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Biogen drug in parliamentary appeal
Posted 26 May 2017
Biogen's Spinraza (nusinersen), yet to come to market but eagerly awaited by spinal muscular atrophy (SMA) patients and their supporters has been the subject of an appeal for quick access in federal parliament.
Labor MP for Holt, Anthony Byrne, told parliament this week that the drug is the first potential genetic treatment option for sufferers of this often, fatal disease, and that SMA is the number one genetic killer of infants under the age of three.
"This drug gives hope for those families who have no other options for treatment," Mr Byrne said.
He added that a constituent, who asked him to bring the matter to the attention of the House of Representatives, hopes this drug will proceed through the testing process of the TGA and the PBAC.
Biogen is already seeking TGA approval for the breakthrough treatment, which was approved by the FDA in late December 2016 for paediatric and adult patients, while Sydney Children's Hospitals Network (SCHN) is offering SMA type 1 patients free access to the novel therapy as part of an Expanded Access Program (EAP).
Biogen Australia and New Zealand also said it would will be seeking PBS reimbursement.
"We recognise the significant need in the SMA community and hope to bring a treatment for SMA to Australian patients, as soon as possible," the company said.
Reports from the US suggest that Spinraza will cost US$125,000 per injection, $US750,000 in the first year and $US375,000 thereafter, which a Biogen US spokesperson has said is "in line with the annual cost of other rare disease therapies".
In early May, the Sydney Children's Hospitals Network (SCHN) said that nusinersen is showing improvements in strength and motor function in babies and children with SMA types 1 and 2.
"Several SMA Type 1 patients have already been treated with nusinersen at Sydney Children's Hospital, Randwick and The Children's Hospital at Westmead with clinicians seeing promising results," SCHN said.
Biogen US said at its first quarter 2017 results late April that it had executed a strong launch of Spinraza with $US47 million in sales.
Warren Beaumont
warren.beaumont@lushmedia.com.au
Posted 26 May 2017
Biogen's Spinraza (nusinersen), yet to come to market but eagerly awaited by spinal muscular atrophy (SMA) patients and their supporters has been the subject of an appeal for quick access in federal parliament.
Labor MP for Holt, Anthony Byrne, told parliament this week that the drug is the first potential genetic treatment option for sufferers of this often, fatal disease, and that SMA is the number one genetic killer of infants under the age of three.
"This drug gives hope for those families who have no other options for treatment," Mr Byrne said.
He added that a constituent, who asked him to bring the matter to the attention of the House of Representatives, hopes this drug will proceed through the testing process of the TGA and the PBAC.
Biogen is already seeking TGA approval for the breakthrough treatment, which was approved by the FDA in late December 2016 for paediatric and adult patients, while Sydney Children's Hospitals Network (SCHN) is offering SMA type 1 patients free access to the novel therapy as part of an Expanded Access Program (EAP).
Biogen Australia and New Zealand also said it would will be seeking PBS reimbursement.
"We recognise the significant need in the SMA community and hope to bring a treatment for SMA to Australian patients, as soon as possible," the company said.
Reports from the US suggest that Spinraza will cost US$125,000 per injection, $US750,000 in the first year and $US375,000 thereafter, which a Biogen US spokesperson has said is "in line with the annual cost of other rare disease therapies".
In early May, the Sydney Children's Hospitals Network (SCHN) said that nusinersen is showing improvements in strength and motor function in babies and children with SMA types 1 and 2.
"Several SMA Type 1 patients have already been treated with nusinersen at Sydney Children's Hospital, Randwick and The Children's Hospital at Westmead with clinicians seeing promising results," SCHN said.
Biogen US said at its first quarter 2017 results late April that it had executed a strong launch of Spinraza with $US47 million in sales.
Warren Beaumont
warren.beaumont@lushmedia.com.au
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