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List of the latest marketing authorisations and orphan medicinal products designations
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
Recent marketing authorisations
> 2016 marketing authorisations
|
Medicinal Product |
Marketing Authorisation Holder |
Therapeutic Indication |
Date of Marketing Authorisation |
|
|
Bavencio® |
Merck Serono Europe Limited | Merkel cell carcinoma | 18/09/2017 | |
|
Besponsa® (inotuzumab ozogamicin) |
Pfizer Limited | Precursor Cell Lymphoblastic Leukemia-Lymphoma | 29/06/2017 | |
|
Brineura® (cerliponase alfa) |
Biomarin International Limited | Neuronal ceroid lipofuscinosis type 2 | 30/05/2017 | |
|
Cystadrops® |
Orphan Europe S.A.R.L. |
Cystinosis |
19/01/2017 |
|
|
Dinutuximab beta Apeiron® |
Apeiron Biologics AG |
Neuroblastoma |
08/05/2017 |
|
|
Ledaga® |
Actelion Registration Ltd. |
T-cell lymphoma |
03/03/2017 |
|
|
(parathyroid hormone) |
Shire Pharmaceuticals Ireland Ltd |
Hypoparathyroidism |
24/04/2017 |
|
|
Oxervate® (cenegermin [recombinant human nerve growth factor]) |
Dompe farmaceutici s.p.a. | Neurotrophic keratitis | 06/07/2017 | |
|
Spinraza® |
Biogen Idec Ltd |
Spinal muscular atrophy |
30/05/2017 |
*Removed from the Community register of orphan medicinal products upon request of the MAH at the time of marketing authorisation
EURORDIS celebrates 20 years
2017 marks the 20th anniversary of EURORDIS-Rare Diseases Europe.
The rare disease community, including all rare disease patient organisations and their partners, can be deservedly proud of its achievements over the last two decades.
News
Find out the latest news from the rare disease community!
RareConnect
A social network of rare disease communities led by EURORDIS in partnership with leading disease-specific patient groups.
- Acanthamoeba keratitis (AK)
- Adrenal Insufficiency
- Alkaptonuria (AKU)
- Allan Herndon Dudley Syndrome MCT8
- Alström Syndrome
- Alternating Hemiplegia
- Atypical Hemolytic Uremic Syndrome
- Amyloidosis
- Aortic Dissection
- Apert Syndrome
- Arachnoiditis (ARC)
- Autoimmune lymphoproliferative syndrome (ALPS)
- Behcet's Syndrome
- Brown-Vialetto-Van Laere syndrome
- CAPS
- Castleman Disease
- CDG
- Choroideremia
- Chromosome 18 abnormalities
- Chronic Recurrent Multifocal Osteomyelitis (CRMO)
- CMTC
- Coats disease
- Cobb Syndrome
- Cone-rod dystrophies
- Cryoglobulinemia
- Cystinosis
- Dravet Syndrome
- DysNet, Dysmelia - Limb Differences
- Ectodermal Dysplasia
- EEC syndrome
- Ehlers-Danlos Syndrome
- Epidermolysis Bullosa
- Erdheim-Chester Disease
- Evans Syndrome
- Familial Mediterranean Fever
- Fetal AntiConvulsant Syndromes
- Fibromuscular dysplasia
- FOXP1
- Glut1 DS
- Hemiconvulsion-hemiplegia-epilepsy syndrome
- Hereditary Leiomyomatosis and Renal Cell Cancer
- Hereditary Sensory and Autonomic Neuropathies (HSAN)
- Hereditary Spastic Paraplegia
- Idiopathic Pulmonary Fibrosis
- Kallmann Syndrome
- Kleefstra syndrome (Ks)
- Leber hereditary optic neuropathy (LHON)
- Leigh Syndrome
- Lipodystrophy
- Lipoprotein Lipase Deficiency
- Liposarcoma
- Lowe Syndrome
- Lymphangiomatosis and Gorham's Disease (LGD)
- Lysosomal acid lipase deficiency (LALD)
- Malignant Infantile Osteopetrosis (MIOP)
- Mastocytosis
- Melkersson-Rosenthal Syndrome (MRS)
- Multiple Myeloma
- Multiple system atrophy
- Moebius Syndrome
- Moyamoya disease
