[{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Blueprint Medicines","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"PANTHERx\u00ae Rare Pharmacy Selected by Blueprint Medicines Corporation to Distribute AYVAKIT\u2122 (avapritinib) for Genomically Defined Patient Population with Rare Cancer, GIST","therapeuticArea":"Oncology","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2020","url1":"","url2":"","graph1":"Oncology","graph2":"Approved"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Blueprint Medicines","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"PANTHERx\u00ae Rare Pharmacy Selected by Blueprint Medicines to Distribute GAVRETO\u2122 for Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer","therapeuticArea":"Oncology","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Oncology","graph2":"Approved"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"PANTHERx Rare\u00ae Pharmacy Chosen as Partner by Alnylam for OXLUMO to Treat Primary Hyperoxaluria Type 1 to Lower Urinary Oxalate Levels","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"December 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Biocodex","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"PANTHERx Rare Announces Partnership with Biocodex for the Distribution of DIACOMIT\u00ae (stiripentol) for the Treatment of Seizures Associated with Dravet Syndrome in Patients 2 Years of Age and Older Taking Clobazam","therapeuticArea":"Neurology","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Neurology","graph2":"Approved"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PANTHERx\u00ae Rare Launches Drug for APDS","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2023","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Approved"}]
Find Clinical Drug Pipeline Developments & Deals by PANTHERx
Joenja (leniolisib) is an oral, selective PI3K-delta inhibitor which inhibits the signalling pathways that lead to increased production of PIP3, hyperactivity of the downstream mTOR/AKT pathway, and to the dysregulation of B and T cells in patients with APDS.
DIACOMIT (stiripentol), is a molecular entity approved by the FDA for the adjunctive treatment of seizures associated with Dravet syndrome in 2 years of age and older taking clobazam. An orally administered, DIACOMIT is given as either a capsule or powder for suspension.
PANTHERx® Rare Pharmacy has been selected by Alnylam Pharmaceuticals as a very limited distribution pharmacy partner for new drug OXLUMO™ (lumasiran). OXLUMO is a subcutaneously administered RNAi therapeutic that targets the messenger RNA of the hydroxyacid oxidase 1 gene.
PANTHERx® Rare Pharmacy has been selected by Blueprint Medicines as a limited distribution partner for new drug GAVRETO™ (pralsetinib), which was granted accelerated approval for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer.