Spinraza Approved for Spinal Muscular Atrophy
December 23, 2016 – The U.S. FDA approved Spinraza™ (nusinersen), developed by Ionia Pharmaceuticals and marketed by Biogen, Inc. Spinraza is the first drug indicated to treat spinal muscular atrophy (SMA), a rare genetic condition that causes increasing weakness in muscles. Spinraza will be given intrathecally, which is directly into the fluid around the spinal cord, by a specially trained healthcare provider. Recommended dosing is three 12mg doses administered at two-week intervals, followed by a fourth dose one month after the third; and then one dose every four months.
SMA is a rare genetic condition that progressively shrinks and weakens muscles due to inadequate amounts of survival motor neuron protein 1 (SMN1), a specific substance needed by nerve cells (motor neurons) in the brain stem and spinal cord. Most patients with the severest forms of the disease increasingly lose the ability to move and most die in childhood. Those with less serious forms may live into adulthood, but they gradually may become paralyzed.
With between 10,000 and 25,000 patients in the United States, the FDA designated Spinraza as an orphan drug and approved it under priority review. The first in the new class of drugs known as survival motor neuron-2 (SMN2)-directed antisense oligonucleotides, Spinraza increases amounts of SMN2, a very similar protein that replaces the missing SMN1. Previous treatments for SMA included only symptom management such as breathing support and physical therapy.
Spinraza must be administered in a sterile setting by a health professional trained in spinal procedures to help prevent spinal cord damage or infections. Prior to each administration of Spinraza, 5mL of spinal fluid must be removed and specific blood and urine tests are required.
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