Treatment for Spinal Muscular Atrophy Now Registered

First Treatment for Spinal Muscular Atrophy Now Registered in New Zealand
SPINRAZA® (nusinersen) was Gazetted on August 23, 2018, by Medsafe for the treatment of 5q spinal muscular atrophy (SMA).1,2
• New treatment a first of its kind for devastating disease: SMA is the most common genetic cause of death in New Zealand in children under the age of two1 and can have a life-changing impact on affected New Zealanders of all ages.2
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• SMA needs to be recognised with urgency: Speed of diagnosis is crucial, as an early diagnosis may lead to early supportive care, reduction in patient/caregiver burden,4 and now treatment.2
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• SPINRAZA acts to address genetic deficiency: SPINRAZA can regulate SMN gene expression and stimulate SMN protein production2 which is critical to the function of muscles that enable movement, speech, breathing and swallowing.3,4
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August 24, 2018: Biogen has announced today that SPINRAZA® (nusinersen) has been registered by Medsafe for the treatment of 5q SMA.1,2

SPINRAZA is the first treatment registered in New Zealand for the treatment of SMA1,2 – a disease which can have a devastating and life-changing impact on affected New Zealanders of all ages.4 5q SMA is the most common form of the disease and represents approximately 95% of all SMA cases.6

There are four known forms of SMA – Type I, Type II, Type III and Type IV – each determined by the age of onset (from those diagnosed before six months of age to those diagnosed in adulthood) and the physical milestones achieved.4

Whilst rare, SMA can have a life-changing impact, particularly when it occurs in young babies and children.4 In its most severe forms, SMA can cause paralysis and difficulty with the most basic functions of life, like breathing and swallowing.4,5 Children with SMA may not hit major motor milestones other children without the disease will (i.e. rolling, sitting, crawling, standing or walking).2

“This is a positive and exciting step towards a life-saving treatment for New Zealanders with SMA” commented Ronelle Baker, Chief Executive Officer of Muscular Dystrophy Association New Zealand “and is particularly significant given we are in SMA Awareness Month”.

SMA occurs when a faulty SMN1 gene is passed on to a child by both parents.5 The SMN1 gene is responsible for the body’s production of SMN protein,5 which is vital to the function of muscles that enable movement, speech, breathing and swallowing.5 Without ample production of SMN protein, the nerves of the spinal cord deteriorate and muscle wasting gradually occurs.5,6

SPINRAZA, an antisense oligonucleotide (ASO), is administered by lumbar puncture directly into the cerebrospinal fluid (the fluid surrounding the spinal cord) to regulate SMN gene expression and stimulate SMN protein production.2

SMA Type I is the most severe; affected babies have a life expectancy of less than two years.3 Those with SMA Type II are usually diagnosed between the ages of six months and two years, and are never able to stand, walk or live independently.4 New Zealanders with SMA Type III, while diagnosed later at around 18 months of age, generally lose the ability to walk during childhood.4

With SPINRAZA already approved in over 19 countries including Australia where 80 patients are already receiving the product following government reimbursement on 1st June 2018, Biogen is committed to working with PHARMAC to ensure SPINRAZA is subsidised as soon as possible.

In the meantime, and in response to the urgent need for treatment, Biogen has made SPINRAZA available to New Zealanders living with SMA Type 1 through an expanded access program. This program was set up in collaboration with the Starship Children's Hospital in Auckland. A decision as to whether SPINRAZA is the right treatment for the patient should be made in consultation with the treating doctor.

“SPINRAZA has received a very speedy Medsafe review and registration which we really appreciate, as we believe this product needs to be made available to patients as soon as is practicably possible. We will now work closely with PHARMAC, the Government and healthcare professionals and hope to ensure that those New Zealanders who could benefit from SPINRAZA, do,” said Christopher Stemple, Managing Director, Biogen Australia and New Zealand.

One in thirty-five people unknowingly carry the faulty SMN1 gene.6 If a couple already has a child with the diease, each of their subsequent children has a one-in-four chance of inheriting SMA.6 Parents of children with SMA are usually unaware they carry the faulty genes and often have not heard of SMA until their child has been diagnosed.6

ENDS

SPINRAZA (nusinersen) 12mg/5mL solution for injection is a Prescription Medicine for patients with 5q Spinal Muscular Atrophy (SMA). SPINRAZA has risks and benefits. For product information check Consumer Medicine Information on www.medsafe.govt.nz. Ask your doctor if SPINRAZA is right for you. Treatment with SPINRAZA should be initiated and supervised by specialist doctors experienced in the diagnosis and management of SMA. SPINRAZA is given as an injection into the lower back known as a lumbar puncture by a trained health care professional. If symptoms persist or you have side effects see your doctor. SPINRAZA is unfunded medicine- a medicine charge, pharmacy charge and special authority criteria will apply. Normal doctors’ charges apply. Biogen, Auckland.

About Biogen

At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp, and today has the leading portfolio of medicines to treat multiple sclerosis; has introduced the first and only approved treatment for spinal muscular atrophy; and is focused on advancing neuroscience research programs in Alzheimer’s disease and dementia, multiple sclerosis and neuroimmunology, movement disorders, neuromuscular disorders, pain, ophthalmology, neuropsychiatry, and acute neurology.

About Muscular Dystrophy Association of New Zealand

The Muscular Dystrophy Association of New Zealand Inc. (MDANZ) began as a support group for families affected by muscular dystrophy. Since its inception MDANZ has broadened its scope to include many other neuromuscular conditions.
The MDANZ logo is a person shown in the form of DNA. This double helix represents the genetic component to many neuromuscular conditions and reflects the organisations commitment to families and the acknowledgement of whakapapa or family histories, which are woven through the stories of members. The unique governance structure of MDANZ ensures leadership of the organisation by individuals or family members with lived experience of a neuromuscular condition.

PHARMAC INFORMATION: THIS PRODUCT IS NOT LISTED ON PHARMAC

BEFORE PRESCRIBING, PLEASE REVIEW APPROVED DATA SHEET ATTACHED.

PREPARATION DATE – August 2018.

Date of Gazette publication: 23 August 2018

SPINRAZA® is a registered trademark of Biogen NZ Biopharma Limited,

References

1New Zealand Gazette. New Zealand Government. Consent to the Distribution of New Medicines. 23 August 2018. https://gazette.govt.nz/notice/id/2018-go4102.
2SPINRAZA (nusinersen) Data Sheet. Biogen. August 2018.
3Rossoll W et al. Spinal Muscular Atrophy and a Model for Survival of Motor Neuron Protein Function in Axonal Ribonucleoprotein Complexes. Results Probl Cell Differ. 2009 ; 48: 289–326. doi:10.1007/400_2009_4.

4Lin C-W et al. Delay in Diagnosis of Spinal Muscular Atrophy: A Systematic Literature Review. Pediatric Neurology 53 (2015) 293e300.

5National Institute of Health. SMN1 gene. [Online] Available at: https://ghr.nlm.nih.gov/gene/SMN1#conditions, last accessed 21 August 2018.

6Farrar MA, Kiernan MC. The Genetics of Spinal Muscular Atrophy: Progress and Challenges. Neurotherapeutics; 2015; 12:290–302.

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