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Orphan Drug Designation for LX1004 from European Commission","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alexion Aims To Advance NMOSD Treatment Landscape With Exceptional Ultomiris Efficacy Data At ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","amount":"$28.0 million","upfrontCash":"Undisclosed","newsHeadline":"Taysha Announces Pricing of Public Offering of Common Stock","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"The British Journal of Dermatology Publishes Results from Amryt\u2019s EASE Phase 3 Trial in Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Small 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Applicable","newsHeadline":"Avilar Therapeutics to Present In Vivo Non-Human Primate Data on ASGPR-Targeting Chimeras (ATACs) at the Keystone Symposium on Targeted Protein Degradation","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Rallybio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rallybio Announces Positive Phase 1 Single Ascending Dose Results for RLYB116, an Innovative Subcutaneously Injected Inhibitor of Complement Component 5","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 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Find Clinical Drug Pipelines for Rare Diseases and Disorders
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.
Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.
Soliris (eculizumab) is a first-in-class C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system.
Subcutaneously administered RLYB116 was observed to be generally well-tolerated at the 100 mg dose, with mild or moderate adverse events and no drug-related serious adverse events reported.
Avilar develops ATACs (ASGPR Targeting Chimeras), a new class of protein degraders that shuttle disease-causing proteins from circulation to the endolysosome where the unwanted proteins are degraded.
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