Patients with cystic fibrosis, asthma and COPD all look set to benefit from new subsidised therapeutic options, following positive recommendations by the Pharmaceutical Benefits Advisory Committee.
The recommendations include an endorsement for the listing of elexacaftor/tezacaftor/ivacaftor (Trikafta) to be expanded to include patients aged two-to-five years.
Per the Section 100 (Highly Specialised Drugs Program) listing, eligible patients would also need to have at least one F508del mutation on the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
“This is fantastic news because we know that there is a significant benefit to commencing therapies early to prevent irreparable damage,” Cystic Fibrosis Australia said in response to the announcement.
“With this listing, 130 children will gain access to a modulator for the first time, while 290 children will have access to three modulator therapies.”
“This represents significant progress in ensuring universal access to necessary therapies.”
As part of its decision, the PBAC also recommended that two granule formulations should be made available under the program to facilitate dosing in children in the age group.
In an outcome statement following its March 2024 meeting (link here), it stressed the evidence presented could not accurately quantify the benefit of treating patients with ELX/TEZ/IVA from a younger age but acknowledged treatment from a young age was likely to be beneficial.
Green light for asthma treatments
Separately, the PBAC backed listings for a number of asthma treatments, with dupilumab getting the nod for patients aged 6 to 11 years of age with uncontrolled severe asthma despite optimised asthma therapy.
Eligibility criteria would include the patient having total serum immunoglobulin E (IgE) of ≥ 30 IU/mL and evidence of atopy, or blood eosinophils ≥ 150 x 109/L, or fractional exhaled nitric oxide ≥ 20 parts per billion.
For patients with IgE ≥ 30 IU/ mL and evidence of atopy, the claim of non-inferior effectiveness and safety compared to omalizumab was “likely reasonable”, the committee said in its decision.
It added: “For patients who had IgE < 30 IU/ mL and no evidence of atopy the PBAC took into consideration that while the clinical evidence presented was limited, there was a clinical need, and that the number of patients and cost was expected to be small.”
Branded Dupixent, the agent would be administered via injection in either 200mg or 300mg dosages, via single dose pre-filled syringe.
Meanwhile, the committee came out in favour of listing the fixed dose combination (FDC) of beclometasone 100 µg with formoterol 6 µg (BEC/FOR 100/6) as maintenance and reliever treatment for asthma.
And the PBAC supported amending the clinical criteria of mepolizumab for the treatment of uncontrolled severe asthma to remove the oral corticosteroids (OCS) requirement within the definition of ‘optimised asthma therapy’ to align with current treatment guidelines.
However, the committee did not recommend changing the authority level of the agent, saying that, while the available data suggested that the growth of mepolizumab was gradually stabilising, the overall uncontrolled severe asthma market continued to grow.
“The PBAC therefore considered that it would not be appropriate to amend the circumstances of mepolizumab in isolation, noting the growth of the overall anti-interleukin-5 market for uncontrolled severe asthma,” it said.
Inhaler product gets the nod for COPD
Finally, the PBAC recommended the listing of a new product containing tiotropium, Tiotropium Lupin for use with LupinHaler as an alternative to the currently PBS-listed Spiriva for use with Handihaler for the treatment of COPD.
As with its competitor products, the therapy would be listed as a restricted benefit on the general schedule.