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June 2024 Milestones for European Medicines Agency

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June 2024 marked significant milestones for the European Medicines Agency (EMA), showcasing its ongoing commitment to public health and therapeutic innovation. Key developments included accelerated assessments and approvals of groundbreaking treatments, highlighting the agency’s pivotal role in advancing medical science. These achievements underscore the EMA’s dedication to enhancing patient care and safety across Europe.

EMA’s human medicines committee (CHMP) recommended ten new medicines for approval, including Balversa for metastatic urothelial carcinoma and Eurneffy, the first nasal spray for emergency allergic reactions. They also endorsed mResvia, an RSV mRNA vaccine for older adults, and Ordspono for certain blood cancers. Positive opinions were given for Piasky for paroxysmal nocturnal haemoglobinuria, Tauvid for Alzheimer’s PET imaging, and Steqeyma, a biosimilar for Crohn’s disease and psoriasis. The CHMP rejected marketing authorisations for Masitinib for ALS and Syfovre for geographic atrophy. They also decided not to renew the conditional approval for Translarna for Duchenne muscular dystrophy. Additionally, the CHMP updated the Comirnaty vaccine to target the new SARS-CoV-2 JN.1 variant for the upcoming vaccination campaign.

EMA has recommended Winrevair (sotatercept) for marketing authorization in the EU to treat adult patients with pulmonary arterial hypertension (PAH). This condition is characterized by abnormally high blood pressure in the lungs, leading to severe physical limitations and a poor prognosis. Winrevair is the first activin signaling inhibitor approved for PAH, working by regulating blood vessel growth in the lungs to alleviate symptoms. A clinical trial demonstrated that sotatercept significantly improved exercise capacity in PAH patients. While generally well-tolerated, the medication has potential side effects, which are manageable with dosage adjustments.

EMA has recommended marketing authorization in the EU for Eurneffy (epinephrine), the first nasal medication for emergency treatment of anaphylaxis. Anaphylaxis is a rapid and severe allergic reaction that can be life-threatening without prompt treatment, and epinephrine is the standard treatment. Eurneffy offers a needle-free alternative, addressing issues such as fear of needles and lack of portability associated with autoinjectors. Clinical studies in healthy individuals demonstrated that nasally-administered adrenaline has comparable effects to intramuscular injections, with no significant adverse events reported. The CHMP’s recommendation includes additional risk minimization measures and is a preliminary step towards EU-wide patient access, pending European Commission approval and subsequent Member State decisions on pricing and reimbursement.

CHMP has also recommended revoking the marketing authorisation of Ocaliva (obeticholic acid) due to an unfavorable risk-benefit balance. Ocaliva, used to treat primary biliary cholangitis (PBC), showed initial promise in reducing markers of liver damage, but subsequent studies, including the key study 747-302, failed to confirm significant clinical benefits over placebo. Despite comprehensive evaluations and input from experts and patient groups, the data indicated that Ocaliva was not more effective in preventing disease progression or death. As a result, the CHMP concluded that the risks associated with Ocaliva outweigh its benefits. The decision now awaits final approval by the European Commission, after which Ocaliva will no longer be authorised in the EU, though it may still be available through compassionate use programs for current patients.

On the other hand CHMP has recommended not renewing the marketing authorisation for Translarna (ataluren), a treatment for Duchenne muscular dystrophy caused by a nonsense mutation. Following an initial negative opinion in September 2023, confirmed in January 2024 after re-examination, the committee concluded that Translarna’s effectiveness remains unproven. Despite further reviews requested by the European Commission, including new real-world data and expert opinions, the CHMP found insufficient evidence to support the medicine’s benefits. Key studies failed to demonstrate significant clinical improvements over placebo, and additional data did not alter the committee’s assessment. Consequently, the CHMP advised revoking Translarna’s marketing authorisation, pending final approval by the European Commission.

