Switzerland
Rare diseases
Every rare disease for which Swissmedic has granted at least one orphan drug status. The label follows the published orphan indication.
A
- AA amyloidosis1
- Achondroplasia2
- Adrenoleukodystrophy (ALD)1
- Acromegaly2
- Acute hepatic porphyria1
- Acute lymphoblastic leukaemia (ALL)14
- Acute myeloid leukaemia (AML)15
- Acute promyelocytic leukaemia (APL)2
- AL amyloidosis (light-chain amyloidosis)5
- Alagille syndrome2
- Alpha-1 antitrypsin deficiency with emphysema3
- Alpha-mannosidosis1
- Amyotrophic lateral sclerosis (ALS)8
- Anal fistulas1
- Anal cancer1
- Anaplastic large cell lymphoma (ALCL)1
- Aneurysmal subarachnoid haemorrhage1
- Anthracycline extravasation1
- Aplastic anaemia1
- Atypical haemolytic uraemic syndrome (aHUS)3
- Autoimmune haemolytic anaemia1
- Autosomal dominant polycystic kidney disease (ADPKD)1
- Acquired hypothalamic obesity1
- Advanced idiopathic Parkinson disease1
- Adrenal insufficiency1
- Adrenocortical carcinoma1
- Adult-onset Still disease1
B
C
- Congenital primary bile acid synthesis defects1
- Congenital factor VII deficiency1
- C3 glomerulopathy3
- Castleman disease1
- CDKL5 deficiency disorder1
- Charcot-Marie-Tooth disease1
- Cholangiocarcinoma (biliary tract cancer)4
- Chondrosarcoma1
- Chronic inflammatory demyelinating polyneuropathy (CIDP)2
- Chronic thromboembolic pulmonary hypertension (CTEPH)6
- Chronic eosinophilic leukaemia and hypereosinophilic syndrome3
- Chronic granulomatous disease1
- Chronic lymphocytic leukaemia (CLL)10
- Chronic myeloid leukaemia (CML)7
- Chronic non-infectious uveitis1
- Cryopyrin-associated periodic syndromes (CAPS)2
- Cushing syndrome3
- Cystinuria1
- Cystic fibrosis11
- Cytomegalovirus infection (CMV)5
- Carcinoid syndrome1
- Colorectal cancer with KRAS G12C mutation1
- Conditioning before stem cell transplantation10
- Congenital adrenal hyperplasia (CAH)1
- Cutaneous T-cell lymphoma5
- Cerebrotendinous xanthomatosis (CTX)1
- Cervical dystonia1
D
E
F
- Fallopian tube carcinoma1
- Familial adenomatous polyposis (FAP)2
- Familial amyloid polyneuropathy1
- Familial chylomicronaemia syndrome (FCS)2
- Familial Mediterranean fever (FMF)1
- Fascioliasis1
- Fibrodysplasia ossificans progressiva (FOP)1
- Focal segmental glomerulosclerosis (FSGS)1
- Follicular lymphoma15
- Fragile X syndrome1
- Friedreich ataxia3
- Fabry disease3
G
- Gastrointestinal stromal tumours (GIST)7
- Generalised pustular psoriasis (GPP)2
- Glioma and glioblastoma8
- GNE myopathy1
- Gorlin syndrome (naevoid basal cell carcinoma syndrome)1
- Graft-versus-host disease (GvHD)3
- Granulomatosis with polyangiitis (Wegener granulomatosis)3
- Gastric cancer6
- Gaucher disease3
- Growth hormone deficiency2
H
- Hairy cell leukaemia (HCL)1
- Hailey-Hailey disease (familial benign pemphigus)1
- Haemophagocytic lymphohistiocytosis (HLH)1
- Haemophilia A14
- Haemophilia B8
- Hepatitis delta virus infection1
- Hepatocellular carcinoma (HCC)5
- Hereditary factor X deficiency1
- Hereditary factor XIII deficiency1
- Hereditary angioedema (HAE)12
- High-grade B-cell lymphoma1
- Hodgkin lymphoma3
- Homozygous familial hypercholesterolaemia (HoFH)1
- Huntington disease1
- Hyperimmunoglobulin D syndrome (HIDS)1
- Hypoparathyroidism1
- Hypophosphataemic rickets1
- Hypophosphatasia2
- Hepatic veno-occlusive disease (VOD)2
I
J
L
M
- Malaria4
- Malignant hyperthermia1
- Malignant ascites1
- Malignant melanoma1
- Malignant mesothelioma4