- Multiple Endocrine Neoplasia (MEN)
- Myasthenia Gravis
- Myhre syndrome
- Narcolepsy
- NBIA
- Neuroacanthocytosis
- Neurofibromatosis type 1
- Neurofibromatosis type 2 and Schwannomatosis
- Neuromyelitis Optica (NMO)
- Niemann-Pick disease type C
- Paraneoplastic Neurological Syndromes
- Pemphigus and Pemphigoid
- Pityriasis Rubra Pilaris (PRP)
- Pitt Hopkins Syndrome
- Pontine Tegmental Cap Dysplasia
- Porphyria
- Primary Orthostatic Tremor (POT)
- Propionic Acidemia
- Pseudomyxoma Peritonei
- Pulmonary Hypertension
- Rett Syndrome
- Ring chromosome 20 syndrome
- Sanfilippo Syndrome (MPS III)
- Spontaneous Coronary Artery Dissection
- Stargardt Disease
- Stickler syndrome
- Stiff Person Syndrome
- TRAPS
- Trichorhinophalangeal syndrome
- Trimethylaminuria
- Undiagnosed diseases
- Urea Cycle Disorders
- Vasculitis
- Von Hippel-Lindau
- Waldenstrom macroglobulinemia
- Whim syndrome
- Williams Syndrome
- Acanthamoeba keratitis (AK)
- Adrenal Insufficiency
- Alkaptonuria (AKU)
- Allan Herndon Dudley Syndrome MCT8
- Alström Syndrome
- Alternating Hemiplegia
- Atypical Hemolytic Uremic Syndrome
- Amyloidosis
- Aortic Dissection
- Apert Syndrome
- Arachnoiditis (ARC)
- Autoimmune lymphoproliferative syndrome (ALPS)
- Behcet's Syndrome
- Brown-Vialetto-Van Laere syndrome
- CAPS
- Castleman Disease
- CDG
- Choroideremia
- Chromosome 18 abnormalities
- Chronic Recurrent Multifocal Osteomyelitis (CRMO)
- CMTC
- Coats disease
- Cobb Syndrome
- Cone-rod dystrophies
- Cryoglobulinemia
- Cystinosis
- Dravet Syndrome
- DysNet, Dysmelia - Limb Differences
- Ectodermal Dysplasia
- EEC syndrome
- Ehlers-Danlos Syndrome
- Epidermolysis Bullosa
- Erdheim-Chester Disease
- Evans Syndrome
- Familial Mediterranean Fever
- Fetal AntiConvulsant Syndromes
- Fibromuscular dysplasia
- FOXP1
- Glut1 DS
- Hemiconvulsion-hemiplegia-epilepsy syndrome
- Hereditary Leiomyomatosis and Renal Cell Cancer
- Hereditary Sensory and Autonomic Neuropathies (HSAN)
- Hereditary Spastic Paraplegia
- Idiopathic Pulmonary Fibrosis
- Kallmann Syndrome
- Kleefstra syndrome (Ks)
- Leigh Syndrome
- Leber hereditary optic neuropathy (LHON)
- Lipodystrophy
- Lipoprotein Lipase Deficiency
- Liposarcoma
- Lowe Syndrome
- Lymphangiomatosis and Gorham's Disease (LGD)
- Lysosomal acid lipase deficiency (LALD)
- Malignant Infantile Osteopetrosis (MIOP)
- Mastocytosis
- Melkersson-Rosenthal Syndrome (MRS)
- Multiple Myeloma
- Multiple system atrophy
- Moebius Syndrome
- Moyamoya disease
- Multiple Endocrine Neoplasia (MEN)
- Myasthenia Gravis
- Myhre syndrome
- Narcolepsy
- NBIA
- Neuroacanthocytosis
- Neurofibromatosis type 1
- Neurofibromatosis type 2 and Schwannomatosis
- Neuromyelitis Optica (NMO)
- Niemann-Pick disease type C
- Paraneoplastic Neurological Syndromes
- Pemphigus and Pemphigoid
- Pityriasis Rubra Pilaris (PRP)
- Pitt Hopkins Syndrome
- Pontine Tegmental Cap Dysplasia
- Porphyria
- Primary Orthostatic Tremor (POT)
- Propionic Acidemia
- Pseudomyxoma Peritonei
- Pulmonary Hypertension
- Rett Syndrome
- Ring chromosome 20 syndrome
- Sanfilippo Syndrome (MPS III)
- Spontaneous Coronary Artery Dissection
- Stargardt Disease
- Stickler syndrome
- Stiff Person Syndrome
- TRAPS
- Trichorhinophalangeal syndrome
- Trimethylaminuria
- Undiagnosed diseases
- Urea Cycle Disorders
- Vasculitis
- Von Hippel-Lindau
- Waldenstrom macroglobulinemia
- Whim syndrome
- Williams Syndrome
