#chmp

Lenacapavir è il primo farmaco a lunga durata approvato per la prevenzione dell’HIV. Somministrato due volte all’anno, ha dimostrato efficacia completa nello studio clinico condotto su oltre 2.000 persone. Il parere positivo del CHMP apre una nuova prospettiva per la gestione della profilassi.

I joined a hardcore minecraft smp with some friends! >:] I’m the little black creature

Champanes “We’re In Trouble” @champanesband #shoegaze #dreampop

Ken Keirns

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Ο EMA (Ευρωπαϊκός Οργανισμός Φαρμάκων) εισηγείται την έγκριση του Teizeild (teplizumab), της πρώτης θεραπείας που μπορεί να καθυστερήσει την εμφάνιση του σταδίου 3 διαβήτη τύπου 1 σε ενήλικες και παιδιά από 8 ετών με στάδιο 2 νόσου. Ο διαβήτης τύπου 1 είναι μια αυτοάνοση πάθηση όπου το ανοσοποιητικό καταστρέφει τα β-κύτταρα που παράγουν ινσουλίνη. Τα συμπτώματα εμφανίζονται στο στάδιο 3, όταν η καθημερινή χορήγηση ινσουλίνης γίνεται απαραίτητη. Η καθυστέρηση της εξέλιξης προς αυτό το στάδιο αποτελεί σημαντικό όφελος, ειδικά για τα παιδιά. Το Teizeild δρα επιβραδύνοντας την αυτοάνοση καταστροφή των β-κυττάρων και χορηγείται ενδοφλεβίως για 14 συνεχόμενες ημέρες. Σε κλινική μελέτη με 76 ασθενείς, το Teizeild έδειξε:• Διπλάσιο χρόνο μέχρι την εκδήλωση του σταδίου 3 (50 μήνες vs 25 μήνες)• Μικρότερο ποσοστό εξέλιξης της νόσου (45% vs 72%)• Σημαντική διατήρηση της λειτουργίας των β-κυττάρων Οι συχνότερες ανεπιθύμητες ενέργειες ήταν χαμηλά λευκά αιμοσφαίρια και εξάνθημα. Σε μικρό ποσοστό ασθενών (2%) καταγράφηκε σύνδρομο απελευθέρωσης κυτοκινών. Η γνωμοδότηση του EMA θα σταλεί στην Ευρωπαϊκή Επιτροπή για την τελική απόφαση. Δείτε την επίσημη ανακοίνωση του EMA εδώ. Read the full article

The European Medicines Agency (EMA) has recommended marketing authorisation in the EU for Teizeild (teplizumab), the first treatment proven to delay the onset of stage 3 type 1 diabetes in adults and children aged 8 and above with stage 2 disease. Type 1 diabetes is an autoimmune condition in which the immune system destroys insulin-producing beta cells. Symptoms appear at stage 3, when daily insulin therapy becomes essential. Delaying progression to this stage offers a major benefit, especially for children. Currently, no approved therapies exist to delay the disease. Teizeild is a monoclonal antibody that slows the immune attack on beta cells. It is administered once daily for 14 days via intravenous infusion. Evidence comes from a placebo-controlled trial in 76 patients, showing:• Median time to stage 3 diabetes: 50 months with Teizeild vs 25 months with placebo• 45% of Teizeild-treated patients progressed to stage 3 vs 72% with placebo• Additional studies confirmed better preservation of beta-cell function The most common side effects were reduced white blood cell counts and rash. A small number of patients (2%) experienced cytokine release syndrome. Teizeild received support under EMA’s PRIME scheme, reflecting its public health importance. The CHMP recommendation now moves to the European Commission for final approval. Official EMA link. Read the full article

The European Medicines Agency (EMA) has recommended granting EU-wide marketing authorisation for Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich syndrome (WAS). The treatment is intended for patients aged 6 months and older who need a haematopoietic stem cell transplant (HSCT) but do not have an available compatible donor. Wiskott-Aldrich syndrome is a rare and life-threatening genetic disorder that affects immune and blood cell function, leading to severe infections, bleeding episodes, and increased cancer risk. For patients without a suitable stem cell donor, there has been no effective treatment option until now, representing a major unmet medical need. Waskyra uses the patient’s own stem cells, which are genetically modified in the laboratory to produce a functional WAS protein. Once infused back into the patient, these modified cells restore the production of healthy immune and blood cells. Waskyra is administered once, via intravenous infusion. Clinical evidence from 27 treated patients shows a dramatic improvement:• Annual severe infections decreased from 2.0 to 0.15–0.12 events post-treatment• Moderate and severe bleeding episodes fell from 2.0 to 0.16 events over two to three years Side effects were mostly associated with pre-treatment conditioning and catheter-related issues. EMA’s Committee for Advanced Therapies (CAT) and the CHMP concluded that the benefits clearly outweigh the risks for patients with no suitable HSCT donor. Waskyra also received support through an EMA pilot for academic and non-profit developers of advanced therapies. The recommendation now moves to the European Commission for final approval. Read the official EMA communication here. Read the full article