- Mantle cell lymphoma11
- Marginal zone lymphoma (MZL)5
- Mastocytosis4
- Medullary thyroid carcinoma (MTC)1
- Merkel cell carcinoma2
- Metachromatic leukodystrophy (MLD)1
- Methotrexate toxicity1
- Mucopolysaccharidoses (MPS)5
- Mucormycosis2
- Multiple myeloma28
- Myasthenia gravis9
- Myelodysplastic syndromes (MDS)10
- Myelofibrosis11
- Myeloid and lymphoid neoplasms with eosinophilia1
N
O
P
- Pancreatic islet cell carcinoma1
- Post-transplant lymphoproliferative disorder (PTLD)1
- Pompe disease (glycogen storage disease II)4
- Pancreatic cancer7
- Paroxysmal nocturnal haemoglobinuria (PNH)6
- Peripheral T-cell lymphoma (PTCL)3
- Phenylketonuria (hyperphenylalaninaemia)5
- PIK3CA-related overgrowth spectrum (PROS)1
- Pneumonia caused by Pseudomonas aeruginosa1
- Polycythaemia vera3
- Pouchitis1
- Primary biliary cholangitis (PBC)3
- Primary malignant bone tumours1
- Primary sclerosing cholangitis (PSC)1
- Primary apnoea of prematurity1
- Primary hyperoxaluria1
- Primary IgA nephropathy7
- Primary membranoproliferative glomerulonephritis1
- Primary membranous nephropathy1
- Primary peritoneal carcinoma1
- Progressive familial intrahepatic cholestasis (PFIC)2
- Proliferating infantile haemangioma1
- Proopiomelanocortin deficiency (POMC)1
- Pseudoxanthoma elasticum1
- Pulmonary arterial hypertension (PAH)27
- Post-exposure rabies prophylaxis1
R
S
- Small cell lung cancer (SCLC)17
- Short bowel syndrome2
- Spinal cord injury1
- Severe heroin dependence3
- Severe congenital protein C deficiency1
- Sickle cell disease5
- Solid tumours with NTRK gene fusion3
- Spinal muscular atrophy (SMA)6
- Staphylococcal enterocolitis1
- Stargardt disease1
- Symptomatic hypertrophic cardiomyopathy2
- Systemic juvenile idiopathic arthritis (sJIA)2
- Systemic sclerosis (scleroderma)7
- Soft tissue sarcoma9
T
- Thyroid carcinoma4
- Tenosynovial giant cell tumour3
- Thrombotic thrombocytopenic purpura (TTP)3
- TNF receptor-associated periodic syndrome (TRAPS)1
- Transfusional iron overload4
- Transplant rejection after solid organ transplantation5
- Transthyretin amyloidosis (ATTR)8
- Tuberculosis (MDR/XDR)1
- Tuberous sclerosis complex (TSC)6
- Tyrosinaemia type 14
U
V
W
X
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Frequently asked questions
How does a medicine obtain orphan drug status in Switzerland?
On application to Swissmedic. It requires a life-threatening or chronically debilitating disease affecting no more than 5 in 10,000 people in Switzerland, or an existing recognition in a country with comparable medicinal product control (Art. 4 para. 1 let. a decies TPA, Art. 4 TPLO).
What does the status actually give you?
The simplified authorisation procedure (Art. 14 para. 1 let. f TPA), priority assessment, a waiver of the flat-rate fee for the new authorisation application and, on request, 15 years of document protection (Art. 11b para. 4 TPA). There is no market exclusivity as in the EU.
How current is the data on Rare diseases?
Swissmedic publishes the list of medicinal products with orphan drug status under Art. 7 TPLO and updates it monthly. The date of the data set processed here is shown at the foot of every page.
See the service
Bring your orphan drug to Switzerland
You can enquire about orphan drug status, authorisation and reimbursement in Switzerland. Any specialist service and its scope are agreed separately with the external partner.
- Public Swissmedic data
- Four language versions