Spinraza – FDA Approved Treatment For Spinal Muscular Atrophy?


At the end of every year, the FDA typically approves a flurry of new drugs. That didn’t happen in 2016. However, the FDA did approve Biogen’s Spinraza as a treatment for Spinal Muscular Atrophy (SMA).

Spinraza (nusinersen) became the first drug approved in the United States to treat children and adults with SMA. The FDA approved the drug just before the Christmas break. SMA is an often fatal genetic disease the affects muscle and strength movement. Approximately 1 in 6,000 to 10,000 babies are affected by SMA, and it’s a leading cause of genetic death in infants.

Patients with SMA can become paralyzed, losing the ability to carry out basic bodily functions like swallowing and breathing.

In summer of 2016, Biogen licensed Spinraza – an antisense oligonucleotide drug – from Ionis Pharma for $75 million upfront and an additional $150m in regulatory milestones and royalties on sales. Industry analysts believe that sales of the drug could pass $1 billion by 2020.

Spinraza Will Cost $750,000 Per Year

Do you or a loved one have spinal muscular atrophy? You better be prepared to open your wallet, because Spinraza is one of the most expensive drugs on the market. Biogen will charge $750,000 for the first year of Spinraza treatment, dropping it down to $375,000 per year thereafter.

The $750,000 per year price tag is based on the cost of $125,000 per injection.

This is a similar pricing model to drugs used to treat other rare diseases. Patients with these rare diseases have no other drug options, so pharmaceutical companies can charge whatever they like.

The high pricing for Spinraza has generated headlines across the industry. One analyst said that Spinraza’s “eye-watering” pricing could be “the straw that breaks the camel’s back”, bringing rare disease therapies to the forefront of the pharma pricing debate.

“The price is going to force payers to closely scrutinise which patients receive access and limit the overall access provided”, industry analyst Geoffrey Porges explained.

Porges added that the pricing policy will mean patients with less severe cases of SMA will likely miss out on treatment. In other words, the price for the drug is so high that insurers will only cover the cost for those with near-fatal cases of SMA.

Another industry analyst argued that Spinraza’s $750,000 price tag isn’t totally unprecedented, stating that other rare disease drugs are priced at $400,000 per year, although “Unfortunately for Biogen, Spinraza has made its debut at a time when public attention on drug prices is at an all-time high.”

The FDA Approved Spinraza After a Clinical Trial of 121 Patients

In December 2016, the FDA approved Spinraza after reading the results of a clinical trial involving 121 patients. Those patients had infantile-onset SMA diagnosed before they were six months old. All patients were less than 7 months old at the time of dosing.

Infants in this trial received an injection of Spinraza into the fluid surrounding the spinal cord. Other infants received a mock procedure (a skin prick) as a control.

Ultimately, the results from 82 subjects showed that 40% of Spinraza-treated patients experienced improvements in motor movements like head control, sitting, ability to kick in supine position, rolling, crawling, standing, and walking. No patients treated in the control group showed any improvement.

Ultimately, the approval of Spinraza brought the final tally of FDA approvals to 22 across all of 2016, which is significantly less than the 40-plus drugs approved in 2014 and 2015.


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