Human medicines European public assessment report (EPAR): Agamree, vamorolone, Date of authorisation: 14/12/2023, Revision: 7, Status: Authorised

Agamree RSS Authorised This medicine is authorised for use in the European Union vamorolone Medicine Human Authorised Application under evaluation CHMP opinion European Commission decision Overview Agamree is a medicine for treating Duchenne muscular dystrophy in patients from 4 years of age. Duchenne muscular dystrophy is a genetic disease that gradually causes weakness and loss of muscle function. Duchenne muscular dystrophy is rare, and Agamree was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 22 August 2014. Further information on the orphan designation can be found on the EMA website . Agamree contains the active substance vamorolone. Expand section Collapse section How is Agamree used? The medicine can only be obtained with a prescription. Treatment should only be started by a specialist doctor with experience in managing Duchenne muscular dystrophy. The medicine is available as a suspension to be taken by mouth once a day. The doctor will prescribe the dose based on the patient’s bodyweight. For more information about using Agamree, see the package leaflet or contact your doctor or pharmacist. How does Agamree work? The active substance in Agamree, vamorolone, is a modified corticosteroid medicine and reduces inflammation by blocking the production of certain inflammatory substances called cytokines. The way it works in patients with Duchenne muscular dystrophy is not fully understood. What benefits of Agamree have been shown in studies? A main study showed that Agamree was more effective than placebo (a dummy treatment) in treating Duchenne muscular dystrophy in patients between the ages of 4 and 7 years who were able to walk. The study, involving 121 patients, looked at their TTSTAND velocity (time to stand), which is the speed at which they can stand up from a lying position. After 24 weeks of treatment, the average TTSTAND velocity increased from 0.19 to 0.24 rises per second in patients who took Agamree, while it decreased slightly from 0.20 to 0.19 rises per second in those who had placebo. This effect was maintained up to week 48. What are the risks associated with Agamree? For the full list of side effects and restrictions with Agamree, see the package leaflet. The most common side effects with Agamree (which may affect more than 1 in 10 people) include Cushingoid features (features caused by long-term use of a corticosteroid, such as fat build-up on the face and bruising), vomiting, increased weight and irritability. The medicine must not be used in patients who have severely impaired liver function or who have recently received a live vaccine (a vaccine that uses a weakened form of the organism). Why is Agamree authorised in the EU? The European Medicines Agency decided that Agamree’s benefits are greater than its risks and it can be authorised for use in the EU. The main study in patients between 4 and 7 years of age showed that Agamree is effective in improving their ability to move. Given that vamorolone works in the same way as currently used corticosteroids, the Agency concluded that it can be used in older patients as well. In terms of safety, Agamree compares well with conventional corticosteroids and does not cause some of the side effects that conventional corticosteroids cause (such as effects on the bone and growth). What measures are being taken to ensure the safe and effective use of Agamree? The company that markets Agamree will provide patients with an alert card with information about the need for daily treatment and the risk of adrenal crisis, a side effect which can occur in patients abruptly stopping corticosteroid treatment. Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Agamree have also been included in the summary of product characteristics and the package leaflet. As for all medicines, data on the use of Agamree are continuously monitored. Suspected side effects reported with Agamree are carefully evaluated and any necessary action taken to protect patients. Other information about Agamree Agamree received a marketing authorisation valid throughout the EU on 14 December 2023. Agamree : EPAR - Medicine overview Reference Number: EMA/489334/2023 English (EN) (108.05 KB - PDF) First published: 04/01/2024 View Other languages (22) български (BG) (132.75 KB - PDF) First published: 04/01/2024 View español (ES) (107.97 KB - PDF) First published: 04/01/2024 View čeština (CS) (129.15 KB - PDF) First published: 04/01/2024 View dansk (DA) (108.13 KB - PDF) First published: 04/01/2024 View Deutsch (DE) (112.18 KB - PDF) First published: 04/01/2024 View eesti keel (ET) (105.7 KB - PDF) First published: 04/01/2024 View ελληνικά (EL) (132.84 KB - PDF) First published: 04/01/2024 View français (FR) (109.21 KB - PDF) First