The EMA and the Heads of Medicines Agencies (HMA) have issued recommendations to address shortages of GLP-1 receptor agonists like Ozempic, Saxenda, Trulicity, and Victoza, used for diabetes and weight management. Since 2022, increased demand and misuse for cosmetic weight loss have exacerbated shortages. The EMA urges optimized distribution, increased manufacturing, and proper use through guidelines and education. They recommend a study on real-world usage and a multistakeholder workshop for additional solutions. Healthcare professionals should prescribe these medications only for authorized uses, and the public should avoid unsupervised use to prevent health risks and exposure to falsified products.

European Medicines Agency

The Committee adopted positive opinions for several veterinary medicines, including a new vaccine for cattle (DIVENCE IBR Marker Live) and a rabies vaccine for dogs (Rabitec) with updated formulations. They also approved updates for Suvaxyn PRRS MLV and Daxocox, and added a new therapeutic indication for Stronghold Plus. Quality-related changes were approved for various products, and new adverse reactions were included for Credelio. Revised guidelines for antimicrobial and intramammary products were adopted for public consultation. Additionally, a new Q&A document on product classification was issued, and Boudewijn Catry was elected vice-chair of the Antimicrobials Working Party.

The updated Clinical Trials Information System (CTIS) now offers earlier and more efficient access to clinical trial information in the EU, thanks to new transparency rules. These changes make information on approximately 4,000 authorised clinical trials immediately accessible, with 500 new trials added monthly. The rules ensure early publication of key trial data while protecting confidential information. Resources like a user guide and support activities help sponsors adapt. CTIS serves as the single-entry point for trial applications and includes a public database to enhance transparency for healthcare professionals and the public.

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EMA has initiated a review of metamizole-containing medicines due to concerns about the effectiveness of measures to minimise the risk of agranulocytosis, a serious side effect. Despite efforts to enhance safety measures, cases continue to be reported, leading the Finnish medicines agency to request this review. The Pharmacovigilance Risk Assessment Committee (PRAC) will evaluate the benefit-risk balance of these medicines and recommend whether their marketing authorisations should be maintained, varied, suspended, or revoked. Stakeholders are invited to submit relevant data to support this review process.

EMA has also initiated a review of metamizole-containing medicines due to concerns that current measures to minimise the risk of agranulocytosis, a severe drop in white blood cells, may not be effective. These medicines, used in various EU countries to treat pain and fever, continue to be associated with cases of agranulocytosis despite being listed as rare or very rare side effects. The PRAC will evaluate the risks and benefits to determine if their marketing authorisations should be maintained, varied, suspended, or revoked. Additionally, PRAC has identified the risk of secondary malignancies of T-cell origin in patients treated with CAR T-cell medicines, prompting updates to product information and lifelong monitoring for new malignancies.

The EMA’s Management Board reviewed and approved the 2023 activity report, highlighting achievements and a new governance structure for managing medical device shortages. They prepared for a new fee regulation starting January 2025 to support the regulatory network’s sustainability. Updates included successful efforts to address medicine shortages and an upcoming workshop on GLP-1 receptor agonist shortages. Dr. Violeta Stoyanova discussed trends in orphan medicine development, emphasizing the importance of patient experience data.

The Accelerating Clinical Trials in the EU (ACT EU) initiative has launched two pilots to improve clinical trial applications. The first pilot offers joint scientific advice from the EMA’s Scientific Advice Working Party and the HMA’s Clinical Trials Coordination Group, aiming to harmonize their assessments for marketing authorisation applications and clinical trial applications. The second pilot provides technical and regulatory support on clinical trial application dossiers before submission, offering consolidated feedback from Member States. Starting today, developers can apply for these pilots, which will be evaluated over time to inform future improvements. These initiatives aim to enhance application quality and strengthen European clinical trial coordination.

EMA also recommends transitioning from quadrivalent to trivalent influenza vaccines for the 2024/2025 season, excluding the B/Yamagata strain. This strain has not been detected since March 2020 due to COVID-19 measures and is no longer considered a public health threat. Manufacturers should include specific A and B strains in their trivalent vaccines.

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