Agenda for June 2025 Rare Disorders Advisory Committee meeting

Note the meeting date is now one day earlier than the date originally shared. This is to accommodate advisor availability. 

What we're doing

Pharmac is sharing what medicine applications will be considered at the Rare Disorders Advisory Committee meeting on Tuesday 10 June 2025.

We understand that people living with rare disorders face many challenges, including access to health care and effective medicines. Sharing the Rare Disorders Advisory Committee meeting agenda offers more transparency to those looking for updates on specific medicines.

The agenda for the meeting includes applications for treatments for paroxysmal nocturnal haemoglobinuria (PNH), hereditary angioedema (HAE), Fabry disease, and spinal muscular atrophy (SMA). It also includes a review of recent evidence for miglustat for Neimann Pick Type C.

The records from the Rare Disorders Advisory Committee meeting will be released later in the year. Funding applications will be reviewed using the Rare Disorders policy principles and will be assessed in line with Pharmac’s usual processes. 

We are working with the consumer group Rare Disorders New Zealand(external link) to make sure the right voices are heard at this meeting – we thank them for their time and expertise.

Applications

Pegunigalsidase alfa-iwxy for Fabry Disease

The Committee will discuss a new application for an Enzyme Replacement Therapy (ERT) for people with Fabry disease. Fabry disease is a neurological condition that can affect many parts of the body, including the kidneys, heart, and skin.

Pegunigalsidase alfa-iwxy (branded as Elfabrio) is another treatment option for this condition.  The Committee had previously recommended funding of agalsidase alfa, agalsidase beta and migalastat for Fabry disease.

Application for pegunigalsidase alfa-iwxj for Fabry disease(external link)

Previous considerations of agalsidase alfa(external link), agalsidase beta(external link) and migalastat(external link) for Fabry disease.

Onasemnogene abeparvovec for spinal muscular atrophy (SMA), pre-symptomatic or type 1

The Committee will rediscuss an application for onasemnogene abeparvovec for the treatment of pre-symptomatic or type 1 spinal muscular atrophy (SMA). SMA is a genetic disorder that causes muscle weakness and wasting due to the loss of nerve cells in the spinal cord.

Previously the Rare Disorders Advisory Committee deferred making a recommendation for onasemnogene abeparvovec, seeking long-term evidence on its efficacy and safety. The Committee will consider the latest data now available.

Application for onasemnogene abeparvovec for SMA(external link)

Garadacimab for hereditary angioedema (HAE)

The Committee will discuss a new application for people with hereditary angioedema (HAE). HAE is an inherited disorder that results in recurrent episodes of severe swelling (angioedema).

The Committee had previously recommended funding of another treatment for HAE, lanadelumab.

Application for garadacimab for HAE(external link)

Previous application for lanadelumab(external link)

Iptacopan, Eculizumab, and Crovalimab for paroxysmal nocturnal haemoglobinuria (PNH)

The Committee will discuss three applications for treatments for paroxysmal nocturnal haemoglobinuria (PNH) - iptacopan, eculizumab, and crovalimab.  PNH is a rare blood disorder in which red blood cells break apart prematurely. This can lead to anaemia, blood clots and other complications.

Pharmac has previously assessed eculizumab for PNH on several occasions. This is a new application which the Committee will consider in the context of rare disorders.

Application for iptacopan for PNH(external link)

New application for eculizumab for PNH(external link)

Previous considerations of eculizumab for PNH(external link)

Application for crovalimab for PNH(external link)

Matters Arising

Miglustat for Neimann Pick Type C

The Committee previously assessed miglustat for Neimann Pick Type C. New evidence was provided by a clinician, and so the Committee will consider if this changes their previous recommendation.

Previous consideration of Niemann Pick Type C(external link)

Advisory meeting agenda setting

The scheduling and agenda setting process for advisory meetings considers multiple factors. We aim to balance the relative priorities of:

• clinical advice needed across indications
• the factors for consideration for each application (for example unmet health need)
• the time since applications were received
• the internal and advisor resource available to support each meeting.

Applications received through the rare disorders call for applications that are not included on this agenda will be considered as soon as practicable via the most appropriate Advisory Committee for the application.