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Find Clinical Drug Development Pipelines & Deals | PipelineProspector
Xenpozyme, (olipudase Alfa-rpcp) hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy, is designed to replace deficient or defective acid sphingomyelinase, an enzyme that allows for the breakdown of the lipid sphingomyelin.
Xenpozyme (Olipudase alfa), is an enzyme replacement therapy designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid sphingomyelin.
Recommendation based on positive results from two clinical trials in which Xenpozyme (olipudase alfa) provided improvement across multiple non-CNS clinical manifestations of ASMD in pediatric and adult patients.
Approval based on positive results of Xenpozyme® (olipudase alfa) from two separate clinical trials in children and adults demonstrating improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes.
Filsuvez (oleogel-S10), a topical therapeutic gel, is a potential treatment for the cutaneous manifestations of Epidermolysis Bullosa, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment.
UPLIZNA (inebilizumab-cdon), is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
ANI’s Levocarnitine Tablets are the generic version of the Reference Listed Drug (RLD) Carnitor®. The launch of Levocarnitine Tablets is another example of ANI’s commitment to increasing patient access to affordable, high-quality medicines.
Empaveli™ having pegcetacoplan, is a targeted C3 therapy designed to regulate the activity of the complement cascade, which can lead to the onset and progression of many serious diseases.
RETHYMIC® (allogeneic processed thymus tissue-agdc) is a novel one-time tissue-based regenerative therapy used for immune reconstitution in pediatric patients with congenital athymia.
Lead Product(s):
Allogeneic Processed Thymus Tissue-agdc
Waylivra (volanesorsen), an antisense oligonucleotide designed to inhibit the formation of apoC-III, is a product of Ionis Pharmaceuticals, Inc.'s proprietary antisense technology. Waylivra has received conditional marketing approval in the EU as a treatment for FCS.