Information sheet for ivacaftor (Kalydeco)
PHARMAC has announced a decision to fund ivacaftor (Kalydeco) tablets and granules for the
treatment of patients with cystic fibrosis with the G551D mutation (or other class III gating mutations).
Ivacaftor will be listed on the Pharmaceutical Schedule as soon as practicable following Medsafe
In the meantime, from 1 March 2020 PHARMAC will consider applications for ivacaftor (Kalydeco)
under the Exceptional Circumstances (EC) Framework for individual patients who meet the following
Applications from respiratory specialist or paediatrician.
Approvals valid without renewal unless notified for applications meeting the following criteria:
All of the following:
1. Patient has been diagnosed with cystic fibrosis; and
2.1. Patient must have G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)
gene on at least 1 allele; or
2.2. Patient must have other gating (class III (G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N
and S549R)) mutation in the CFTR gene on at least 1 allele; and
s must have a sweat chloride value of at least 60 mmol/L by quantitative pilocarpine iontophoresis or
by Macroduct sweat collection system; and
4. Treatment with ivacaftor must be given concomitantly with standard therapy for this condition; and
5. Patient must not have an acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
therapy (including antibiotics) for pulmonary disease in the last 4 weeks prior to commencing treatment with
6. The dose of ivacaftor will not exceed one tablet or one sachet twice daily; and
7. Applicant has experience and expertise in the management of cystic fibrosis.
will remain in place until such time as ivacaftor is listed on the Pharmaceutical Schedule
(following regulatory approval).
It is important to note that prior to regulatory approval, the supply, sale and prescribing of ivacaftor tablets
and granules will need to meet the requirements in Section 25 and 29 of the Medicines Act 1981.
Information about the use of unapproved medicines and the obligations of the prescriber for use of an
unapproved medicine can be found on the Medsafe website
Following Medsafe approval, ivacaftor will be listed as a PCT only-Specialist pharmaceutical in Section
B of the Pharmaceutical Schedule, meaning that only DHB hospitals will be able to make subsidy claims.
Therefore, we will only consider EC applications for dispensing through a DHB hospital pharmacy.
Any EC approvals will be converted to standard Special Authority approvals following listing on the
Schedule and applicants will be advised of the new approval number.