Early Access Program for Nusinersen


There has been a lot of activity in the clinical trial space for the treatment of Spinal Muscular Atrophy (SMA) over the last two years, and Nusinersen, has been identified as the first effective drug to treat SMA Type 1.


Developed in California, Nusinersen (know in the USA as Spinraza) is producing promising results, with 40 per cent of babies in a recent blinded trial of various SMA types reaching milestones such as sitting, crawling and walking.


Nusinersen works by enabling backup gene -SMN2- to produce increased quantities of SMN protein. It is injected into the spinal fluid in a manner similar to receiving an epidural anaesthetic, with patients likely to require these injections several times a year for life. Initial treatment is four times over the first two months, with maintenance therapy every four months.


Nusinersen is not yet licensed in Australia, however at the Royal Children’s Hospital, we are establishing a new Extended Access Program (EAP) in the interim. The EAP will enable SMA type 1 patients to access Nusinersen prior to the Australian Therapeutic Goods Administration approval. Approval for the EAP is expected within the next 6 weeks.


Due to the intensive nature of the SMA Extended Access Program including the complexities of administering the drug to patients, managing the number of treatments involved and the careful sedation and monitoring required, this is a challenging process for us.


Following the establishment of the SMA type 1 program, our intention is to provide an access program for Nusinersen for a subpopulation of children with SMA type 2. SMA type 2 patients will become eligible for the program based on medical needs and clinical severity.


Possible cases of SMA1 should be referred urgently to the neuromuscular clinics in each state; contact numbers are below.


Children’s Health Queensland (Lady Cilento Children’s Hospital):

Kate Munro, Clinical Nurse Consultant Neurology

Phone: 3068 2880 email: [email protected]


Royal Children’s Hospital Melbourne: Daniella Villano, Neuromuscular Nurse Coordinator. Phone 9345 4633, email: [email protected]


Sydney Children’s Hospital Network:

Margot Morrison and Sandra Holland, Neuromuscular Nurse Consultant. Phone 0293821845 email: [email protected] and [email protected]


Perth Children’s Hospital:

Jodi Mann the PMH/PCH Neuromuscular Nurse Coordinator on 0893407186


Women’s and Children’s Hospital, Adelaide:

Mark Francis, Clinical Practice Consultant. Phone (08) 8161 7308 email: [email protected]