medicines for marketing authorisation – the highest number of recommendations in a year. Of these, two had a new active substance. Fourteen were vaccines, including seven that had been developed using a biotechnological process.

The Annual Report highlights the Agency’s efforts to invest in better processes and evidence-based regulatory decisions, using initiatives such as Accelerating Clinical Trials in the EU (ACT EU) and the Data Analysis and Real World Interrogation Network (DARWIN EU), facilitating the uptake of advanced manufacturing methods and utilising artificial intelligence for the benefit of patients and public health; and EMA’s work to strengthen collaboration across the European medicines regulatory network and internationally.

Drug evaluation changing fast

The EMA Board also held its annual meeting in June at its Amsterdam headquarters to reflect on 2024 and look ahead at regulatory trends. Bruno Sepodes, chair of EMA’s key human medicines committee (CHMP), presented an overview of achievements and challenges in the committee’s work. ‘2025 is a year of great possibility, with innovative medicines and vaccines advancing through the pipeline,’ said Professor Sepodes.

‘Our regulatory environment is evolving rapidly. The CHMP expects expanded responsibilities from the groundbreaking revision of the EU pharmaceutical legislation; at the same time, advances in science and technology are reshaping how we evaluate medicines, while digitalisation, automation and artificial intelligence will improve and streamline the way we work.’

Professor Sepodes also highlighted the ongoing concerns regarding quality and timeliness of some marketing authorisation applications which can impact regulatory efficiency.

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This almost doubles the number of drugs approved in 2025, bringing the annual total to 33. A further ten products had their therapeutic indications extended.

The newly-approved products were:

• Alyftrek (deutivacaftor / tezacaftor / vanzacaftor), a medicine intended for the treatment of cystic fibrosis in people aged six years and older who have at least one non class I mutation in the cystic fibrosis transmembrane conductance regulator gene.
• Attrogy (diflunisal) for the treatment of hereditary transthyretin-mediated amyloidosis, a disease in which abnormal proteins called amyloids build up in tissues around the body including around the nerves.
• Duvyzat (givinostat), conditionally approved as a treatment for Duchenne muscular dystrophy (DMD) in patients from the age of six who can walk.
• Sephience (sepiapterin) for the treatment of hyperphenylalaninaemia in adults and children with phenylketonuria.
• Tepezza (teprotumumab) for the treatment of adults with moderate to severe Thyroid Eye Disease (TED), also known as Graves’ Eye Disease, a rare autoimmune disease that triggers inflammation of muscles, fat, and other tissues around and behind the eyes.
• Ziihera (zanidatamab), for the treatment of adults with unresectable locally advanced or metastatic HER2-positive biliary tract cancer.
• Oczyesa (octreotide) for the treatment of adults with acromegaly, a disease that leads to excessive growth, particularly of bones in the hands, feet and face caused by too much growth hormone.

The committee adopted positive opinions for nine biosimilar medicines:

• Dazublys (trastuzumab) for the treatment of metastatic and early breast cancer.
• Denbrayce (denosumab), Enwylma (denosumab), Vevzuo (denosumab) and Yaxwer (denosumab), for the prevention of skeletal related events in adults with advanced malignancies, treatment of adults and skeletally mature adolescents with giant cell tumour of bone.
• Denosumab BBL (denosumab), Izamby (denosumab), Junod (denosumab) and Zadenvi (denosumab), for the treatment of osteoporosis and bone loss.

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At the latest meeting of its key human medicines committee (CHMP), gave its backing to the following:

• Deqsiga (human normal immunoglobulin), intended for replacement therapy in people with primary or secondary immunodeficiencies and immunomodulation in people with certain autoimmune diseases.
• Lynozyfic (linvoseltamab) received conditional authorisation for the treatment of patients with relapsed and refractory multiple myeloma, a cancer of the bone marrow.
• Vyjuvek (beremagene geperpavec) to treat wounds in patients of all ages with dystrophic epidermolysis bullosa, a serious, ultra-rare genetic skin blistering disease caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene.
• A generic medicine, Trabectedin Accord (trabectedin), received a positive opinion for the treatment of advanced soft tissue sarcoma and of relapsed platinum-sensitive ovarian cancer.

The committee also recommended extending the therapeutic indication of two cystic fibrosis medicines, Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor), to include their use in combination in patients aged two years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The CHMP extended the existing indication of the chikungunya vaccine Ixchiq (chikungunya vaccine (live)) to include the active immunisation of adolescents from 12 years of age. This vaccine was initially approved to protect adults against the disease caused by Chikungunya virus.

Fabhalta (iptacopan) received an extension of indication for the treatment of adult patients with complement 3 glomerulopathy, an ultra-rare kidney disease that previously had no treatment options.

The other 12 extensions of indication for medicines that are already authorised in the EU were: Abrysvo, Calquence, Columvi, Darzalex, Enhertu, Imfinzi, Jaypirca, Prevymis, Rinvoq, Stelara, Supemtek Tetra and Tremfya.

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Tapinarof is an aryl hydrocarbon receptor (AhR) agonist. It reduces inflammation and normalises skin barrier function by ligand-dependent activation of AhR, resulting in downregulation of inflammatory Th2 cytokines implicated in AD, modulation of keratinocyte differentiation and increased expression of skin-barrier components, including proteins (filaggrin, loricrin, hornerin, and involucrin) and ceramides.¹

The approval was supported by the pivotal ADORING 1 and ADORING 2 studies. These were double-blind, randomised, vehicle-controlled phase 3 trials (NCT05014568, NCT05032859), that assessed the efficacy and safety of tapinarof cream 1%, once daily, in adults and children of two years of age and older with AD.

