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FIRST TREATMENT FOR THE LEADING CAUSE OF GENETIC DEATH IN CHILDREN UNDER TWO NOW FUNDED1,2
From 1 January 2023 SPINRAZA® (nusinersen) will be funded by Pharmac for the treatment of paediatric patients (18 years and younger) with infantile-onset or childhood-onset of Spinal Muscular Atrophy (SMA) with onset of symptoms prior to three years of age and infants with pre-symptomatic SMA (3 copies SMN2 or less).1,3
•
SPINRAZA is a first-of-its-kind medicine for an often-deadly disease: SMA is the number one cause of genetic death in New Zealand in children under the age of two,2 and can have a life-changing impact on affected New Zealanders of all ages.4
•
With SMA, delays to diagnosis can have a significant impact, it must be recognised with urgency: Speed of diagnosis is crucial, as an early diagnosis may lead to early supportive care, reduction in disease and caregiver burden,4 and now treatment.3
•
SPINRAZA acts to address the underlying cause of SMA: SPINRAZA works to stimulate production of SMN protein,3 which is critical to the function of muscles that enable movement, speech, breathing and swallowing.5,6
8th December 2022: Biogen has today announced that SPINRAZA® (nusinersen) will be funded by Pharmac from 1 January 2023 for the treatment of paediatric patients (18 years and younger) with infantile-onset or childhood-onset of Spinal Muscular Atrophy (SMA) with onset of symptoms prior to three years of age and infants with pre-symptomatic SMA (3 copies of Survival Motor Neuron 2 [SMN2] or less).1,2 Up to 50 people could be eligible for funded treatment with nusinersen. The number of people receiving treatment is anticipated to increase over time, with up to four people each year diagnosed with SMA and be eligible for treatment. 3
SPINRAZA is the first medicine to be funded in New Zealand for SMA.1 The funding follows Medsafe registration of SPINRAZA in August 20184 and a positive recommendation from Pharmac’s Rare Diseases Sub-Committee and endorsement from Pharmacology and Therapeutics Advisory Committee (PTAC) in January 2020 following an initial submission by Biogen in August 2018.5
SMA is a rare genetic disease that can have a devastating and life-changing impact.6 In its most severe forms, affecting young babies and children, SMA can cause paralysis and difficulty with the most basic functions of life, like breathing and swallowing.6 Children with SMA may not hit major motor milestones other children without the disease will (i.e., rolling, sitting, crawling, standing or walking).7
“A diagnosis of SMA is devastating to give to any family. Whilst it is the most common genetic cause of death in infancy, it is a condition the majority of New Zealanders have never heard of. In the most severe form, a family won't see their baby live until their second birthday. Older children live a really tough life with severe muscle weakness and frequent contact with medical services.
“Many families have waited a very long time to be able to access Spinraza. It is a true breakthrough that Spinraza is now the first treatment to be made available to eligible New Zealanders with SMA. As a medical community, we are so excited to be able to celebrate with families, knowing the potential that a treatment for SMA has in changing their child’s future," said Dr Gina O’Grady, Paediatric Neurologist at Starship Child Health, Auckland.
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.6
SMA Type I is the most severe; affected babies have a life expectancy of less than two years.7 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.6 New Zealanders with SMA Type III, while diagnosed later at around 18 months of age, generally lose the ability to walk during childhood.7
“Today is a momentous occasion for those children and their whānau in New Zealand who have been waiting for access to this medicine for so long” said David Henderson, Managing Director, Biogen Australia and New Zealand.
“We have been steadfast in our efforts to reach this agreement and it’s a first for Biogen in this country, a first for New Zealand, and – mostly importantly – a first for those living with SMA in New Zealand who may benefit from this funding.
“It has been a long and sometimes turbulent journey since our first submission for funding in 2018 and the people at Biogen would like to extend their thanks and deep gratitude to those who have made this journey with us. Their dedication and commitment to the SMA community have greatly assisted in ensuring that this medicine is now available.
“We are aware that our work is not yet complete though, as we continue to work towards enabling broader access for those who can benefit from this medicine within the SMA community, including those with different SMA types and ages at symptom onset.”
