Trusted Resources: News & Meetings
Latest announcements and gatherings
Europe Grants Orphan Drug Status to JR-441, ERT for Sanfilippo A
The European Commission has granted an orphan drug designation to JR-441, JCR Pharmaceuticals’ brain-penetrating experimental enzyme replacement therapy (ERT) for Sanfilippo syndrome type A, the company has announced. The designation is given to investigative therapies with the potential to be safe and effective for rare, life-threatening, or chronically debilitating conditions that have no approved treatments, or where the potential therapy is showing significant benefit over existing treatments.
It is meant to accelerate JR-441’s clinical development and review by providing regulatory support and financial benefits. It also provides a 10-year marketing exclusivity period in Europe upon regulatory approval, if granted. JCR is planning to launch a global clinical trial testing the therapy in the first half of 2023. Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA, is caused by the absence or reduced activity of the heparan N-sulfatase enzyme due to mutations in the SGSH gene.
BookmarkRelated Content
-
Elosulfase Alfa in the Treatment of Mucopolysaccharidosis Type IVA: Insights From the First Managed Access AgreementManaged access agreements provide a cruc...
-
Developing a Treatment Plan for Sanfilippo SyndromeSanfilippo syndrome is a neurodegenerati...
-
Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the ArtThe need for long-lasting and transforma...
-
A Multicenter Open-Label Extension Study of Intrathecal Heparan-N-Sulfatase in Patients With Sanfilippo Syndrome Typ...Sanfilippo syndrome type A (mucopolysacc...
-
Patricia Dickson, MDDr. Dickson is Division Chief of Genetic...
-
Sanfilippo Syndrome Type B: Analysis of Patients Diagnosed By the MPS Brazil NetworkMucopolysaccharidosis type IIIB is a rar...
-
Progression of Cardiovascular Manifestations in Adults and Children With Mucopolysaccharidoses With and Without Enzy...Cardiovascular involvement is among the ...
Powered by
