[{"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"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"X4 Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"PANTHERx\u00ae Rare Partnering with X4 Pharmaceuticals Inc. for the Distribution of XOLREMDI\u2122 (mavorixafor), the First FDA-approved Therapy Indicated for Use in Patients with WHIM Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"}]
Find Clinical Drug Pipeline Developments & Deals by PANTHERx Rare Pharmacy
Through the partnership, X4 Pharmaceuticals will distribute Xolremdi (mavorixafor), recently approved by the FDA. It is indicated for use in patients 12 years of age and older with warts, hypogammaglobulinemia, infections and myelokathexis syndrome.
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.