Table 2.
Individuals enrolled in the Humanitarian Program by disease
| Disease | Year disease introduced to program | Historical data | Current datab | ||||
|---|---|---|---|---|---|---|---|
| Number of individuals since inception | Number of countries supported since inception | Number of individuals receiving humanitarian treatment annually | Number of countries supported today | Average number of new cases approved each year; over past 5 years | Average time current individuals have received humanitarian treatment (years) | ||
| Gaucher disease | 1991 | 1248 | 75 | 652 | 52 | 48.2 | 9.8 |
| Fabry disease | 2003 | 972 | 50 | 150 | 21 | 59.2 | 3.4 |
| Pompe disease | 2006 | 766 | 70 | 142 | 37 | 45.8 | 4.8 |
| MPS I | 2003 | 390 | 55 | 55 | 20 | 9.8 | 6.6 |
| MPS IIa | 2014 | 35 | 7 | 24 | 6 | 5 | 3.0 |
| Total | 3411 | 103 | 1023 | 69 | 168 | 5.5 | |
Data as of May 2021
MPS, Mucopolysaccharidosis
aHumanitarian access in MPS II is limited to Sanofi Genzyme territory only
bData for individuals entering the Humanitarian Program are dynamic; with a rolling submission process, new individuals are approved throughout the year. Individuals also depart the program owing to a number of reasons, including death, individual or physician decision to stop treatment, and commercial transition