Health Canada has approved the use of Biogen Canada’s orphan drug, Spinraza (nusinersen) for the treatment of patients with 5q spinal muscular atrophy (SMA).
SMA is the number one genetic cause of death for infants, according to the Cure SMA, an organization funds groundbreaking SMA treatment. The disease affects the motor nerve cells in the spinal cord and weakens muscles leaving people unable to walk, eat or breathe.
5q SMA is the most common form of the disease and represents approximately 95 per cent of all SMA cases.
“Today, Biogen is very proud to share the news that Health Canada has approved Spinraza for the treatment of 5q SMA,” said Wildon Farwell, senior director for clinical development at Biogen. “Biogen is committed and willing to continue to work with healthcare professionals, advocacy groups, and government agencies to ensure people who could benefit from Spinraza receive access to this important treatment as quickly as possible.”
Japan’s Ministry of Health, Labour Welfare also approved Spinraza for the treatment of infantile-onset SMA. Biogen Japan will import the drug from Italy.
At least one in two out of 100,000 children under 18 months in Japan, fall victim to SMA, according to Japan’s Intractable Diseases Information Centre. Spinraza is the only drug approved in Japan for the treatment of SMA, according to reports.
On June 21, 2017, the European Commission (EC) granted a marketing authorization for Spinraza for the treatment of 5q spinal muscular atrophy (SMA) in the European Union. The company has also submitted regulatory filings in Australia, Brazil, and Switzerland and plans to initiate additional filings in other countries this year.
Biogen licensed the global rights to develop, manufacture and commercialize Spinraza from Ionis Pharmaceuticals.
Health Canada’s approval of Spinraza was based on positive results from multiple clinical studies in more than 170 patients.
The data package included the interim analysis of ENDEAR, a Phase 3 controlled study evaluating Spinraza in infantile-onset, as well as open-label data in pre-symptomatic and symptomatic patients with, or likely to develop, infantile and later-onset SMA.
SMA stems from the loss of or defect in, the SMN1 gene.
“In a healthy person, this gene produces a protein that is critical to the function of the nerves that control our muscles,” according to Cure SMA. “Without it, those nerve cells cannot properly function and eventually die, leading to debilitating and often fatal muscle weakness.”
People with SMA do not produce enough survival motor neuron (SMN) protein, which is critical for the maintenance of motor neurons.
SMA affects approximately one in 10,000 babies, and about 1 in every 50 Americans is a genetic carrier. SMA can affect any race or gender.
Spinraza is an antisense oligonucleotide (ASO) that is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q. The drug alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein.11 ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression.
Through the use of this technology, Spinraza has the potential to increase the amount of full-length SMN protein in individuals with SMA.
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