FDA drug approvals were at a low in 2016, with just 22 new drugs making it to market, down from 45 in 2015. This is the lowest number of approvals in a year since 2010.
However, while the number may be low, some of the drugs approved in the past year will have a great impact. Take Biogen and Ionis Pharmaceuticals’ spinal muscular atrophy (SMA) drug, Spinraza (nusinersen), for example. As the first drug approved to treat pediatric and adult patients with SMA, Spinraza is an exciting new product that will provide hope for many across the US.
The approval, which was announced in late December, was based on data from a clinical trial with 121 patients, younger than the age of 7 months at the time of first dose, with infantile-onset SMA. Of those treated with the drug, 40% achieved improved motor responses – including head control, rolling, crawling, standing and walking – compared to none of the patients in the control group. In addition, data was also considered from open-label, uncontrolled, studies with patients from 30 days to 15 years of age at the time of the first dose.
SMA is currently the most common genetic cause of death in infants, although it is a disease that can affect people at any stage of life. As such, the FDA approval of Spinraza is a huge step in the right direction for the SMA community. However, as has been the case with many of the drugs approved in the past year for rare disease indications, cost is a significant issue for patients.
The drug is being sold on the market for a whopping $125,000 per dose. That totals $750,000 for the first year, in which patients will require 6 doses, and $375,000 for the subsequent years, in which patients will require three doses each year. As this is a treatment that SMA patients will require for life, the high cost of the drug is a substantial burden for patients to bear.
However, in response to early public scrutiny of this high cost, Biogen has claimed that the drug is appropriately priced given the ultra-orphan-nature of the disease, meaning that there are few target patients out there to buy the drug, making it difficult to offset the high research and development costs incurred by the pharma company.
Of course, the idea is that most patients will not be expected to cover the entirety of the cost; insurance companies will take care of that. But some worry that the high cost of the drug will deter insurers from covering it, or will force them to limit providing the drug to only those patients who are the most severely affected.
While it’s too early to say for sure what effect this high market price will have on the drugs uptake, SMA patients can at least take some comfort in knowing that research into providing treatment for their disease is on the right track.