SMA occurs when a fault in the Survival Motor Neuron 1 (SMN1) gene is passed on to a child by both parents.8 The SMN1 gene is responsible for the body’s production of survival motor neuron (SMN) protein,8 which is vital to the function of muscles that enable movement, speech, breathing and swallowing.6 Without adequate production of SMN protein, the nerves of the spinal cord deteriorate and muscle wasting gradually occurs.6,8
SPINRAZA, a treatment known as 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
One in 35 people of all ethnicities unknowingly carries the faulty SMN1 gene. If a couple already has a child with the disease, each of their subsequent children has a one in four chance of inheriting SMA.8 Parents of children with SMA are usually unaware they both carry the faulty genes and often have not heard of SMA until their child has been diagnosed.8
The red flags (signs and symptoms) of SMA include:8
•
A noticeable weakness or reduced function in limbs
•
A loss of, or a reduction of tendon reflexes in the legs, and weakness in both sides of the body (especially the legs)
•
Fine twitches of the muscles in the limbs
•
Tremor (fasciculation) of the tongue.
Biogen is committed to continued investment in research and development to find new ways to change the lives of underserved populations and those with rare diseases.
ENDS
For further information, please contact:
•
Fiona Tigar / fiona.tigar@biogen.com /: M: +61 400 741 286
•
Eliza Roel / eliza.roel@biogen.com / M: +61 437 819 421
To hear from the community, we suggest contacting long-term campaigner for access to treatment and SMA patient Fiona Tolich via patientvoicenz@gmail.com. She leads the SMA advocacy efforts and is a Trustee of Patient Voice Aotearoa.
SPINRAZA Data Sheet: https://www.medsafe.govt.nz/profs/Datasheet/s/spinrazainj.pdf
SPINRAZA Consumer Medicine Information: https://www.medsafe.govt.nz/Consumers/CMI/s/spinraza.pdf
About Biogen
As pioneers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has a leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and developed the first and only treatment approved by the FDA to address a defining pathology of Alzheimer’s disease. Biogen is also commercialising biosimilars and focusing on advancing one of the industry’s most diversified pipelines in neuroscience that will transform the standard of care for patients in several areas of high unmet need.
For more information about Biogen please visit: www.biogen.co.nz.
BEFORE PRESCRIBING, PLEASE REVIEW APPROVED DATA SHEET AVAILABLE AT https://www.medsafe.govt.nz/profs/Datasheet/s/spinrazainj.pdf
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 https://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.
From Jan 1, 2023, SPINRAZA is funded on the Pharmaceutical Schedule – a prescription charge, pharmacy charge and special authority criteria will apply
Biogen, Auckland.
NAME AND ADDRESS OF SPONSOR:
Australia: Biogen Australia Pty Ltd, Level 4, 2 Banfield Road, Macquarie Park NSW 2113
New Zealand: Biogen NZ Biopharma Ltd, Office 152, 155 Fanshawe Street, Auckland, 1010
SPINRAZA® is a registered trademark of Biogen Australia Pty Ltd. ABN: 30 095 760 115.Level 4, 2 Banfield Road, North Ryde, NSW 2113.
References:
1
Pharmac. News and Resources. Available at: https://pharmac.govt.nz/news-and-resources/news/
2
Biogen. SPINRAZA (nusinersen) Data Sheet. July 2020. Available at: https://www.medsafe.govt.nz/profs/Datasheet/s/spinrazainj.pdf
3
Pharmac. News and Resources. (28 September 2022). Proposal to fund nusinersen (Spinraza) for spinal muscular atrophy. Available at: https://pharmac.govt.nz/news-and-resources/consultations-and-decisions/2022-09-28-proposal-to-fund-nusinersen-spinraza-for-spinal-muscular-atrophy/.
4
Medsafe. Medsafe Product Detail. 23 August 2018. Available at: https://www.medsafe.govt.nz/regulatory/ProductDetail.asp?ID=19619
5
Pharmac. Pharmac application tracker. Available at: https://connect.pharmac.govt.nz/apptracker/s/application-public/a0R2P000000alfkUAA/nusinersen-spinraza
6
Lin C-W et al. (2015). Delay in Diagnosis of Spinal Muscular Atrophy: A Systematic Literature Review. Pediatric Neurology 53; 293e300.
7
Rossoll W et al. (2009). Spinal Muscular Atrophy and a Model for Survival of Motor Neuron Protein Function in Axonal Ribonucleoprotein Complexes. Results Probl Cell Differ. 48: 289–326. doi:10.1007/400_2009_4.
8
National Institute of Health. SMN1 gene. Available at: https://ghr.nlm.nih.gov/gene/SMN1#conditions.
