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Table 1 Eligible populations and genotypes for the treatments with the currently approved CFTR modulators

From: The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis

  

IVA ≥ 4 months (and ≥ 5 kg)

LUM/IVA ≥ 2 years

TEZ/IVA ≥ 6 years

ELX/TEZ/IVA ≥ 6 years

FDA

EMA

FDA

EMA

FDA

EMA

FDA

EMA

F508del

F508del

–

–

✓

✓

✓

✓

✓

✓

Gating

F508del

Any Gating mutation responsive to IVA* or **

Any Gating mutation responsive to IVA*

–

–

Any Gating mutation responsive to TEZ/IVA**

–

✓

✓

Non-F508del

Any Gating mutation responsive to ELX/TEZ/IVA**

–

Conduction

F508del

R117H or any Conduction mutation responsive to IVA**

R117H

–

–

Any Conduction mutation responsive to TEZ/IVA**

–

✓

✓

Non-F508del

Any Conduction mutation responsive to ELX/TEZ/IVA**

–

RF

F508del

Any RF mutation responsive to IVA* or **

–

–

–

Any RF mutation responsive to TEZ/IVA* or **

Any RF mutation responsive to TEZ/IVA*

✓

✓

Non-F508del

–

Any RF mutation responsive to ELX/TEZ/IVA**

–

MF

F508del

Any MF mutation responsive to IVA**

–

–

–

Any MF mutation responsive to TEZ/IVA**

–

✓

✓

Non-F508del

Any MF mutation responsive to ELX/TEZ/IVA**

–

Other

F508del

Any Other mutation responsive to IVA**

–

–

–

Any Other mutation responsive to TEZ/IVA**

–

✓

✓

Non-F508del

Any Other mutation responsive to ELX/TEZ/IVA**

–

  1. (The complete list of each single eligible mutation is reported in Figs. 1 and 2) Definitions: (✓) = eligible; (–) = not eligible; *based on clinical evidence; **based only on in vitro data
  2. IVA ivacaftor (VX-770), LUM lumacaftor (VX-809), TEZ tezacaftor (VX-661), ELX elexacaftor (VX-445), RF residual function CFTR mutation, MF minimal function CFTR mutation
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172