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List of the marketing authorisations
Detailed information on European orphan medicinal products designation applications is available on the EMA website. A full list of designated and authorised orphan medicinal products in Europe available at: ec.europa.eu.
Learn more about the Orphan designation process in Europe
| Medicinal Product | Marketing Authorisation Holder | Therapeutic Indication | Date of Marketing Authorisation |
|---|---|---|---|
| Spexotras® (Trametinib dimethyl sulfoxidetinib) |
Novartis Europharm Limited | Glioma | 05/01/2024 |
| Rystiggo® (Rozanolixizumab) |
UCB Pharma | Myasthenia Gravis | 05/01/2024 |
| Omjjara® (Momelotinib) |
GlaxoSmithKline Trading Services Limited |
Splenomegaly | 21/01/2024 |
| Skyclarys® (Omaveloxolone) |
Reata Ireland Limited | Friedreich’s ataxia | 09/02/2024 |
| Casgevy® (Exagamglogene autotemcel) |
Vertex Pharmaceuticals (Ireland) Limited | Beta- Tahalassemia, Anemia, Sickle Cell | 09/02/2024 |
| Voydeya® (Danicopan) |
Alexion Europe SAS | Paroxysmal nocturnal haemoglobinuria (PNH) | 19/04/2024 |
| Zynyz® (Exagamglogene autotemcel) |
Incyte Biosciences Distribution B.V. | Merkel cell carcinoma (MCC) | 19/04/2024 |
| Filspari® (Sparsentan) |
Vifor France | Primary immunoglobulin A nephropathy | 19/04/2024 |
| Fabhalta® (Iptacopan) |
Novartis Europharm Limited | Paroxysmal nocturnal haemoglobinuria (PNH) | 17/05/2024 |
| Qalsody® (Tofersen) |
Biogen Netherlands B.V. | Amyotrophic lateral sclerosis (ALS) | 29/05/2024 |
| Altuvoct® efanesoctocog alfa |
Swedish Orphan Biovitrum AB (publ) | Hemophilia A | 17/06/2024 |
| Adzynma® (rADAMTS13 Purpura) |
Takeda Manufacturing Austria AG | Congenital thrombotic thrombocytopenic purpura (cTTP) | 01/08/2024 |
| Winrevair® (Sotatercept) |
Merck Sharp & Dohme B.V. | Pulmonary arterial hypertension (PAH) | 22/08/2024 |
| Medicinal Product | Marketing Authorisation Holder | Therapeutic Indication | Date of Marketing Authorisation |
|---|---|---|---|
| Hemgenix® (etranacogene dezaparvovec) |
CSL Behring Gmbh | Haemophilia B | 20/02/2023 |
| Tibsovo® (ivosidenib) |
Les Laboratoires Servier | Acute Myeloid Leukaemia and cholangiocarcinoma | 12/05/2023 |
| Pombiliti® (cipaglucosidase alfa) |
Amicus Therapeutics Europe Limited | Glycogen Storage Disease Type II | 16/05/2023 |
| Hyftor® (sirolimus) |
Plusultra pharma GmbH | Facial angiofibroma | 15/05/2023 |
| Ztalmy® (ganaxolone) |
Marinus Pharmaceuticals Emerald Limited | Epileptic seizures in children with CDKL5 | 26/07/2023 |
| Talvey® (talquetamab) |
Janssen-Cilag International N.V. | Multiple Myeloma | 21/08/2023 |
| Tepkinly® (epcoritamab) |
AbbVie Deutschland GmbH & Co. KG | Diffuse large B-cell lymphoma (DLBCL) | 22/09/2023 |
| Finlee® (dabrafenib esylate) |
Novartis Europharm Limited | Glioma | 15/11/2023 |
| Yorvipath® (palopegteriparatide) |
Ascendis Pharma Bone Diseases A/S | Chronic hypoparathyroidism | 17/11/2023 |
| Loargys® (pegzilarginase) |
Immedica Pharma AB | Hyperargininemia | 15/12/2023 |
| Agamree® (vamorolone) |
Santhera Pharmaceuticals (Deutschland) GmbH | Duchenne muscular dystrophy | 14/12/2023 |
| Rezzayo® (rezafungin) |
Mundipharma GmbH | Invasive candidiasis | 22/12/2023 |
| Medicinal Product | Marketing Authorisation Holder | Therapeutic Indication | Date of Marketing Authorisation |
|---|---|---|---|
| Tavneos® (avacopan) |
Vifor Fresenius Medical Care Renal Pharma France | Adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) | 11/01/2022 |
| Voraxaze® (glucarpidase) |
SERB SAS | Adults and children (aged 28 days and older) with delayed methotrexate elimination or at risk of methotrexate toxicity | 11/01/2022 |