From 1 January 2023 SPINRAZA® (nusinersen) will be funded by Pharmac for the treatment of paediatric patients (18 years and younger) with infantile-onset or childhood-onset of Spinal Muscular Atrophy (SMA) with onset of symptoms prior to three years of age and infants with pre-symptomatic SMA (3 copies SMN2 or less).1,3
| • | SPINRAZA is a first-of-its-kind medicine for an often-deadly disease: SMA is the number one cause of genetic death in New Zealand in children under the age of two,2 and can have a life-changing impact on affected New Zealanders of all ages.4 |
| • | With SMA, delays to diagnosis can have a significant impact, it must be recognised with urgency: Speed of diagnosis is crucial, as an early diagnosis may lead to early supportive care, reduction in disease and caregiver burden,4 and now treatment.3 |
| • | SPINRAZA acts to address the underlying cause of SMA: SPINRAZA works to stimulate production of SMN protein,3 which is critical to the function of muscles that enable movement, speech, breathing and swallowing.5,6 |
8th December 2022: Biogen has today announced that SPINRAZA® (nusinersen) will be funded by Pharmac from 1 January 2023 for the treatment of paediatric patients (18 years and younger) with infantile-onset or childhood-onset of Spinal Muscular Atrophy (SMA) with onset of symptoms prior to three years of age and infants with pre-symptomatic SMA (3 copies of Survival Motor Neuron 2 [SMN2] or less).1,2 Up to 50 people could be eligible for funded treatment with nusinersen. The number of people receiving treatment is anticipated to increase over time, with up to four people each year diagnosed with SMA and be eligible for treatment. 3
SPINRAZA is the first medicine to be funded in New Zealand for SMA.1 The funding follows Medsafe registration of SPINRAZA in August 20184 and a positive recommendation from Pharmac’s Rare Diseases Sub-Committee and endorsement from Pharmacology and Therapeutics Advisory Committee (PTAC) in January 2020 following an initial submission by Biogen in August 2018.5
SMA is a rare genetic disease that can have a devastating and life-changing impact.6 In its most severe forms, affecting young babies and children, SMA can cause paralysis and difficulty with the most basic functions of life, like breathing and swallowing.6 Children with SMA may not hit major motor milestones other children without the disease will (i.e., rolling, sitting, crawling, standing or walking).7
“A diagnosis of SMA is devastating to give to any family. Whilst it is the most common genetic cause of death in infancy, it is a condition the majority of New Zealanders have never heard of. In the most severe form, a family won't see their baby live until their second birthday. Older children live a really tough life with severe muscle weakness and frequent contact with medical services.
“Many families have waited a very long time to be able to access Spinraza. It is a true breakthrough that Spinraza is now the first treatment to be made available to eligible New Zealanders with SMA. As a medical community, we are so excited to be able to celebrate with families, knowing the potential that a treatment for SMA has in changing their child’s future," said Dr Gina O’Grady, Paediatric Neurologist at Starship Child Health, Auckland.
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.6
SMA Type I is the most severe; affected babies have a life expectancy of less than two years.7 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.6 New Zealanders with SMA Type III, while diagnosed later at around 18 months of age, generally lose the ability to walk during childhood.7
“Today is a momentous occasion for those children and their whānau in New Zealand who have been waiting for access to this medicine for so long” said David Henderson, Managing Director, Biogen Australia and New Zealand.
“We have been steadfast in our efforts to reach this agreement and it’s a first for Biogen in this country, a first for New Zealand, and – mostly importantly – a first for those living with SMA in New Zealand who may benefit from this funding.
“It has been a long and sometimes turbulent journey since our first submission for funding in 2018 and the people at Biogen would like to extend their thanks and deep gratitude to those who have made this journey with us. Their dedication and commitment to the SMA community have greatly assisted in ensuring that this medicine is now available.
“We are aware that our work is not yet complete though, as we continue to work towards enabling broader access for those who can benefit from this medicine within the SMA community, including those with different SMA types and ages at symptom onset.”
