Table 8 Approved products (N = 44) for designated intractable diseases
From: Clinical data required for the approval of pediatric pharmaceuticals in Japan
Generic name | Indication | Designated intractable disease number | Administrative category |
|---|---|---|---|
Risdiplam | Spinal muscular atrophy | 3 | Orphan drug |
Nusinersen sodium | Spinal muscular atrophy without manifestation but onset predicted by genetic testing | 3 | Orphan drug |
Eculizumab (genetic recombinant) | Generalized myasthenia gravis (only for patients with symptoms not controlled by high-dose intravenous immunoglobulin therapy or plasmapheresis) | 11 | Orphan drug |
Satralizumab (genetic recombinant) | Prevention of relapse of neuromyelitis optica spectrum disorders (including neuromyelitis optica) | 13 | Orphan drug |
Cerliponase alfa (genetic recombinant) | Ceroid lipofuscinosis type II | 19 | Orphan drug |
Idursulfase beta (genetic recombinant) | Mucopolysaccharidosis type II | 19 | Orphan drug |
Pabinafusp alfa (genetic recombinant) | Mucopolysaccharidosis type II | 19 | Orphan drug Pioneer drug designation system Sakigake |
Avalglucosidase alfa (genetic recombinant) | Pompe disease | 19 | Orphan drug |
Vestronidase alfa (genetic recombinant) | Mucopolysaccharidosis type VII | 19 | Orphan drug Pre-reviewed in “Review Meeting of Unapproved Drugs and Off-Label Drugs with High Medical Needs” |
Olipudase alfa (genetic recombinant) | Acid sphingomyelinase deficiency | 19 | Orphan drug Pioneer drug designation system Sakigake |
Migarastat hydrochloride | Fabry disease with GLA variant responsive to migarastat | 19 | Orphan drug |
Selumetinib sulfate | Plexiform neurofibroma in neurofibromatosis type 1 | 34 | Orphan drug |
Secukinumab (genetic recombinant) | Psoriasis vulgaris, psoriatic arthritis, and pustular psoriasis (generalized) | 37 | None |
Belimumab (genetic recombinant) | Systemic lupus erythematosus inadequately responsive to existing treatments | 49 | None |
Rituximab (genetic recombinant) | Lupus nephritis inadequately responsive to conventional treatment | 49 | Public knowledge-based application after pre-evaluation |
Anti-human thymocyte immunoglobulin, equine | Moderate-to-severe aplastic anemia | 60 | Orphan drug Pre-reviewed in “Review Meeting of Unapproved Drugs and Off-Label Drugs with High Medical Needs” |
Eltrombopag olamine | Aplastic anemia | 60 | Orphan drug |
Caplacizumab (genetic recombinant) | Acquired thrombotic thrombocytopenic purpura | 63 | Orphan drug |
Defibrotide sodium | Hepatic sinusoidal obstruction syndrome (central veno-occlusive disease of the liver) | 65 | Orphan drug Pre-reviewed in “Review Meeting of Unapproved Drugs and Off-Label Drugs with High Medical Needs” |
pH4-treated normal human immunoglobulin | Agammaglobulinemia or hypogammaglobulinemia | 65 | None |
Berotralstat hydrochloride | Acute attacks of hereditary angioedema | 65 | Orphan drug Pioneer drug designation system Sakigake |
Lanadelumab (genetic recombinant) | Acute attacks of hereditary angioedema | 65 | Orphan drug |
Icatibant acetate | Acute attack of hereditary angioedema | 65 | Orphan drug |
Human C1-inactivator | Acute attacks of hereditary angioedema | 65 | Orphan drug |
Human normal immunoglobulina | Agammaglobulinemia or hypogammaglobulinemia | 65 | None |
Human normal immunoglobulinb | Agammaglobulinemia or hypogammaglobulinemia | 65 | None |
Evinacumab (genetic recombinant) | Homozygous familial hypercholesterolemia | 79 | Orphan drug |
Ambrisentan | Pulmonary arterial hypertension | 86 | Orphan drug |
dalimumab (genetic recombinant) | Moderate or severe ulcerative colitis | 97 | None |
Ravulizumab (genetic recombinant) | Atypical hemolytic uremic syndrome | 109 | None |
Viltolarsen | Duchenne muscular dystrophy with confirmed deletion of the dystrophin gene that can be treated by exon 53 skipping | 113 | Orphan drug Pioneer drug designation system Sakigake Conditional early approval system |
Fenfluramine hydrochloride | Concomitant therapy with antiepileptic drugs for epileptic seizures in patients with Dravet syndrome who do not respond adequately to other antiepileptic drugs | 140 | Orphan drug |
Everolimus | Tuberous sclerosis | 158 | Orphan drug |
Somatropin (genetic recombinant) | Body composition abnormalities in Prader-Willi syndrome | 193 | Orphan drug |
Levothyroxine sodium hydrate | Myxedema coma Hypothyroidism (only if treatment with oral levothyroxine sodium is not appropriate) | 235 | Pre-reviewed in “Review Meeting of Unapproved Drugs and Off-Label Drugs with High Medical Needs” |
Burosumab (genetic recombinant) | FGF23-related hypophosphatemic rickets and osteomalacia | 238 | Orphan drug |
Givosiran sodium | Acute hepatic porphyria | 254 | Orphan drug |
Vosoritide (genetic recombinant) | Achondroplasia without epiphyseal closure | 276 | Orphan drug |
Sirolimus | Refractory lymphatic diseases (lymphangioma, lymphangiomatosis, Goham’s disease, lymphangiectasia) | 277 | Orphan drug |
Albutrepenonacog alfa (genetic recombinant) | Inhibition of bleeding tendency in patients with blood coagulation factor IX deficiency | 288 | None |
Simoctocog alfa (genetic recombinant) | Inhibition of bleeding tendency in patients with blood coagulation factor VIII deficiency | 288 | None |
Concizumab (genetic recombinant) | Inhibition of bleeding tendency in congenital hemophilia patients with inhibitors of coagulation factors VIII or IX | 288 | Orphan drug |
Efanesoctocog alfa (genetic recombinant) | Inhibition of bleeding tendency in patients with blood coagulation factor VIII deficiency | 288 | None |
Lonafarnib | Hutchinson-Gilford-Progeria syndrome and processing-deficient progeroid laminopathy | 333 | Orphan drug |