DRUG BLOW FOR BRAVE STEPH
FAMILY DEVASTATED $1M DOSE LEFT OFF SUBSIDY LIST
A FAMILY’s Christmas wish for their daughter to receive life-saving treatment has been shattered by a decision not to recommend the subsidy of a $1 million drug.
Amanda and Mark Nave publicly appealed for the drug Spinraza (nusinersen) to be listed on the Pharmaceutical Benefits Sceheme after it was approved by the Therapeutic Goods Administration in No- vember. Their daughter Stephanie, 2, has a severe form of degenerative motor neurone disease Spinal Muscular Atrophy Type II. The bubbly toddler was diagnosed with the genetic condition at 17 months of age and has been given a “grave” prognosis by doctors.
SMA occurs when a faulty SMN1 gene that fails to produce protein is passed down. The faulty gene causes the deterioration of spinal cord nerves and muscle wasting.
The Spinraza spinal cord in- jection, given every four months after an initial loading period, aims to replace missing motor neuron protein that is critical for the nerves that control muscles.
Yesterday the Pharmaceutical Benefits Advisory Committee released its decision not to recommend the listing of Spinraza for the treatment of patients with infantileonset (Type I) and childhood-onset (Types II and III) spinal muscular atrophy.
While the committee recog- nised a “high and urgent” clinical need for treatment of SMA it said “uncertainty about the clinical effectiveness of nusinersen” contributed to its decision.
Dr Nave said Stephanie is one of about 10 children in Australia with the most severe form of type II SMA whose lives depends of the drug. Children with the more severe type I SMA are currently receiving the drug through drug company Biogen.
“This drug needs to be pub- licly listed as a matter of urgency. For children like Stephanie access to this drug is the difference between life and death,” Dr Nave said.
Spinraza’s current listed price is $1 million for a 12month dose. Subsequent doses are priced at $500,000, Dr Nave said.
An estimated eight Australians are diagnosed with SMA Type II each year.
In a statement, SMA Australia chief executive officer Julie Cini said the decision to give Spinraza “not a positive recommendation” for the PBS was “devastating news for the entire SMA community here in Australia”.
PBAC has requested more detail about Australia’s patient population.
“SMA Australia will be highlighting our dissapointment in this decision to the health minister,” Ms Cini said.
Corangamite MP Sarah Henderson said: “This decision is a real blow and just terrible for Stephanie and her family.”