Αρχική World News EMA Recommends Refusal of the Marketing Authorisation for Tagraxofusp

EMA Recommends Refusal of the Marketing Authorisation for Tagraxofusp

The European Medicines Agency (EMA) has recommended the refusal of the marketing authorisation for tagraxofusp (Elzonris), a medicine intended for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). The Agency issued its opinion on 23 July 2020.

The company that applied for authorisation, Stemline Therapeutics B.V, may ask for re-examination of the opinion within 15 days of receiving the opinion.

Elzonris was developed as a medicine for treating adults with BPDCN, a rare and aggressive type of acute myeloid leukaemia. In BPDCN, the bone marrow produces large numbers of immature white blood cells called plasmacytoid dendritic cells. These build up in the bone marrow, taking the place of normal blood cells, and spread to the skin: most patients develop non-itchy damaged areas, which often look like bruises or nodules. The disease may also cause enlargement of the spleen or liver and a reduction of the number of circulating blood cells.

There is no approved treatment for BPDCN in the EU.

Elzonris contains the active substance tagraxofusp and was to be available as an infusion given into a vein.

Elzonris was designated an orphan medicine (a medicine used in rare diseases) on 11 November 2015 for the treatment of BPDCN.

The active substance in Elzonris, tagraxofusp, is made up of a diphtheria toxin, which has been linked to interleukin-3. The interleukin-3 attaches to receptors, which are found at high levels on the surface of BPDCN cancer cells. Once attached to cancer cells, it is taken up by them, allowing the toxin to be released inside and kill them. This is expected to prevent cancer spread and reduce symptoms of the disease.

To support its application, the company presented results from a single small study involving 47 adults with BPDCN that could not be treated in other ways. The main measure of effectiveness which was recorded in a group of 13 first line patients was the number of patients without any signs of disease after treatment or with just minimal skin damage remaining.

The Agency was concerned that due to the design of the study and the small number of patients involved, it was not possible to be sure how effective the medicine was in treating BPCDN. In addition, the medicine could cause capillary leak syndrome (an unpredictable, potentially life-threatening side effect due to increased permeability of small blood vessels), which had led to some fatal outcomes.

Therefore, the Agency’s opinion was that the benefits of Elzonris did not outweigh its risks and it recommended refusing marketing authorisation.

The company informed the Agency that there are no consequences for patients in clinical trials or in compassionate use programmes with Elzonris.

Source

NEWSLETTER

Συμπληρώστε το email σας για να λαμβάνετε τις σημαντικότερες ειδήσεις από το ogkologos.com

Βρείτε μας

2,449ΥποστηρικτέςΚάντε Like
57ΑκόλουθοιΑκολουθήστε

Διαβαστε Επίσης

ΕΞΕΛΙΞΕΙΣ ΣΤΗ ΘΕΡΑΠΕΙΑ ΤΟΥ ΜΗ-ΜΙΚΡΟΚΥΤΤΑΡΙΚΟΥ ΚΑΡΚΙΝΟΥ ΤΟΥ ΠΝΕΥΜΟΝΑ (ΜΜΚΠ)

ΕΞΕΛΙΞΕΙΣ ΣΤΗ ΘΕΡΑΠΕΙΑ ΤΟΥ ΜΗ-ΜΙΚΡΟΚΥΤΤΑΡΙΚΟΥ ΚΑΡΚΙΝΟΥ ΤΟΥ ΠΝΕΥΜΟΝΑ (ΜΜΚΠ) Γράφει ο Δρ Παπαδούρης Σάββας, Παθόλογος-Ογκολόγος   Ο ΜΜΚΠ βρίσκεται αναλογικά στο 80% και πλέον του συνολικού...

