EMA accepts marketing authorisation application for ALS treatment, tofersen

pharmafile | December 7, 2022 | News story | Sales and Marketing  

The European Medicines Agency (EMA) has accepted a marketing authorisation application (MAA) for tofersen. The drug would treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). If approved it would target the rare genetic cause of ALS, the first drug to do so.

At the moment, there are no genetically-targeted therapy options for ALS, so the approval of tofersen would be a huge step forward in treating the disease. Life expectancy for patients with SOD1-ALS is already shorter than other forms of the disease.

The MAA follows results from the phase 3 VALOR study, a phase 1 study in healthy volunteers, as well as a phase 1/2 study to assess dose levels of the drug. The phase 3 ATLAS study is continuing to evaluate tofersen, considering whether the drug can delay onset of the disease in presymptomatic patients with a SOD1 genetic mutation and biomarker evidence of ALS activity. 

Tofersen is also under review by the FDA, with a Prescription Drug User Fee Act action date of 25 April 2023.

Priya Singhal, head of global safety and regulatory sciences and interim head of R&D at Biogen, commented: “Through our clinical development programme, we have seen that tofersen has the potential to slow the progression of this relentless and ultimately fatal disease. Regulatory submissions in the US and now EU represent an important step in our efforts to bring the first genetically-targeted treatment for SOD1-ALS to the ALS community as quickly as possible.”

 

Betsy Goodfellow


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