The European Medicines Agency (EMA) has accepted to review Sanofi’s marketing authorization application (MAA) for olipudase alfa to treat acid sphingomyelinase deficiency (ASMD).
An investigational enzyme replacement therapy, olipudase alfa is being assessed to treat patients suffering with non-central nervous system (CNS) manifestations of ASMD, which was previously referred to as Niemann-Pick disease (NPD) type A and type B.
The company’s application was based on favourable data obtained from ASCEND and ASCEND-Peds clinical trials.
Both the clinical trial programmes have assessed the enzyme replacement therapy to treat adult and paediatric patients with non-CNS manifestations of ASMD type A/B and ASMD type B.
Sanofi Rare Diseases Medical Affairs global head Alaa Hamed said: “Today’s milestone has been decades in the making and our gratitude goes to the ASMD community who has stood by us with endless patience while olipudase alfa advanced through clinical development.
“Olipudase alfa represents the kind of potentially life-changing innovation that is possible when industry, medical professionals and the patient community work together toward a common goal.”
The decision from the European regulatory is expected in the second half of next year.
If approved, olipudase alfa will be the first and only therapy of its kind to treat ASMD.
It has also received Breakthrough Therapy designation from the US Food and Drug Administration (FDA), PRIority MEdicines (PRIME) designation from the EMA as well as SAKIGAKE designation in Japan.
Sanofi Genzyme executive vice-president Bill Sibold said: “Scientific innovation is the greatest source of hope for people living with diseases like ASMD where there are no approved treatments and is a critical component for ensuring a viable healthcare ecosystem.
“At Sanofi, we have a long history of pioneering scientific innovation, and we remain committed to finding solutions to address unmet medical needs, including those of the rare disease community.”