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(NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Chugai Pharmaceutical"},{"orgOrder":0,"company":"American Regent","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"American Regent Introduces Levocarnitine Injection, USP; FDA-Approved, \"AP\" Rated, and Therapeutically Equivalent to Carnitor\u00ae\u00b9","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"American Regent"},{"orgOrder":0,"company":"Pint Pharma","sponsor":"OrphanDC","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not 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Find Clinical Drug Development Pipelines & Deals | PipelineProspector
Nexviazyme (avalglucosidase alfa) is a USFDA approved GAA stimulator enzyme replacement therapy. It is being evaluated for the treatment of treatment-naïve Infantile-onset Pompe disease.
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.
The company launched desmopressin acetate for injection in the United States as a therapeutic equivalent generic for DDAVP for treating is multi-indicated for patients with central diabetes insipidus, hemophilia A, and von willebrand’s disease (Type I).
Voydeya (danicopan) is a first-in-class, oral medicine in development as an add-on to C5 inhibitor therapy eculizumab or ravulizumab for patients with paroxysmal nocturnal hemoglobinuria (PNH) who have residual haemolytic anaemia.
Adzynma (apadamtase alfa /cinaxadamtase alfa) is the first and only recombinant ADAMTS13 enzyme replacement therapy, which is approved for the treatment of congenital thrombotic thrombocytopenic purpura in patients 12 years of age and older.
Lead Product(s):
Apadamtase Alfa,Cinaxadamtase Alfa
Piasky (crovalimab) is a humanized complement inhibitor C5 monoclonal antibody. It is being developed for the treatment of adults and adolescents (12 years of age and above) with Paroxysmal Nocturnal Hemoglobinuria.
Ultomiris (ravulizumab) has been approved in the US as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) 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. 
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