MigalastatGalafold
Recommended Rare diseaseAuthority RequiredFirst-line line 💬 consumer voice
Treatment of Fabry disease in patients aged 12 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation.
5
Submissions
4 resub
2017–24
On the record
—
ICER range
Cost-min
Cost basis
Decisions on record
- Meeting Mar 2025 Recommended Fabry disease no PSD
- Meeting May 2024 Recommended Fabry disease no PSD
- Meeting Mar 2024 Recommended Fabry disease
- Meeting Dec 2022 Recommended Fabry disease in patients aged 16 years and older who have an amenable mutation
- Meeting Nov 2017 Not recommended Indicated for long- term treatment of adult and adolescent patients 16 years and older with a confirmed diagnosis of Fabry disease (α- galactosidase A deficiency) and who have an amenable mutation.
- Meeting Jul 2017 Not recommended Fabry disease
- Meeting Mar 2017 Deferred Fabry disease
Access path
- TGA registered · Galafold
TGA label narrower than the PBS population
- Mar 2017Deferred
vs enzyme replacement therapies (agalsidase alfa and…
- Jul 2017Not recommended
uncertain clinical claim of non-inferior effectiveness versus ERT, concerns over nature and patient relevance of…
- Nov 2017Not recommended
uncertain clinical claim of non-inferior comparative effectiveness compared with ERT, cost-minimisation approach not…
- ↻ resubmittedDec 2022Recommended · restricted
- May 2024Recommended
- PBS listing · Authority Required
RecommendedDeferredNot recommended
From the public summary
PBAC outcome
The PBAC recommended the listing of migalastat for the treatment of Fabry disease in patients aged 12 years of age and older who have an amenable mutation and evidence of organ involvement/injury (including severe gastrointestinal symptoms, and uncontrolled chronic pain, renal disease, cardiac disease, ischaemic and cerebrovascular disease).PSD · Mar 2024
The PBAC noted the strong consumer support for adding migalastat to the PBS and the importance of continuity of access. The PBAC reiterated the importance of patients with Fabry disease having ongoing access to effective therapies.PSD · Mar 2024
Economic analysis
The PBAC previously considered that migalastat would be appropriate for PBS listing at a price that ensured it was less expensive than the lowest cost available ERT on the LSDP for Fabry disease for all patients aged 16 years of age and older, and that this could be achieved with a cost per patient per year no higher than the cost of ERT for a patient weighing 45 kg (paragraph 5.6, migalastat PSD, December 2022 PBAC meeting).PSD · Mar 2024
The submission proposed a % price reduction from the current LSDP list price, but claimed that with the proposed RSA, a % price reduction will be achieved if the Tier 2 cap was reached with the proposed restriction. This is discussed further in paragraphsPSD · Mar 2024
Clinical claim
The submission described migalastat as non-inferior in terms of effectiveness compared to ERT. The evaluation considered this claim was not adequately supported. The key issues were:PSD · Mar 2024
• No new comparative evidence was provided in the submission to support the claim of non-inferiority. The clinical claim was based on two head-to-head trials (ATTRACT and FACETS). However, both studies have been previously considered by the PBAC and the Committee previously found the available evidence for migalastat insufficient to support a claim of non-inferior effectiveness compared to ERT;PSD · Mar 2024
Consumer comments
The health care professionals also commented on the benefits of oral treatment (versus infusion), but noted the education required relating to administration outside of food consumption and the need for regular clinician oversight.PSD · Mar 2024
The comments from individuals to support migalastat listing were received from Fabry disease patients (both current migalastat patients and those who would like to access the medicine) and family members/carers. In addition to the comments provided by health care professionals, individual comments focused on ability to travel and access in rural/regional areas.PSD · Mar 2024
Cost-effectiveness
Cost-minimisation approach used; no ICER calculated. Previous submissions (March 2017 onwards) used cost-minimisation with equi-effective dosing assumptions rather than ICER modelling.
The PBAC recalled that, at its March 2017 meeting, it considered that with the currently available evidence in treatment naïve patients and in treatment experienced or switch patients, it was reasonable to accept the claim of non-inferior comparative safety of migalastat compared to ERT. PBAC · 2017
ICER uncertainEconomic model disputed ×3Price cut / RSA needed
Decision context
PopulationAdult and adolescent patients aged 12 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and a documented migalastat amenable GLA gene variant.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| May 2024 | Recommended | enzyme replacement therapy (ERT) | — | RCT · Non-inferiority |
| Dec 2022 | Recommended · restricted | enzyme replacement therapy (ERT) | — | RCT · Surrogate |
| Nov 2017 | Not recommended | enzyme replacement therapy (ERT) | — | RCT · Surrogate |
| Jul 2017 | Not recommended | agalsidase alfa and agalsidase beta (enzyme replacement therapy) | — | RCT · Surrogate |
| Mar 2017 | Deferred | enzyme replacement therapies (agalsidase alfa and agalsidase beta) | — | RCT · Surrogate |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| ATTRACT | Ph 3 | 68 | Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate | completed |
| FACETS | Ph 3 | 67 | Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The A… | completed |
| NCT02194985 | Ph 3 | 84 | Number Of Participants Experiencing Adverse Events (AEs) | completed |
| AT1001-041 | Ph 3 | 85 | Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | terminated |
Consumer voice
Consumers and clinicians highlighted that migalastat offers significant quality-of-life benefits by providing oral tablet treatment compared to fortnightly intravenous infusions, reducing physical and time burdens while maintaining appropriate patient selection and safety.
described the benefits of treatment such as being a tablet compared to the current treatments that result in avoiding the physical and time burden of fortnightly intravenous infusion, which would improve people's quality of life. Consumer comments · PSD
treatment burdenquality of lifeunmet needaccess barriersclinical efficacy
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to first-line use and requires documented migalastat amenable GLA gene variant; TGA label does not specify treatment line.