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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.
Lead Product(s): Mavorixafor
Therapeutic Area: Genetic Disease Product Name: Xolremdi
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: X4 Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership April 29, 2024
Details:
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.
Lead Product(s): Leniolisib
Therapeutic Area: Rare Diseases and Disorders Product Name: Joenja
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 28, 2023
Details:
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.
Lead Product(s): Stiripentol
Therapeutic Area: Neurology Product Name: Diacomit
Highest Development Status: ApprovedProduct Type: Small molecule
Recipient: Biocodex
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership June 27, 2022
Details:
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.
Lead Product(s): Lumasiran
Therapeutic Area: Genetic Disease Product Name: Oxlumo
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Alnylam Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership December 03, 2020
Details:
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.
Lead Product(s): Pralsetinib
Therapeutic Area: Oncology Product Name: Gavreto
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Blueprint Medicines
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement September 14, 2020
Details:
With nationwide distribution of AYVAKIT, PANTHERx establishes a formidable presence in the precision oncology space.
Lead Product(s): Avapritinib
Therapeutic Area: Oncology Product Name: Undisclosed
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Blueprint Medicines
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership January 17, 2020
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