JP

Japan

JP

East Asia

Regulatory Overview

Japan is regulated by the PMDA (Pharmaceuticals and Medical Devices Agency). 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

PMDA (Pharmaceuticals and Medical Devices Agency)

Regulatory Pathways (2)

Japan's early conditional approval system allowing accelerated approval of certain drugs with post-marketing commitments for patients with serious unmet medical needs.

Application Form: PMDA Early Conditional Approval Application
Legal Basis: PMD Act – Early Approval System
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: PMDA Early Approval Guidance

PMDA's compassionate use (Dojo) system allows access to promising investigational drugs for patients who have exhausted other treatments and cannot wait for full approval.

Application Form: PMDA CU Application
Legal Basis: Pharmaceutical and Medical Device Act (PMD Act)
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: PMDA Compassionate Use Guidance

Drugs Available (105)

DrugAccess RouteStatusLead Time
alglucosidase alfa
cytarabine
eliglustat
eribulin mesylate
Sucralfate
Dorzolamide
Pravastatin
Memantine
Everolimus
metoprolol
carbamazepine
ruxolitinib
mycophenolate mofetil
etanercept
Denosumab
mavacamten
minoxidil
ibuprofen
bosentan
Artificial tears
phacoemulsification surgery
amiodarone
Total Parenteral Nutrition
Growth hormone (somatropin)
ascorbic acid
Digoxin
Crizotinib
physical therapy and orthotic management
eculizumab
Octreotide
aspirin
Valproic Acid
tocilizumab
sclerostin antibody
Immunoglobulin
sirolimus
temozolomide
Carnitine
Pyridoxine
Acetazolamide
vutrisiran
Somatrogon-ghla
levothyroxine sodium
botulinum toxin type A
ambrisentan
Brentuximab vedotin
insulin
eplontersen
prednisolone
vosoritide
Arsenic trioxide
Edaravone
rituximab
Dapagliflozin
allopurinol
Carboplatin
calcium carbonate
coenzyme Q10
Tolvaptan
adalimumab
tacrolimus ophthalmic
ranibizumab
Methotrexate
Alendronate Sodium
calcitriol
doxorubicin
Levetiracetam
prednisone
efgartigimod alfa
salicylic acid
Insulin glargine
cochlear implant
Pyridostigmine Bromide
aflibercept
Bleomycin
acetaminophen
pregabalin
riboflavin
ezetimibe
atorvastatin
bevacizumab
proton pump inhibitors (omeprazole)
Biotin
cyclosporine ophthalmic
Growth Hormone
Tafamidis
Levodopa/Carbidopa
Olipudase alfa
cyanocobalamin
Azacitidine
Zidovudine
warfarin
Vitamin K1 (Phytonadione)
asciminib
Cisplatin
imatinib mesylate
Colchicine
surgical repair
Tacrolimus
Artificial tears (Hypromellose)
Ribavirin
vitamin D3
Sildenafil
Hematopoietic stem cell transplantation
Timolol maleate
JP
Japan
ISO CodeJP
RegionEast Asia
Regulatory AuthorityPMDA (Pharmaceuticals and Medical Devices Agency)
Pathways2 documented
Drugs Available105 drug(s)