List of Drugs Used in Treatment of Orphan Diseases in Nepal

The 4th amendment of Drug Registration Regulation-2038 on 2078 includes the following 55 Drugs Used in Treatment of Orphan Diseases in Nepal.

List of Drugs Used in Treatment of Orphan Diseases in Nepal

S.N.	Name of Medicine	Dosage form and Strength
1.	Adrenocorticotropic Hormone	Powder for injection: 0.25 mg/vial
2.	Vigabatrin	Tablet: 500 mg Powder for oral solution 500 mg/packet
3.	Pyridoxine	Tablet: 25 mg (hydrochloride)
4.	Immunoglobulins	Intramuscular administration: 16% protein solution Intravenous administration: 5%, 10% protein solution Subcutaneous administration: 15%, 16% protein solution Indicated for primary immune deficiency Indicated for primary immune deficiency and Kawasaki disease
5.	Botulinum Toxins	Injectable powder for reconstitution 50 units/vial 100 units/vial 200 units/vial
6.	Tetrabenazine	Tablet 12.5mg, 25 mg
7.	Pyridoxal phosphate	Tablet 25, 50, 100, 250, 500 mg Extended release 200 mg Capsule 250 mg
8.	Folinic acid	Tablets 5mg, 10mg, 15mg, 25mg Injection powder for reconstitution 50mg, 100mg, 200mg, 350 mg, 500mg Injectable solution 10mg/ml
9.	Deferasirox	Tablet for oral suspension 125mg, 250mg, 500mg Oral tablet 90mg, 180mg, 360mg Oral granule 90mg, 180mg, 360mg
10.	Deferoxamine	Powder for injection: 500 mg (mesilate) in vial
11.	6-Thioguanine	Tablet 40mg
12.	Testosterone	Oral capsule 158 mg, 198 mg, 237 mg Injectable solution Cypionate 100mg/ml (Depo-Testosterone) 200mg/ml (Depo-Testosterone) Injectable solution Enanthate 50 mg/0.5ml 75 mg/0.5ml 100 mg/0.5ml 200 mg/ml
13.	Dihydrotestosterone gel	2.5% gel for topical application
14.	Hydrocortisone	Powder for injection: 100 mg (as sodium succinate) in vial Tablet: 10mg, 20 mg
15.	Growth hormone	Injection: 0.2mg, 0.4, 0.6 0.8, 1, 1.2, 1.4, 1.6, 1.8, 2 mg
16.	Anakinra	Prefilled syringes 100mg/0.67ml
17.	Tocilizumab	Injectable solution 20 mg/ml
18.	Methotrexate	Powder for injection 1g/vial (25 mg/ml)
19.	Methylphenidate	Oral tablets 5, 10, 20 mg
20.	Atomoxetine	Oral capsule 5, 10, 18, 25, 40, 60, 80, 100 mg

21.	Caffeine citrate	10 mg/ml (each 1 ml solution contains 10 mg Caffeine citrate)
22.	Prostaglandin	5-10 nanogram/kg/min
23.	Surfactants (beractant)	Intratracheal suspension 25 mg/ml
24.	Concentrated RL	3 mEq of sodium per ml
25.	Semaglutide	Injection prefilled single dose pen 2 mg/1.5 ml ; delivers dose of 0.25 mg, 0.5 mg or 1 mg per injection
26.	Testosterone Patch Gel	2mg/24 hr 4mg/24 hr
27.	Desmopressin nasal spray	5 ml bottle with spray pump delivering 50 sprays of 10 mcg
28.	Hydrocortisone	Oral tablet 5, 10, 20 mg
29.	Fludrocortisone	0.1 mg tablet
30.	Testosterone	100 mg/ml 200 mg/ml
31.	Octreotide acetate	1 ml single dose vials in 3 strengths containing 50, 100 or 500 mcg Octreotide
32.	Pegvisomant	Powder for injection 10, 15, 20 mg 40 mg SC
33.	Teriparaide	Prefilled injection pen 250 mcg/ml
34.	Cabergoline	0.5 mg tablets
35.	Crizanlizumab	Injectable solution 10 mg/ml
36.	Voxelotor	500 mg tablets
37.	Recombinant human thyrotropin	Powder for injection 1.1 mg/vial
38.	Riluzole	50 mg tablet Oral suspension 5 mg/ml (300 ml multidose bottle)
39.	Pyridostigmine	Injection solution 5 mg/ml Tablet controlled release 180 mg Tablet 60 mg Syrup 60 mg/5 ml (250ml)
40.	Interferon Beta	Prefilled IM syringe 30 mcg/0.5 ml Prefilled IM autoinjector pen 30 mcg/0.5ml Powder for injection 30 mcg/vial Prefilled SC syringes titration pack 8.8 mcg/0.2 ml (6 syringes)
41.	Glatiramer acetate	Solution for injection 20 mg/ ml, 40 mg/ml
42.	Intranasal triptans	5 mg/ actuation 10 mg/actuation 20 mg/actuation
43.	D-penicillamine	Tablet 250 mg Capsule 125 mg, 250 mg
44.	Trientine	Capsule 250 mg
45.	Antihemophilic factor VIII	250 IU (Alphanate Humate P) 450 IU FVIII/450 VWF:RCo(Wilate)
46.	Imiglucerase	Powder for injection 200 units 400 units
47.	Anti Thymocyte Globulin	Injectable solution 50 mg/ml
48.	FEIBA	Powder for reconstitution 500 units 1000 units 2500 units
49.	Buprenorphine	Sublingual tablets 2 mg/8mg
50.	Pomalidomide	Capsule 1 mg, 2mg, 3mg, 4mg
51.	Ibrutinib	Capsule 70 mg, 140 mg Tablet 140 mg, 280 mg, 420 mg, 560 mg
52.	Tolvaptan	Tablet 15 mg, 30 mg
53.	Melatonin	1-5 mg tablet
54.	Pilocarpine	Tablet 5 mg, 7.5 mg
55.	Panhematin	Lyophilized powder for injection 301 mg/vial 7mg/ml