Table 3 Categories of EMA-approved OMPs for HMDs according to accessibility and delivery to patients in the MetabERN centres
| OMP accessible in > 2/3 of the centres (mean = 88.5%) | OMP accessible in < 2/3 of the centres (mean = 40.2%) | ||
|---|---|---|---|
| Delivered to > 1/3 of the patients (mean = 70.1%, range 38–100%) | Delivered to < 1/3 of the patients (mean = 15.8% range 7–23%) | Delivered to > 1/3 of the patients (mean = 66.3% range 45–100%) | Delivered to < 1/3 of the patients (mean = 6.0% range 0–18%) |
| Sodium Phenylbutyrate | Carglumic acid | Chenodeoxycholic acid • | Glycerol phenylbutyrate |
| Nitisinone • | Sapropterin | Cysteamine hydrochloride | |
| Miglustat | Migalastat | Cysteamine bitartrate | Alipogene tiparvovec |
| Agalsidase alpha | Eliglustat | Cerliponase | ADA CD34 cells |
| Agalsidase beta | Velaglucerase | Elosulfase | |
| Imiglucerase | Sebelipase | ||
| Alglucosidase alpha * | Afamelanotide | ||
| Laronidase | |||
| Idursulfase • | |||
| Galsulfase • | |||