The treatment is the first (and only) SMA-specific therapy approved by the U.S. Food and Drug Administration (FDA) for both children and adults, based on its statistically significant clinical studies’ results, which demonstrated increased survival and motor function.
“Spinraza is the only treatment for SMA, a rare neuromuscular disease without treatment options, and Biogen’s representative new drug,” Hwang Se-eun, CEO of Biogen Korea, said in a press release. “We are pleased to have quickly gained Korean approval for the novel drug quickly following approvals in the U.S., Europe, and Japan.”
Spinraza was approved in the United States in December 2016, gaining additional approvals in the European Union, Canada, Japan, Brazil and Australia since then.
In January, one month after FDA approval, new results from the Phase 3 ENDEAR study (NCT02193074) showed that Spinraza reduced the risk of death or permanent ventilation in infants with SMA. These were presented at the British Paediatric Neurology Association annual conference and were the first of a comprehensive body of research data presented for Spinraza in 2017.
In April, a Phase 3 (NCT02292537) clinical trial showed promising results of Spinraza in the treatment of children with type 2 SMA. These findings were the focus of an American Academy of Neurology meeting and contributed significantly to the debate over insurance coverage of Spinraza.
In May, Cure SMA advocates and other members of the community made headlines when they came together to demand answers from insurance companies that were still reluctant to pay for Spinraza, only a few months after the FDA had approved it. Spinraza ranks as one of the world’s most expensive drugs.
Also in May, aiming to contribute to the insurance debate, a group of researchers published their efforts to determine which SMA patients could benefit most from treatment with Spinraza. Looking at the final results of ENDEAR, researchers concluded that Spinraza was most beneficial at a regimen that created a managing problem for physicians, emphasizing that an earlier treatment was still needed. For these authors, it was still unclear if Spinraza remained effective over time; they pleaded for additional long-term studies.
In June, Spinraza was a hot topic again, after the European Medicines Agency (EMA) decided to approve the SMA drug in the European Union, becoming the first treatment available for almost all types of SMA on the continent.
SMA is caused by mutations in the copy of the survival motor neuron 1 (SMN1) gene that is in chromosome 5. The absence of the SMN1 exon 7 has been observed in the majority of patients and is currently used as an SMA diagnostic test.