Table 2.
Main information of the OMP clinical dossier
| Active principle | Trade name | Indication | Prevalence disease (1/10.000) | Dose finding | Type of trial | Control | End-point | n |
|---|---|---|---|---|---|---|---|---|
| **Agalsidase alpha | Replagal | Fabry disease | 0.25 | Yes | RCT | Placebo | Reduction of pain; reduction of GB3 (NS); Reduction of cardiac mass;improvement of renal function | 41 |
| **Agalsidase beta | Fabrazyme | Fabry disease | 0.25 | Yes | RCT | Placebo | Reduction of GL-3 | 56 |
| **Anagrelide | Xagrid | Essential thrombocythaemia | 2–3 | Yes | Open label; nonrandomized; uncontrolled | None | Platelet count <600 × 109/l or reduction >50% from baseline and maintenance of the reduction for at least 4 weeks (= CR) | 1446 |
| **Arsenic trioxide | Trisenox | Acute promyelocytic leukaemia | NA | No | Uncontrolled phase II | None | Complete response; overall survival | 52 |
| **Bosentan | Tracleer | Pulmonary arterial hypertension | 0.005–0.07 | No | RCT | Placebo | Exercise (walking) | 32 |
| Busulfan | Busilvex | Conditioning haematopoietic progenitor cell transplantation | 0.66 | No | Uncontrolled phase II | None | Same effect of i.v. as oral busulfan | 104 |
| **Carglumic acid | Carbaglu | N-acetyl glutamate synthase deficiency | 0.00125 | No | Retrospective study | None | Decrease of ammonia concentration | 20• |
| Celecoxib | Onsenal | Familial adenomatous polyposis | 0.3–1 | No | RCT | Placebo | Decrease of colorectal polyps | 970 |
| Cladribine | Litak | Hairy cell leukaemia | NA | No | Uncontrolled phase II (+ literature analysis) | None | Complete + partial responses | 120 |
| Ibuprofen | Pedea | Patent ductus arteriosus in preterm newborn infants (<34 weeks of gestational age) | NA | Yes | RCT; controlled (+ metanalysis) | Placebo | Proportion of patients requiring surgical ligation of PDA after prophylactic or curative/or treatment with i.v. ibuprofen | 131 |
| **Iloprost | Ventavis | Primary pulmonary hypertension | 0.005–0.07 | No | RCT | Placebo | Improvement in walking; improvement of 1 NYHA class | 203 |
| **Imatinib | Glivec | Chronic myeloid leukaemia GIST (unresectable) | 0.18–0.5 | Yes | Uncontrolled phase II | None | (CML) Haematological and cyto-genetic response; (GIST) Tumour response | 1225; 147 |
| **Laronidase | Aldurazyme | Mucopoly-saccharidosis MPS-1 | 0.025 | No | RCT | Placebo | Reduction of urinary GAG; reduction of hepatosplenomegaly; increase forced vital capacity; exercise-walking (NS) | 45 |
| **Miglustat | Zavesca | Gaucher disease type 1 | 0.33 | No | Uncontrolled phase II | None | Reduction of linea and spleen volume | 28 |
| Mitotane | Lysodren | Adrenal cortical carcinoma | 0.1 | No | Literature analysis | None | Survival; remission time; tumour size reduction | 500 |
| Pegvisomant | Somavert | Resistant acromegaly | 0.5–0.7 | No | RCT | Placebo | Decrease in IGF-1 | 112 |
| Porfimer sodium | Photobarr | Dysplasia in Barrett's oesophagus | 2.3 | No | RCT | Omeprazole | Complete responses | 208 |
| Zinc acetate dihydrate | Wilzin | Wilson's disease | 0.6 | Yes | Open label; nonrandomized; uncontrolled | None | Effects on copper metabolism 24 h copper excretion and non-coeruloplasmin plasma copper (NCPC); effect on speech and neurological function measured on integer scale; effect on liver function tests, liver enzymes (bilirubin, albumin) | 148 |
n, number of patients; NA, not available;
•
retrospective patients;
**
approved under exceptional circumstances.