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 • | |||
Information about the number of patients followed in the centres is missing for Cholic acid (accessible in more than 2/3 of the centres), Idebenone (accessible in less than 2/3 of the centres) and Asfotase alpha (accessible in less than 2/3 of the centres). • indicates products prescribed to almost all the patients with the considered condition