Rare Disease Report

Biogen's Orphan Drug for SMA to Cost $750,000 in First Year

JANUARY 01, 2017
James Radke, PhD
On December 23rd, the rare disease community rejoiced in the news that the FDA had approved Biogen’s Synraza (nusinersen) to treat all patients with spinal muscular atrophy (SMA), a rare and devastating neuromuscular disease.  The reality of the drug pricing may dampen that enthusiasm.
Last Wednesday, Biogen listed the drug's wholesale acquisition cost (WAC) at $125,000 per dose.
According to the dosing schedule, new patients would receive 4 doses in the first 2 months of therapy and then a maintenance dose every 4 months.
That means in the first year, the patient would receive 6 doses ($750,000) and in subsequent years, 3 maintenance doses ($375,000).

Drug Pricing Media Storm (and a Trump Tweet) Predicted

Analysts Geofrey Porges and Bradley Caninoo at Leerink reported on the pricing of the drug and made several predictions about what the high cost of the drug – especially in the first year for new patients—will illicit. First and foremost, there will be a media storm, including a likely caustic tweet from President-elect Donald Trump.
The analysts also noted:
“The sticker-shock presented in the media could turn Spinraza into the Sovaldi of rare disease drugs, or the straw that breaks the camel’s back in terms of the US market’s tolerance for rare disease drug pricing.”

Some Patients May Have Difficulty Obtaining the Drug

The analyst also warns that the high cost may cause payers to be more stringent on which patients receive the drug. Leerink speculated that:
“…the price is going to force payers to closely scrutinize which patients receive access and limit the overall access provided. To us it seems certain that patients who have the less severe Type III and IV forms of the disease or who are older with relatively milder symptoms will find it difficult to obtain treatment.”

What is Spinal Muscular Atrophy?

SMA is a genetic condition that leads to a deficiency in the spinal motor neuron (SMN) protein as a result of mutations of the survival motor neuron 1 (SMN1) gene. The severity of SMA correlates with the amount of SMN protein. Generally, the muscles most affected are those near the shoulders, hips, thighs and upper back. Muscles used for breathing and swallowing may also be affected. Infants with Type I SMA produce very little SMN protein and have a life expectancy of less than two years. Children with Type II have greater amounts of SMN protein but still have a shortened lifespan and are never able to stand independently. Children with Type III have a normal lifespan but accumulate life-long physical disabilities as they grow.
Spinraza (nusinersen) is an antisense oligonucleotide (ASO) designed to alter the splicing of pre-mRNA from the SMN2 gene in order to increase production of fully functional SMN protein. Spinraza is approved for Type I, II, and III SMA populations.
Biogen has launched SMA360 to provide certain services that address nonmedical barriers to access in the United States (i.e., logistical assistance, product education, insurance benefits investigations and financial assistance. For more information, visit www.spinraza.com.

Image courtesy of http://torange.biz/19324.html

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