STA, 1 June 2019 - The US Food and Drug Administration has approved the first gene therapy to treat babies with spinal muscular atrophy, an innovative therapy that also involves technology developed by the Slovenian company Bia Separations.
The medication, Zolgensma, is manufactured by the US company Avexis, part of the Swiss group Novartis, using Bia Separations's purification technology.
Aleš Štrancar, the CEO of the Ajdovščina-based company, has hailed the approval of Zolgensma as Slovenian science's breakthrough among leading global strategic technologies.
Purification is a key process in production of medicines, in particular biological ones, because the active substance needs to be separated from other substances, which are often toxic.
The purification process represents up to 90% of the production costs of a drug.
Bia Separations's smart filters have become part of the therapy's registration, which means exclusive use of the Slovenian company's products for 20 to 30 years.
The approval makes Avexis a leading manufacturer of active agents for gene therapy, Štrancar said, adding that for Bia Separations this was a key reference project in the field.
"Our company is looking at the potential to generate more than 500 million euro in annual sales in a decade," Štrancar said.
He underscored that without this innovative gene therapy babies with spinal muscular atrophy would die before reaching the age of two years.
The medication replaces part of the flawed gene and after treatment the patients can live normally.
Štrancar noted that the therapy's approval is paving the way for development of active agents to treat rare genetic disorders in the short term, and to treat all types of disease in the long run.