Breakthroughs in medicine (gene therapy, innovations in allergies), as well as changes in the healthcare system (electronic health data in the EU).

Breakthrough in gene therapy for eye diseases 

The American FDA (reviews new food, drinks, medicines, and supplements and their suitability for use), approved the first of its kind gene therapy for a rare degenerative eye disease – macular telangiectasia type 2 (MacTel). The disease is progressive and debilitating, leading to permanent deterioration of vision. 

The drug Encelto is an allogeneic (donor) encapsulated cell therapy, which is implanted in the eye and releases a neurotrophic factor that supports the survival of photoreceptors. 

This new treatment gives hope to patients with MacTel, in whom the gradual disappearance of receptor cells leads to loss of central vision. The approval marks an important advance in ocular gene therapy, demonstrating the potential of biotechnology to treat diseases previously considered untreatable. 

Needle-free treatment for allergic shocks in children 

The FDA has expanded the indication of the first nasal spray with epinephrine – neffy – now also for small children (4+ years, weighing 15–30 kg). 

Neffy was approved in 2024 as the first non-injectable emergency treatment for anaphylaxis (severe allergic reaction) in adults and adolescents. Now, with the approval for a younger age and lower weight, the spray provides an easier and faster way to save a life during an anaphylactic shock – without the need for epinephrine (adrenaline) injections. 

This is especially valuable for schools and parents, as using a nasal spray is easier and less frightening for children. The expanded availability of neffy can significantly improve emergency care for severe allergies. 

European Health Data Space (EHDS) 

On March 5, 2025, a new regulation was published in the Official Journal of the EU, creating a pan-European space for electronic health data (European Health Data Space). 

This regulation enters into force on March 26, 2025, and marks the beginning of transformations in the exchange of health information in the EU. The goals are to enable citizens to easily access and share their electronic health records between countries, as well as securely use anonymized health data for scientific research, policies, and innovations (under strict privacy control). 

It is expected that gradually by 2029, all member states will implement the exchange of basic health data – discharge summaries, prescriptions, tests – which will facilitate the treatment of patients abroad and accelerate the development of new medicines through better access to large volumes of health information. The regulation is a key step towards the digitalization of European healthcare and a single market for digital health services. 

Final thoughts 

In recent days, quite a bit of health news has come to light, although we expected some of it in recent months. Research in the field of eye diseases, allergies, and all news that concerns us will be available to learn about on drgogo.bg 

Wang J, Fan W, Liu B, Pu N, Wu H, Xue R, Li S, Song Z, Tao Y. Encapsulated cell technology: Delivering cytokines to treat posterior ocular diseases. Pharmacol Res. 2024 May;203:107159. doi: 10.1016/j.phrs.2024.107159. Epub 2024 Mar 29. PMID: 38554790. 

Ellis AK, Casale TB, Kaliner M, Oppenheimer J, Spergel JM, Fleischer DM, Bernstein D, Camargo CA Jr, Lowenthal R, Tanimoto S. Development of neffy, an Epinephrine Nasal Spray, for Severe Allergic Reactions. Pharmaceutics. 2024 Jun 14;16(6):811. doi: 10.3390/pharmaceutics16060811. PMID: 38931932; PMCID: PMC11207568. 

An epinephrine nasal spray (neffy) for anaphylaxis. Med Lett Drugs Ther. 2024 Oct 14;66(1713):163-164. doi: 10.58347/tml.2024.1713b. PMID: 39382429. 

Fleischer DM, Li HH, Talreja N, Lockey RF, Kaliner MA, Wainford RD, Greenhawt MJ, Casale TB, Lowenthal R, Tanimoto S. Pharmacokinetics and pharmacodynamics of neffy, epinephrine nasal spray, in pediatric allergy patients. J Allergy Clin Immunol Pract. 2025 Mar 20:S2213-2198(25)00269-7. doi: 10.1016/j.jaip.2025.03.019. Epub ahead of print. PMID: 40120808. 

Pouessel G, Neukirch C. Alternatives to Injectable Adrenaline for Treating Anaphylaxis. Clin Exp Allergy. 2025 Jan;55(1):36-51. doi: 10.1111/cea.14598. Epub 2024 Nov 24. PMID: 39581195; PMCID: PMC11707320.