Russia | List of Pediatric Rare Diseases and Orphan Drugs Covered by Special Fund Drug Cost Number of Per Child Total Cost Disease Drug Children Per Year (RUB) Per Year (RUB) Takeda's Gattex 91 18,900,000 1,700,000,000 (teduglutide) Novartis' Zykadia 20 500,000 10,000,000 (ceritinib) Pfizer's Lorviqua 20 10,000,000 200,000,000 (lorlatinib) EUSA's Qarziba Short Bowel Syndrome 150 20,000,000 3,000,000,000 (dinutuximab beta) Roche's Rozlytrek 10 10,000,000 100,000,000 (entrectinib) Bristol-Myers Squibb's 5 Sprycel (dasatinib) Takeda's Inclusig 5 (ponatinib) Malignant Neoplasms Bayer's Vitrakvi with NTRK Gene 50 36,000,000 1,800,000,000 (loratrectinib) Mutation Novartis' Tafinlar 40 1,200,000 48,000,000 (dabrafenib) Brain Tumors Novartis' Mekinist 60 840,000 50,400,000 (trametinib) Catecholaminergic nadolol (no rug in Russia Polymorphic 32 8,000 256,000 formulary) Ventricular Tachycardia nadolol 100 8,000 800,000 Long QT Syndrome Actelion's Uptravi Jervel-Lange-Neilsen 10 2,400,000 24,000,000 (selexipag) Syndrome sildenafil 178 170,400 30,300,000 Bayer's Ventavis (iloprost) 5 1,200,000 5,600,000 Congenital diazoxide 114 17,700,000 Hyperinsulinism Hyper IgD Syndrome/Mevalonate Novartis' Ilaris 21 24,000,000 504,000,000 Kinase Deficiency (canakinumab) Syndrome (HIDS/MKD) Novartis' Ilaris Cryopyrin-Associated 48 12,000,000 576,000,000 (canakinumab) Periodic Syndrome Roche's Actemra (CAPS) 10 2,000,000 20,000,000 (tocilizumab) Roche's Actemra 20 2,000,000 40,000,000 (tocilizumab) adalimumab 15 937,000 14,100,000 etanercept 10 754,000 7,500,000 Novartis' Ilaris 120 12,000,000 1,400,000,000 (canakinumab) Familial Mediterranean Roche's Actemra 15 2,000,000 30,000,000 Fever (tocilizumab) adalimumab 10 937,000 9,400,000 etanercept 10 754,000 7,500,000 Pompe Disease alglucosidase alfa 32 18,400,000 588,800,000 Hypophosphatasia asfotase alfa 15 38,700,000 579,800,000 (HPF) Familial Amgen's Repatha Hypercholesterolemia (evolocumab) and 20 24,500,000 490,000,000 (Homozygous Form) lomitapide Lysosomal Acid Lipase Alexion's Kanuma 38 24,600,000 934,800,000 (LALL) Deficiency (sebelipase alfa) Mucopolysaccharidosis elosulfase alfa 39 38,600,000 1,500,000,000 Type IV A Urea Cycle Disorder sodium glycerol butyrate 30 6,000,000 180,000,000 (UCM) and carboxylic acid Biogen's Spinraza (nusinersen) Spinal Muscular Roche's Evrysdi (risdiplam) 890 23,000,000,000 Atrophy (SMA) Novartis' Zolgensma (onasemnogene abeparvovec) Epilepsy and Epileptic tetracosactide 6,000 40,000 240,000,000 Encephalopathy hexaacetate Novartis' Afinitor Tuberous Sclerosis 425 4,000,000 1,700,000,000 (everolimus) Alpha Mannosidosis velmanase alfa 16 25,000,000 400,000,000 Amryt's Myalepta Lipodystrophy 70 25,000,000 1,800,000,000 (metreleptin) Duchenne Muscular PTC's Translarna (ataluren) 60 27,900,000 1,700,000,000 Dystrophy (DMD) Neuronal Ceroid BioMarin's Brineura 30 56,000,000 1,700,000,000 Lipofuscionsis Type 2 (cerliponase alfa) Kyowa Kirin's Crysvita Rickets (burosumab) and special 150 13,000,000 2,000,000,000 phosphorous preparations Violation of Beta- Oxidation of Fatty Acids triheptanoin 50 6,000,000 300,000,000 (FAA) Phenylketonuria (PKU) pegvaliase 150 2,800,000 420,000,000 biotin (high-dose not Biotinidase Deficiency 30 100,000 3,000,000 registered in Russia) Bile Acid Synthesis cholic acid 3 3,500,000 10,500,000 Disorders Leber Congenital Novartis' Luxturna 2 50,000,000 100,000,000 Amaurosis (voretigene neparvovec)