By Fineline Cube 
US-based Biogen Inc. (NASDAQ: BIIB) announced that the Center for Drug Evaluation (CDE) in China has accepted a market filing for a high-dose regimen of its Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA). This acceptance marks a significant step in expanding therapeutic options for patients with this rare and often severe neuromuscular disease.
New High-Dose Regimen
The high-dose regimen of nusinersen involves a more aggressive loading phase with two 50 mg doses administered 14 days apart, followed by a maintenance dose of 28 mg every four months. This contrasts with the previously approved regimen, which includes lower doses and less frequent administration.
Clinical Evidence and Benefits
The filing was supported by positive results from the DEVOTE study, which demonstrated that the high-dose regimen could slow neurodegeneration more rapidly and significantly improve motor function in infant patients compared to the approved regimen. Additionally, the high-dose regimen showed advantages across multiple secondary efficacy endpoints.
Mechanism and Impact
Nusinersen, an antisense oligonucleotide (ASO) drug, is administered via intrathecal injection. It directly targets the disease origin site in the spinal cord and brainstem motor neurons. This mechanism allows for precise therapeutic effects, improving motor function, increasing survival rates, and altering the disease progression of SMA.
Market Presence
Nusinersen was first approved in China in February 2019 through a priority review process. It became the first SMA therapy available in the country and was subsequently included in the National Reimbursement Drug List (NRDL) two years later. The acceptance of the high-dose regimen for review highlights Biogen’s ongoing commitment to advancing treatments for SMA and addressing unmet medical needs.-Fineline Info & Tech