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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.
Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. It is being recommended for the treatment of neuromyelitis optica spectrum disorder.
Daewoong Pharmaceutical has added benign masseteric hypertrophy (BMH), commonly known as the square jaw, to the indications for its botulinum toxin (BTX) product, Nabota.
Under the agreement, Orsini will be the exclusive specialty pharmacy partner for Veopoz (pozelimab-bbfg), a monoclonal antibody and the first and only treatment for those living with CHAPLE disease.
Veopoz™ (pozelimab-bbfg) is the proprietary VelocImmune® technology and is a fully human, monoclonal antibody designed to block the activity of complement factor C5 and prevent the CHAPLE Disease.
Under the agreement, Pharming will sell its Rare Pediatric Disease Priority Review Voucher, in connection with the approval of Joenja® (leniolisib), an oral, selective PI3Kδ inhibitor, for activated PI3Kδ syndrome, a rare and progressive primary immunodeficiency, to Novartis.
Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. It is being recommended for the treatment of neuromyelitis optica spectrum disorder.
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Sanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme. 
