Cystic fibrosis patients in their second year of life now have subsidised access to lumacaftor with ivacaftor, with the federal government agreeing to an expanded PBS listing.

It comes after the Pharmaceutical Benefits Advisory Committee recommended increasing eligibility to the combination, branded Orkambi (Vertex), to include patients aged one to less than two years.

The disease must be homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, as per the listing under the Highly Specialised Drugs Program.

The PBAC said its backing came in spite of “limited” supporting evidence, but acknowledged this reflected the difficulties in obtaining efficacy data from paediatric patients.

Overall, it considered that the claim of superior efficacy over best supportive care in one-year-old patients was “biologically plausible and likely to be beneficial”.

Risk sharing arrangement financial caps for lumacaftor with ivacaftor should be increased to accommodate patients commencing treatment earlier, the committee added (link here).

About 30 infants and their families are expected to benefit from the expanded listing, without which they would pay around $232,300 per year of treatment, according to the government.

Other respiratory therapies supported by the PBAC

Meanwhile, the PBAC came in favour of further listings for CF therapy, as well as treatments for NSCLC and generic inhalers at its most recent meeting (link here).

Among the recommendations was for the listing for ivacaftor granules and tablets be extended to include the treatment of CF in patients aged four months and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data.

It also recommended that an additional strength of granules (ivacaftor 25 mg) should be made available for that population.

In NSCLC, two therapies were recommended for subsidy. The first of these was osimertinib for the treatment of Stage IB to IIIA EGFR mutation positive NSCLC as adjuvant therapy after surgical resection.

In its comments, it noted the drug provided a substantial benefit in terms of disease free survival compared to standard of care, although the magnitude of overall survival benefit remained uncertain.

The drug’s importer, AstraZeneca, would also need to reduce its asking price, the committee said.

Also recommended was cemiplimab to be used with platinum doublet chemotherapy as a first-line treatment of adult patients with Stage IV (metastatic) NSCLC with no evidence of epidermal growth factor receptor (EGFR), anaplastic lymphoma kinase (ALK), or c-ROS-proto-oncogene 1 (ROS1) aberrations.

Generic inhalers containing budesonide+formoterol (Bufomix Easyhaler) and fluticasone propionate with salmeterol (Salflumix Easyhaler) were also supported at the meeting.