Cure SMA to Host May 4 Webinar on Spinraza Access, Insurance Coverage and Trial Results

The patient advocacy group Cure SMA will host a webinar May 4 on access to Spinraza (nusinursen), the first drug ever to be approved to treat spinal muscular atrophy (SMA).

The webinar will cover several topics, including the current status of Spinraza dosing at U.S. administration sites, commercial and government insurance policies, trial results and community support.

Anyone wishing to join an informal discussion on how to access Spinraza may submit questions before the webinar or ask them during the event. To register, click here.

The U.S. Food and Drug Administration (FDA) approved Spinraza last December. It is the only approved therapy available for SMA patients. It works by increasing the expression levels of the SMN gene and, consequently, increasing the levels of the encoded SMN protein, whose absence in neurons leads to the development of the disease.

Biogen, which is licensing Spinraza from Ionis Pharmaceuticals, lists the drug’s current price at $125,000 per injection, or $750,000 for the first year of treatment, consisting of six injections. Common side effects include respiratory infections, constipation and possible kidney damage. Spinraza must be administered through a spinal tap in specialized facilities, which may limit access to the therapy.

Cure SMA has estimated that Spinraza should become available in 200 to 300 sites across the United States. To date, 26 states have access to Spinraza. Some of these 26 states have only one or two sites, while others have four or five. All sites are running at full capacity and already have waiting lists.

“We have to work together to greatly increase the number of sites involved in both delivering this first-ever new SMA therapy and providing SMA care,” Cure SMA said April 24 in a press release. “This process will take time, and there should be the expectation of delays in gaining access to Spinraza while this process takes place.”

Cure SMA, a nonprofit organization based in Elk Grove Village, Ill., promotes SMA research and provides support for patients and their families.