Διατρέχουν όντως οι καρκινοπαθείς μεγαλύτερο κίνδυνο λόγω κοροναϊού;

Σε πρακτικό επίπεδο, τα δεδομένα των σχετικών μελετών υποδηλώνουν ότι η χημειοθεραπεία ή οι άλλες αντι-νεοπλασματικές θεραπείες δεν αυξάνουν σημαντικά τον κίνδυνο θνησιμότητας από...

FDA: Η ακτινοβολία των smartphones δεν προκαλεί καρκίνο

Σε μια νέα έκθεσή της, η Υπηρεσία Τροφίμων και Φαρμάκων (FDA) των ΗΠΑ αναφέρει ότι επανεξέτασε τις σχετικές επιστημονικές έρευνες που δημοσιεύθηκαν τα τελευταία...

Νέα ανακάλυψη, νέα ελπίδα για τον καρκίνο

Ένα νεοανακαλυφθέν τμήμα του ανοσοποιητικού μας συστήματος θα μπορούσε να αξιοποιηθεί για την αντιμετώπιση όλων των ειδών καρκίνου, σύμφωνα με επιστήμονες του πανεπιστημίου Cardiff...

Καρκίνος: Ευεργετική για τους καρκινοπαθείς η άσκηση

Σημαντικά ωφέλη προσφέρει η άσκηση, ακόμη και σε ασθενείς με καρκίνο. Οι περισσότεροι ασθενείς αγνοούν τα οφέλη που μπορεί να έχει γι’ αυτούς η συχνή...

Prostate Cancer

The article is provided by Scientific Communications Officer of NIPD Genetics, Ms Marina Charitou (MSc) Prostate Cancer Awareness Prostate cancer is the second most common cancer...
- Advertisment -

Ροή Ειδήσεων

Targeted Therapy Based on Molecular Profiling of Malignant Mesothelioma Tumours Is Feasible

Targeted therapy recommendations can be made based on the molecular profiles of tumour samples from patients with metastatic malignant mesothelioma (MM) according to findings...

News digest – targeted radiation beam, precision medicine and our early detection roadmap

<img width="620" height="348" src="https://zniup3zx6m0ydqfpv9y6sgtf-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/Radiation_hero-1.jpg" class="attachment-hero size-hero" alt srcset="https://zniup3zx6m0ydqfpv9y6sgtf-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/Radiation_hero-1.jpg 620w, https://zniup3zx6m0ydqfpv9y6sgtf-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/Radiation_hero-1-300x168.jpg 300w, https://zniup3zx6m0ydqfpv9y6sgtf-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/Radiation_hero-1-220x123.jpg 220w, https://zniup3zx6m0ydqfpv9y6sgtf-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/Radiation_hero-1-150x84.jpg 150w" sizes="(max-width: 620px) 100vw, 620px" data-attachment-id="18059" data-permalink="https://scienceblog.cancerresearchuk.org/2020/10/17/news-digest-targeted-radiation-beam-precision-medicine-and-our-early-detection-roadmap/radiation_hero-3/" data-orig-file="https://scienceblog.cancerresearchuk.org/wp-content/uploads/2020/10/Radiation_hero-1.jpg" data-orig-size="620,348" data-comments-opened="1"...

A Pet Pug Helped Two Sisters Realize They Both Had Breast Cancer

Sisters Amanda Tasca and Amy Niosi are grateful for one very perceptive pug. Amanda’s dog Daisy, a gift from her husband Steve and one...

Woman with Breast Cancer May Have Saved Her Twin’s Life by Revealing Her Lumpectomy Scar

When Meagan McCallum was diagnosed with breast cancer, she had no idea that her experience might actually save her sister’s life. Meagan underwent a...

Lo que debe saber sobre el cáncer de pulmón

La Dra. Jyoti D. Patel es profesora de Medicina y directora de Oncología Torácica de la Universidad de Chicago y es editora asociada para...

Alternatives for Outdoor Foods

Autumn has arrived, but that doesn’t mean outdoor dining has to end! You might be wondering what foods you could prepare that are healthy...