SMA occurs when a fault in the Survival Motor Neuron 1 (SMN1) gene is passed on to a child by both parents.8 The SMN1 gene is responsible for the body’s production of survival motor neuron (SMN) protein,8 which is vital to the function of muscles that enable movement, speech, breathing and swallowing.6 Without adequate production of SMN protein, the nerves of the spinal cord deteriorate and muscle wasting gradually occurs.6,8
SPINRAZA, a treatment known as 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
One in 35 people of all ethnicities unknowingly carries the faulty SMN1 gene. If a couple already has a child with the disease, each of their subsequent children has a one in four chance of inheriting SMA.8 Parents of children with SMA are usually unaware they both carry the faulty genes and often have not heard of SMA until their child has been diagnosed.8
The red flags (signs and symptoms) of SMA include:8
| • | A noticeable weakness or reduced function in limbs |
| • | A loss of, or a reduction of tendon reflexes in the legs, and weakness in both sides of the body (especially the legs) |
| • | Fine twitches of the muscles in the limbs |
| • | Tremor (fasciculation) of the tongue. |
Biogen is committed to continued investment in research and development to find new ways to change the lives of underserved populations and those with rare diseases.
ENDS
For further information, please contact:
| • | Fiona Tigar / fiona.tigar@biogen.com /: M: +61 400 741 286 |
| • | Eliza Roel / eliza.roel@biogen.com / M: +61 437 819 421 |
To hear from the community, we suggest contacting long-term campaigner for access to treatment and SMA patient Fiona Tolich via patientvoicenz@gmail.com. She leads the SMA advocacy efforts and is a Trustee of Patient Voice Aotearoa.
SPINRAZA Data Sheet: https://www.medsafe.govt.nz/profs/Datasheet/s/spinrazainj.pdf
SPINRAZA Consumer Medicine Information: https://www.medsafe.govt.nz/Consumers/CMI/s/spinraza.pdf
About Biogen
As pioneers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has a leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and developed the first and only treatment approved by the FDA to address a defining pathology of Alzheimer’s disease. Biogen is also commercialising biosimilars and focusing on advancing one of the industry’s most diversified pipelines in neuroscience that will transform the standard of care for patients in several areas of high unmet need.
For more information about Biogen please visit: www.biogen.co.nz.
BEFORE PRESCRIBING, PLEASE REVIEW APPROVED DATA SHEET AVAILABLE AT https://www.medsafe.govt.nz/profs/Datasheet/s/spinrazainj.pdf
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 https://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.
From Jan 1, 2023, SPINRAZA is funded on the Pharmaceutical Schedule – a prescription charge, pharmacy charge and special authority criteria will apply
Biogen, Auckland.
NAME AND ADDRESS OF SPONSOR:
Australia: Biogen Australia Pty Ltd, Level 4, 2 Banfield Road, Macquarie Park NSW 2113
New Zealand: Biogen NZ Biopharma Ltd, Office 152, 155 Fanshawe Street, Auckland, 1010
SPINRAZA® is a registered trademark of Biogen Australia Pty Ltd. ABN: 30 095 760 115.Level 4, 2 Banfield Road, North Ryde, NSW 2113.
References:
| 1 | Pharmac. News and Resources. Available at: https://pharmac.govt.nz/news-and-resources/news/ | |
| 2 | Biogen. SPINRAZA (nusinersen) Data Sheet. July 2020. Available at: https://www.medsafe.govt.nz/profs/Datasheet/s/spinrazainj.pdf | |
| 3 | Pharmac. News and Resources. (28 September 2022). Proposal to fund nusinersen (Spinraza) for spinal muscular atrophy. Available at: https://pharmac.govt.nz/news-and-resources/consultations-and-decisions/2022-09-28-proposal-to-fund-nusinersen-spinraza-for-spinal-muscular-atrophy/. | |
| 4 | Medsafe. Medsafe Product Detail. 23 August 2018. Available at: https://www.medsafe.govt.nz/regulatory/ProductDetail.asp?ID=19619 | |
| 5 | Pharmac. Pharmac application tracker. Available at: https://connect.pharmac.govt.nz/apptracker/s/application-public/a0R2P000000alfkUAA/nusinersen-spinraza | |
| 6 | Lin C-W et al. (2015). Delay in Diagnosis of Spinal Muscular Atrophy: A Systematic Literature Review. Pediatric Neurology 53; 293e300. | |
| 7 | Rossoll W et al. (2009). Spinal Muscular Atrophy and a Model for Survival of Motor Neuron Protein Function in Axonal Ribonucleoprotein Complexes. Results Probl Cell Differ. 48: 289–326. doi:10.1007/400_2009_4. | |
| 8 | National Institute of Health. SMN1 gene. Available at: https://ghr.nlm.nih.gov/gene/SMN1#conditions. |