published: 04/01/2024 View hrvatski (HR) (128.16 KB - PDF) First published: 04/01/2024 View italiano (IT) (108.02 KB - PDF) First published: 04/01/2024 View latviešu valoda (LV) (136.93 KB - PDF) First published: 04/01/2024 View lietuvių kalba (LT) (128.26 KB - PDF) First published: 04/01/2024 View magyar (HU) (128.91 KB - PDF) First published: 04/01/2024 View Malti (MT) (131.63 KB - PDF) First published: 04/01/2024 View Nederlands (NL) (108.64 KB - PDF) First published: 04/01/2024 View polski (PL) (132.79 KB - PDF) First published: 04/01/2024 View português (PT) (109.28 KB - PDF) First published: 04/01/2024 View română (RO) (124.41 KB - PDF) First published: 04/01/2024 View slovenčina (SK) (128.96 KB - PDF) First published: 04/01/2024 View slovenščina (SL) (126.75 KB - PDF) First published: 04/01/2024 View Suomi (FI) (105.56 KB - PDF) First published: 04/01/2024 View svenska (SV) (106.94 KB - PDF) First published: 04/01/2024 View Agamree : EPAR - Risk management plan English (EN) (596.8 KB - PDF) First published: 04/01/2024 Last updated: 15/11/2024 View Product information Agamree : EPAR - Product information English (EN) (446.24 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View Other languages (24) български (BG) (541.03 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View español (ES) (468.85 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View čeština (CS) (541.1 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View dansk (DA) (457.32 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View Deutsch (DE) (483.49 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View eesti keel (ET) (464.92 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View ελληνικά (EL) (530.47 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View français (FR) (472.75 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View hrvatski (HR) (522.37 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View íslenska (IS) (479.42 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View italiano (IT) (484.05 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View latviešu valoda (LV) (546.01 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View lietuvių kalba (LT) (537.14 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View magyar (HU) (552.19 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View Malti (MT) (572.35 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View Nederlands (NL) (474.33 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View norsk (NO) (443.97 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View polski (PL) (556.83 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View português (PT) (469.87 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View română (RO) (523.74 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View slovenčina (SK) (557.88 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View slovenščina (SL) (545.16 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View Suomi (FI) (460.44 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View svenska (SV) (460.42 KB - PDF) First published: 04/01/2024 Last updated: 08/04/2026 View Latest procedure affecting product information: VR/0000338132 08/04/2026 This medicine’s product information is available in all official EU languages . Select 'available languages' to access the language you need. Product information documents contain: summary of product characteristics (annex I); manufacturing authorisation holder responsible for batch release (annex IIA); conditions of the marketing authorisation (annex IIB); labelling (annex IIIA); package leaflet (annex IIIB). Agamree : EPAR - All authorised presentations English (EN) (40.21 KB - PDF) First published: 04/01/2024 View Other languages (24) български (BG) (57.51 KB - PDF) First published: 04/01/2024 View español (ES) (41.27 KB - PDF) First published: 04/01/2024 View čeština (CS) (58.08 KB - PDF) First published: 04/01/2024 View dansk (DA) (42.08 KB - PDF) First published: 04/01/2024 View Deutsch (DE) (42.37 KB - PDF) First published: 04/01/2024 View eesti keel (ET) (39.83 KB - PDF) First published: 04/01/2024 View ελληνικά (EL) (54.94 KB - PDF) First published: 04/01/2024 View français (FR) (41.27 KB - PDF) First published: 04/01/2024 View hrvatski (HR) (60.08 KB - PDF) First published: 04/01/2024 View íslenska (IS) (44.51 KB - PDF) First published: 04/01/2024 View italiano (IT) (40.5 KB - PDF) First published: 04/01/2024 View latviešu valoda (LV) (59.27 KB - PDF) First published: 04/01/2024 View lietuvių kalba (LT) (59.09 KB - PDF) First published: 04/01/2024 View magyar (HU) (59.55 KB - PDF) First published: 04/01/2024 View Malti (MT) (60.96 KB - PDF) First published: 04/01/2024 View Nederlands (NL) (40.83 KB - PDF) First published: 04/01/2024 View norsk (NO) (44.56 KB - PDF) First published: 04/01/2024 View polski (PL) (51.63 KB - PDF) First