[{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Horizon Therapeutics Plc Announces U.S. FDA Approval of New Dosage Form for PROCYSBI\u00ae (Cysteamine Bitartrate) Delayed-Release Oral Granules","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Horizon Therapeutics"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Horizon Therapeutics Plc Announces Availability of PROCYSBI\u00ae (Cysteamine Bitartrate) Delayed-Release Oral Granules in Packets in the United States","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Horizon Therapeutics"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Receives Reimbursement Approval from the French Ministry of Social Affairs and Health for Myalepta\u00ae (metreleptin)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Peptide","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Peptide","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Receives Reimbursement Approval from the Dutch Ministry of Health for Myalepta\u00ae (metreleptin)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Peptide","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Announces Positive Top Line Results from Phase 3 Pediatric Trial of Lomitapide in HoFH","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"January 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"}]
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Lojuxta (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the rare cholesterol disorder, Homozygous Familial Hypercholesterolemia ("HoFH").
Myalepta (metreleptin) is approved in the US as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired GL and in the EU as an adjunct to diet for the treatment of leptin deficiency.
French Ministry of Social Affairs and Health has approved Myalepta® (metreleptin) for reimbursement in France as an adjunct to diet as replacement therapy for the treatment of leptin deficiency.
Procysbi (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.
Procysbi (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.