Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network

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