published: 04/01/2024 View português (PT) (41.44 KB - PDF) First published: 04/01/2024 View română (RO) (57.28 KB - PDF) First published: 04/01/2024 View slovenčina (SK) (59.03 KB - PDF) First published: 04/01/2024 View slovenščina (SL) (57.25 KB - PDF) First published: 04/01/2024 View Suomi (FI) (39.92 KB - PDF) First published: 04/01/2024 View svenska (SV) (40.98 KB - PDF) First published: 04/01/2024 View Product details Name of medicine Agamree Active substance vamorolone International non-proprietary name (INN) or common name vamorolone Therapeutic area (MeSH) Muscular Dystrophy, Duchenne Anatomical therapeutic chemical (ATC) code H02AB18 Pharmacotherapeutic group Glucocorticoids Therapeutic indication Agamree is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older. Authorisation details EMA product number EMEA/H/C/005679 Additional monitoring This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring . Orphan This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation . Marketing authorisation holder Santhera Pharmaceuticals (Deutschland) GmbH Marie-Curie Strasse 8 79539 Lorrach Germany Opinion adopted 12/10/2023 Marketing authorisation issued 14/12/2023 Revision 7 Assessment history Expand section Collapse section Changes since initial authorisation of medicine Agamree : EPAR - Procedural steps taken and scientific information after authorisation English (EN) (164.25 KB - PDF) First published: 16/12/2025 Last updated: 08/04/2026 View Agamree : EPAR - Procedural steps taken and scientific information after authorisation (archive) English (EN) (149.88 KB - PDF) First published: 13/05/2024 Last updated: 16/12/2025 View Initial marketing authorisation documents Agamree : Orphan maintenance assessment report - initial authorisation Adopted Reference Number: EMA/OD/0000141144 English (EN) (299.47 KB - PDF) First published: 04/01/2024 View Agamree : EPAR - Public assessment report Adopted Reference Number: EMA/CHMP/487504/2023 English (EN) (2.36 MB - PDF) First published: 04/01/2024 View CHMP summary of positive opinion for Agamree Adopted Reference Number: EMA/CHMP/458203/2023EMA/CHMP/458203/2023 English (EN) (159.17 KB - PDF) First published: 13/10/2023 View News on Agamree Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 9-12 October 2023 13/10/2023 Product information - with tracked changes The approved product information for this medicine is available below showing the changes since the previous procedure affecting the product information. The same document without tracked changes is above under 'Product information'. Agamree : EPAR - Product information - tracked changes English (EN) (277.88 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View Other languages (24) български (BG) (293.6 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View español (ES) (232.24 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View čeština (CS) (231.48 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View dansk (DA) (225.08 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View Deutsch (DE) (287.2 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View eesti keel (ET) (280.11 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View ελληνικά (EL) (242.96 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View français (FR) (233.63 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View hrvatski (HR) (232.7 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View íslenska (IS) (233.22 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View italiano (IT) (230.06 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View latviešu valoda (LV) (286.33 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View lietuvių kalba (LT) (286.39 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View magyar (HU) (239.12 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View Malti (MT) (281.36 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View Nederlands (NL) (234.34 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View norsk (NO) (231.62 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View polski (PL) (284.46 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View português (PT) (236.83 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View română (RO) (285.49 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View slovenčina (SK) (234.84 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View slovenščina (SL) (235.89 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View Suomi (FI) (236.97 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View svenska (SV) (274.16 KB - DOCX) First published: 25/03/2025 Last updated: 20/01/2026 View More information on Agamree EU/3/14/1309 - orphan designation for treatment of Duchenne muscular dystrophy EMEA-001794-PIP02-16-M09 - paediatric investigation plan This page was last updated on 08/04/2026 Share this page