The results of the eight-week trials showed that significantly higher proportions of patients achieved the efficacy endpoint (a score of 0 or 1 (clear or almost clear) on the Validated Investigator Global Assessment for Atopic Dermatitis (vIGA-AD), along with at least a 2-grade improvement from baseline to week 8).

• ADORING 1: 45.4% with tapinarof vs 13.9% with vehicle (P < .0001)
• ADORING 2: 46.4% with tapinarof vs 18% with vehicle (P < .0001)

Significant improvements were also reported in the secondary endpoints – Eczema Area and Severity Index (EASI) score and the Peak Pruritus Numerical Rating Scale (PP-NRS).  Notably, patients and caregivers reported itch improvement as early as 24 hours after first application of Vtama cream.

Safety

Common treatment-emergent adverse events (TEAEs) (≥5% in any arm) were folliculitis, headache, and nasopharyngitis. Most TEAEs were mild or moderate and were associated with low discontinuation rates. There were few serious TEAEs and none were considered treatment related. Trial discontinuation due to AEs was more frequent in the vehicle arms than with tapinarof.

It is of interest that studies show minimal-to-no systemic absorption tapinarof from the cream under maximal usage conditions, including in children down to two years of age with extensive AD, and in adults with plaque psoriasis and extensive disease.¹

ADORING 3 – Long-term evaluation

ADORING 3, a 48-week open-label, LTE study, enrolled eligible patients from ADORING 1, ADORING 2, and other suitable patients who did not meet pivotal studies’ inclusion criteria.

In ADORING 3, patients (N=728) were followed for up to 48 weeks, with safety and efficacy endpoints that included the achievement of complete disease clearance (vIGA-AD=0), and the achievement of clear or almost clear skin (vIGA-AD=0 or 1). Patients entering with any disease activity (vIGA-AD≥1) were treated with Vtama cream, 1% until complete disease clearance was achieved (vIGA-AD=0) or study completion. For the 378 patients who entered with or achieved complete disease clearance (vIGA-AD=0) in ADORING 3 and discontinued treatment with Vtama cream, the mean duration of the first period of remission was approximately 80 consecutive days. Patients whose AD returned to mild or above (vIGA-AD≥2) were re-treated with VTAMA cream until complete disease clearance was achieved again or study completion. The safety profile with long term use was generally consistent with the safety profile observed at Week 8.

Itch-relief

“Vtama cream approval in AD is important because it can be prescribed for children as young as 2 years old. Vtama cream has the potential to bring much needed relief to a significant number of children suffering from this disease,” said Adelaide A. Hebert, MD, Professor and Chief of Paediatric Dermatology at McGovern Medical School at UT Health Houston and Children’s Memorial Hermann Hospital and lead investigator for the ADORING program. “Moreover, because the prevalence of itch makes this condition extremely burdensome to patients and their families, the itch data from the ADORING program demonstrates reduction of one of the condition’s most prevalent symptoms with use of Vtama cream.”

Take home message

In these 8-week trials, tapinarof cream 1%, once daily was superior to vehicle in the treatment of AD in adults and children. Tapinarof potentially fills a gap in the treatment armamentarium for a highly effective, nonsteroidal topical treatment that can be used down to two years of age without restrictions on the severity of disease, duration of use, total body surface area treated, or sites of application.¹

Reference

1. Silverberg J, Eichenfield LF, Hebert AA et al. Tapinarof cream 1% once daily: Significant efficacy in the treatment of moderate to severe atopic dermatitis in adults and children down to 2 years of age in the pivotal phase 3 ADORING trials. J Am Acad Dermatol. 2024; 94: 457-465

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The committee recommended authorisation for the following:

Balversa (erdafitinib) for the treatment of adult patients with unresectable or metastatic urothelial carcinoma, a cancer of the bladder and urinary system.

Eurneffy (epinephrine), the first emergency treatment against allergic reactions that is administered as a nasal spray, not as an injection.

mResvia (Respiratory Syncytial Virus (RSV) mRNA vaccine) for prevention in adults 60 years of age and older of lower respiratory tract disease and acute respiratory disease caused by respiratory syncytial virus (RSV). RSV is a common respiratory virus that usually causes mild, cold-like symptoms but can lead to serious consequences in older adults. This is the first mRNA vaccine targeting a different pathogen than SARS-CoV-2 that receives a positive opinion from the CHMP.

Ordspono (odronextamab) received a conditional recommendation for the treatment of follicular lymphoma and diffuse large B-cell lymphoma, two types of blood cancer that affect the immune system.

Piasky (crovalimab) for the treatment of paroxysmal nocturnal haemoglobinuria, a rare genetic disorder that causes the premature breakdown of red blood cells by the immune system and is potentially life-threatening.

Tauvid (flortaucipir (¹⁸F)) for positron emission tomography (PET) imaging of the brain in adult patients with cognitive impairment who are being evaluated for Alzheimer’s disease.

Winrevair (sotatercept) to treat adult patients with pulmonary arterial hypertension, a rare, long-term, debilitating and life-threatening condition in which patients have abnormally high blood pressure in the arteries in the lungs.

Steqeyma (ustekinumab), a biosimilar medicine for the treatment of adult patients with moderate/severe active Crohn’s disease, plaque psoriasis, paediatric plaque psoriasis and psoriatic arthritis.

The committee also adopted positive opinions for two generic medicines: Enzalutamide Viatris (enzalutamide) for the treatment of prostate cancer, and Nilotinib Accord (nilotinib) for the treatment of Philadelphia chromosome positive chronic myelogenous leukaemia.

Extensions of indication were recommended for 11 medicines that are already authorised in the EU: Betmiga, Beyfortus, Cresemba, Imcivree, Imfinzi, Infanrix hexa, Lynparza, Pegasys, Tepkinly, Vabysmo and Xalkori.

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