| Lonapegsomatropin Ascendis Pharma® (lonapegsomatropin) |
Ascendis Pharma Endocrinology Division A/S | Children who do not produce enough growth hormone (GHD) | 11/01/2022 |
| Oxbryta® (voxelotor) |
Global Blood Therapeutics Netherlands B. V. |
Haemolytic anaemia, and +12 years old sickle cell disease |
14/02/2022 |
| Ngenla® (somatrogon) |
Pfizer Europe MA EEIG | Children and adolescents with growth hormone deficiency |
14/02/2022 |
|
Kimmtrak®
(tebentafusp) |
Immunocore Ireland Limited | Adult patients with unresectable or metastatic uveal melanoma | 01/04/2022 |
| Uplizna® (inebilizumab) |
Viela Bio | Adults with neuromyelitis optica spectrum disorders (NMOSD) | 25/04/2022 |
| Carvykti® (ciltacabtagene autoleucel) |
Janssen-Cilag International NV | Adults with multiple myeloma | 25/05/2022 |
| Lunsumio® (mosunetuzumab) |
Roche Registration GmbH | Adults with follicular lymphoma | 03/06/2022 |
| Filsuvez® (birch bark extract) |
Amryt Pharmaceuticals DAC | Adults and children aged 6 months or older with epidermolysis bullosa (EB). | 21/06/2022 |
| Kinpeygo® (budesonide) |
Calliditas Therapeutics AB | Adults with primary immunoglobulin A nephropathy (IgAN) | 15/07/2022 |
| Zokinvy® (lonafarnib) |
EigerBio Europe Limited | 12 months and older living with progeria and laminopathies | 18/07/2022 |
| Upstaza® (eladocagene exuparvovec) |
PTC Therapeutics International Limited | Adults and children aged 18 months and older with severe aromatic L-amino acid decarboxylase (AADC) deficiency | 18/07/2022 |
| Vyvgart® (efgartigimod alfa) |
Argenx | Adults with myasthenia gravis | 10/08/2022 |
| Roctavian® (valoctocogene roxaparvovec) |
BioMarin International Limited | Haemophilia A | 24/08/2022 |
| Scemblix® (asciminib) |
Novartis Europharm Limited | Chronic myeloid leukaemia (CML) | 25/08/2022 |
| Enjaymo® (sutimlimab) |
Genzyme Europe BV | Haemolytic anaemia in adults with cold agglutinin disease (CAD) |
15/11/2022 |
| Livtencity® (maribavir) |
Takeda Pharmaceuticals | Cytomegalovirus (CMV) in adults | 9/11/2022 |
| Pyrukynd® (mitapivat) |
Agios Netherlands | Adults with pyruvate kinase deficiency (PKD) | 9/11/2022 |
| Livmarli® (maralixibat chloride) |
Mirum Pharmaceuticals | 2 months and older patients with cholestatic pruritis | 9/11/2022 |
| Mycapssa® (octreotide) |
Amryt Pharmaceuticals DAC | Acromegalia | 2/12/2022 |
| Ebvallo® (tabelecleucel) |
Atara Biotherapeutics Ireland Limited | Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) | 16/12/2022 |
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Orphan drug designations
See the list of orphan medicines currently authorised in the EU
Overview of Orphan Medicinal Product Designation Procedure since 2000
Including and prior to 2018 the EMA also published the detailed information summarised below:
Year
Applications submitted
Positive COMP opinions
Applications withdrawn
Negative COMP opinions
Designations granted by the Commission
Orphan medicinal products authorised
Orphan designations included in authorised therapeutic indication
2018
203
152
79
3
126
12
15
2017
260
144
96
2
138
14
15
2016
330
220
82
2
209
14
14
2015
258
177
94
1
190
14
21
2014
329
196
61
2
187
15
16
2013
201
136
60
1
136
7
8
2012
197
139
52
1
148
10
12
2011
166
111
45
2
107
5
5
2010
174
123
51
2
128
4
4
2009
164
113
23
0
106
9
9
2008
119
86
31
1
73
6
7
2007
125
97
19
1
98
13
13
2006
104
81
20
2
80
9
11
2005
118
88
30
0
88
4
4
2004
108
75
22
4
73
6
6
2003
87
54
41
1
55
5
5
2002
80
43
30
2
49
4
4
2001
83
62
27
1
64
3
3
2000
72
26
6
0
14
0
0
Total
3211
2134
881
28
2121
164
185
Adapted from the July 2018 Committee for Orphan Medicinal Products (COMP) meeting report