OrphanDrug
OrphanDrug
Pharma Knowledge Base
FR

France

FR

Europe

Contents

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

Regulatory Overview

France is regulated by the ANSM (Agence Nationale de Sécurité du Médicament). 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

ANSM (Agence Nationale de Sécurité du Médicament)

Regulatory Pathways (2)

France 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 France.

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
AAP (Accès Précoce) – France 60 days

France's early access authorization (formerly ATU) allows patients to access promising medicines before marketing authorization. Managed by ANSM under the reformed 2021 system (Article L.5121-12 Code de la Santé Publique).

Application Form: ATU/AAP Application
Legal Basis: Article L.5121-12 Code de la Santé Publique
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: Article L.5121-12 Code de la Santé Publique

Drugs Available (167)

DrugAccess RouteStatusLead Time
alglucosidase alfa———
cytarabine———
Ataluren———
eliglustat———
furosemide———
eribulin mesylate———
Sucralfate———
Dorzolamide———
Pravastatin———
Memantine———
propranolol———
Everolimus———
Ursodeoxycholic acid———
tamoxifen citrate———
atropine sulfate———
metoprolol———
carbamazepine———
ruxolitinib———
mycophenolate mofetil———
Epoprostenol———
etanercept———
Denosumab———
dapsone———
mavacamten———
mannose———
minoxidil———
cyclophosphamide———
Anakinra———
bosentan———
bimatoprost———
testosterone———
Artificial tears———
Rho(D) immune globulin———
Baclofen———
Dexrazoxane———
Vigabatrin———
triheptanoin———
loncastuximab tesirine———
phacoemulsification surgery———
melatonin———
amiodarone———
Total Parenteral Nutrition———
Growth hormone (somatropin)———
ascorbic acid———
hormone replacement therapy (estrogen/progesterone)———
Busulfan———
Digoxin———
Interferon-gamma———
physical therapy and orthotic management———
Retinoids (Tretinoin)———
eculizumab———
miglustat———
lonafarnib———
Betaine anhydrous———
Octreotide———
oxybutynin———
Valproic Acid———
tocilizumab———
sclerostin antibody———
Immunoglobulin———
alpha-L-iduronidase———
Phenytoin———
Bisphosphonates (pamidronate)———
Metronidazole———
Nitisinone———
treprostinil———
sirolimus———
temozolomide———
Carnitine———
brivaracetam———
Pyridoxine———
vutrisiran———
Angiotensin-Converting Enzyme Inhibitors (Enalapril)———
idebenone———
ivermectin———
botulinum toxin type A———
mexiletine———
azathioprine———
sunscreen (broad spectrum SPF 50+)———
galantamine———
ambrisentan———
L-carnitine———
luspatercept———
trabectedin———
vosoritide———
cysteamine bitartrate———
rituximab———
Dapagliflozin———
allopurinol———
Carboplatin———
Topical corticosteroids———
estradiol———
Erythromycin———
coenzyme Q10———
eladocagene exuparvovec———
Tolvaptan———
cerliponase alfa———
avalglucosidase alfa———
atidarsagene autotemcel———
adalimumab———
Amphotericin B liposomal———
ranibizumab———
diacerein———
Methotrexate———
carglumic acid———
Alendronate Sodium———
calcitriol———
doxorubicin———
Pyridostigmine———
Levetiracetam———
Acitretin———
prednisone———
efgartigimod alfa———
salicylic acid———
cochlear implant———
Pyridostigmine Bromide———
aflibercept———
laronidase———
Bleomycin———
hydroxycobalamin———
Metreleptin———
Laser photocoagulation therapy———
pregabalin———
riboflavin———
N-acetylcysteine———
Occupational and Physical Therapy Support———
Pamidronate———
Diosmin———
nitroglycerin———
ezetimibe———
Belzutifan———
bevacizumab———
mecasermin———
celecoxib———
Biotin———
Imiquimod———
cyclosporine ophthalmic———
Growth Hormone———
Gonadotropin———
deferasirox———
Tafamidis———
urea———
Levodopa/Carbidopa———
Lanreotide———
cyanocobalamin———
diazoxide———
baricitinib———
Azacitidine———
Zoledronic Acid———
Hypertonic saline———
emollients with keratolytics———
cornstarch———
disopyramide———
selumetinib———
Cisplatin———
imatinib mesylate———
nimodipine———
Colchicine———
surgical repair———
Tamoxifen———
Artificial tears (Hypromellose)———
Ribavirin———
Sildenafil———
sotalol———
Hematopoietic stem cell transplantation———
Timolol maleate———
Amnioreduction———
FR
France
ISO CodeFR
RegionEurope
Regulatory AuthorityANSM (Agence Nationale de Sécurité du Médicament)
Pathways2 documented
Drugs Available167 drug(s)