OrphanDrug
OrphanDrug
Pharma Knowledge Base
DE

Germany

DE

Europe

Contents

  1. 1. Regulatory Overview
  2. 2. Regulatory Pathways
  3. 3. Available Drugs

Regulatory Overview

Germany is regulated by the BfArM (Federal Institute for Drugs and Medical Devices). The country has 2 documented regulatory pathways for accessing unapproved or unlicensed medicines, covering named patient programs, compassionate use schemes, and special access arrangements.

Regulatory Authority

BfArM (Federal Institute for Drugs and Medical Devices)

Regulatory Pathways (2)

§21a AMG Compassionate Use – Germany 30 days

Compassionate use under §21a of the German Medicinal Products Act (AMG), authorised by BfArM for patients with serious or life-threatening diseases who cannot be adequately treated with authorised medicines.

Application Form: BfArM CU Application
Legal Basis: §21a Arzneimittelgesetz (AMG)
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: BfArM Compassionate Use Guidance
Germany Compassionate Use (Article 83 EMA) 30 days

EMA Article 83 compassionate use framework allowing access to unauthorised medicinal products for groups of patients with a chronically or seriously debilitating or life-threatening disease in Germany.

Application Form: National compassionate use form
Legal Basis: Regulated under EU Directive 2001/83/EC
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: EMA Article 83 Compassionate Use

Drugs Available (210)

DrugAccess RouteStatusLead Time
alglucosidase alfa———
cytarabine———
Ataluren———
eliglustat———
furosemide———
eribulin mesylate———
Sucralfate———
Dorzolamide———
Pravastatin———
Temsirolimus———
Memantine———
propranolol———
Berotralstat———
Everolimus———
Ursodeoxycholic acid———
tamoxifen citrate———
atropine sulfate———
mitapivat———
metoprolol———
carbamazepine———
ruxolitinib———
Corticosteroids———
mycophenolate mofetil———
Epoprostenol———
Fludarabine———
etanercept———
Denosumab———
dapsone———
Tofacitinib———
mavacamten———
mannose———
minoxidil———
cyclophosphamide———
Anakinra———
bosentan———
bimatoprost———
testosterone———
Artificial tears———
Rho(D) immune globulin———
Baclofen———
Dexrazoxane———
Vigabatrin———
triheptanoin———
C1 esterase inhibitor (human)———
loncastuximab tesirine———
phacoemulsification surgery———
fosdenopterin———
melatonin———
amiodarone———
Total Parenteral Nutrition———
Growth hormone (somatropin)———
ascorbic acid———
hormone replacement therapy (estrogen/progesterone)———
Busulfan———
Digoxin———
Bimekizumab-bkzx———
Interferon-gamma———
Crizotinib———
physical therapy and orthotic management———
Retinoids (Tretinoin)———
Branched-Chain Amino Acids———
eculizumab———
miglustat———
lonafarnib———
Betaine anhydrous———
Octreotide———
oxybutynin———
Valproic Acid———
tocilizumab———
sclerostin antibody———
Immunoglobulin———
Mesalamine———
alpha-L-iduronidase———
L-DOPA———
Intravenous Immunoglobulin (IVIG)———
Phenytoin———
Bisphosphonates (pamidronate)———
Metronidazole———
Nitisinone———
Lomitapide———
treprostinil———
sirolimus———
temozolomide———
Carnitine———
brivaracetam———
Infliximab———
Pyridoxine———
vutrisiran———
Angiotensin-Converting Enzyme Inhibitors (Enalapril)———
idebenone———
Lorenzo's oil———
ivermectin———
botulinum toxin type A———
mexiletine———
Crizanlizumab———
azathioprine———
sunscreen (broad spectrum SPF 50+)———
Somatropin———
galantamine———
ambrisentan———
Brentuximab vedotin———
L-carnitine———
luspatercept———
trabectedin———
eplontersen———
vosoritide———
cysteamine bitartrate———
Cerebrolysin———
rituximab———
Dapagliflozin———
allopurinol———
Carboplatin———
Topical corticosteroids———
estradiol———
Erythromycin———
Erythropoietin———
coenzyme Q10———
Patisiran———
eladocagene exuparvovec———
Tolvaptan———
cerliponase alfa———
avalglucosidase alfa———
atidarsagene autotemcel———
Sodium Thiosulfate———
adalimumab———
Imiglucerase———
Amphotericin B liposomal———
ranibizumab———
Lumasiran———
diacerein———
Methotrexate———
carglumic acid———
Alendronate Sodium———
calcitriol———
doxorubicin———
Pyridostigmine———
Levetiracetam———
Cenegermin———
Acitretin———
prednisone———
efgartigimod alfa———
salicylic acid———
cochlear implant———
Pyridostigmine Bromide———
aflibercept———
ravulizumab———
laronidase———
Romiplostim———
Bleomycin———
hydroxycobalamin———
Metreleptin———
Laser photocoagulation therapy———
pregabalin———
riboflavin———
N-acetylcysteine———
NovoSeven———
Occupational and Physical Therapy Support———
Pamidronate———
nitroglycerin———
ezetimibe———
Belzutifan———
bevacizumab———
Ketoconazole———
mecasermin———
celecoxib———
Biotin———
Imiquimod———
cyclosporine ophthalmic———
Growth Hormone———
Gonadotropin———
deferasirox———
Tafamidis———
urea———
Levodopa/Carbidopa———
Lanreotide———
cyanocobalamin———
diazoxide———
baricitinib———
Azacitidine———
Cystagon———
Bezafibrate———
Zoledronic Acid———
Hypertonic saline———
copper histidinate———
Clonazepam———
emollients with keratolytics———
cornstarch———
disopyramide———
asciminib———
selumetinib———
Sunitinib———
mannose-1-phosphate———
Cisplatin———
Calcipotriol———
Imatinib———
imatinib mesylate———
nimodipine———
Colchicine———
Follitropin alfa———
surgical repair———
Tamoxifen———
Tacrolimus———
Artificial tears (Hypromellose)———
Ribavirin———
Sildenafil———
sotalol———
Hematopoietic stem cell transplantation———
Tretinoin———
Timolol maleate———
Amnioreduction———
DE
Germany
ISO CodeDE
RegionEurope
Regulatory AuthorityBfArM (Federal Institute for Drugs and Medical Devices)
Pathways2 documented
Drugs Available210 drug(s)