Santhera Pharmaceuticals shared its 2019 Annual Results and Highlights Piepleine Progress for Duchenne muscular dystrophy.
“Santhera is emerging as a leader in addressing rare neuromuscular diseases and 2019 saw us prepare the business for several significant value inflection points this year for our drug candidatesto treat DMD,” said Dario Eklund, CEO of Santhera. “The regulatory review for Puldysa in the EU, where we are seeking a conditional marketing authorization (CMA) for the treatment of respiratory dysfunction in patients with DMD who are not using glucocorticoids, is on track and we anticipate obtaining an opinion from the European regulators in mid-2020. Our goal is to provide the first approved drug in non-ambulant DMD patients who are in respiratory decline and we are planning for launch in the first European markets by the end of the year.”
“Our second DMD product, vamorolone, is currently being investigated by the originator company ReveraGen in a pivotal study (called VISION-DMD) in ambulant patients with DMD, and our ambition is to replace glucocorticoids as standard of care. We expect read-out of topline data from this trial in the fourth quarter 2020, followed by filing of a US new drug application (NDA) in the first quarter of 2021.Positive data will also pave the way for Santhera to exercise its sub-licensing option for the product.”
About Santhera Pharmaceuticals
Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company committed to developing and commercializing innovative medicines to meet the needs of patients living with rare and other diseases with high unmet medical needs.
They are focusing on the development of treatments for neuromuscular and pulmonary diseases that currently lack treatment options, such as Duchenne muscular dystrophy, congenital muscular dystrophy, and cystic fibrosis.