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Under the agreement, Orsini will dispense Pombiliti™ (cipaglucosidase alfa-atga), a hydrolytic lysosomal glycogen-specific enzyme, and Opfolda™ (miglustat), a two-component treatment approved for certain patients with late-onset Pompe disease.
Lead Product(s): Cipaglucosidase Alfa,Miglustat
Therapeutic Area: Genetic Disease Product Name: Pombiliti
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Amicus Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement September 28, 2023
Details:
Under the agreement, Orsini will be the exclusive specialty pharmacy partner for Veopoz (pozelimab-bbfg), a monoclonal antibody and the first and only treatment for those living with CHAPLE disease.
Lead Product(s): Pozelimab-bbfg
Therapeutic Area: Rare Diseases and Disorders Product Name: Veopoz
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Regeneron Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement August 23, 2023
Details:
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
Lead Product(s): Valoctocogene Roxaparvovec-rvox
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: BioMarin Pharmaceutical
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 11, 2023
Details:
Vowst (fecal microbiota spores, live-brpk) is approved by FDA as the first orally administered microbiota-based therapeutic for the prevention of recurrent C. difficile infection (CDI).
Lead Product(s): Fecal Microbiota Spores Live-brpk
Therapeutic Area: Infections and Infectious Diseases Product Name: Vowst
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 23, 2023
Details:
Vyjuvek (beremagene geperpavec-svdt) is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. Vyjuvek received FDA Approval for the First-Ever Redosable Gene Therapy for DEB treatment.
Lead Product(s): Beremagene Geperpavec-svdt
Therapeutic Area: Genetic Disease Product Name: Vyjuvek
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Recipient: Krystal Biotech
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 23, 2023
Details:
Hemgenix (etranacogene dezaparvovec-drlb) is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with Hemophilia B who Currently use Factor IX prophylaxis therapy, or Have current or historical life-threatening hemorrhage.
Lead Product(s): Etranacogene Dezaparvovec-drlb
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Recipient: CSL Behring
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership December 20, 2022
Details:
AMVUTTRA (vutrisiran), is indicated to treat the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The drug works to reduce the production of the transthyretin (TTR) protein in the liver, thereby reducing the levels of TTR in the body.
Lead Product(s): Vutrisiran
Therapeutic Area: Genetic Disease Product Name: Amvuttra
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Alnylam Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership July 20, 2022
Details:
Orsini Specialty Pharmacy been selected by Alnylam® Pharmaceuticals as a limited distribution partner for OXLUMO™. OXLUMO is the first and only treatment approved for primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
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:
Orsini Pharmaceutical Services has been selected by Ultragenyx Pharmaceutical as a limited distribution specialty pharmacy partner for Dojolvi (triheptanoin). Dojolvi™ is indicated as a source of calories and fatty acids for the treatment of molecularly confirmed LC-FAOD.
Lead Product(s): Triheptanoin
Therapeutic Area: Genetic Disease Product Name: Dojolvi
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Ultragenyx Pharmaceutical
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership July 10, 2020
Details:
Orsini Pharmaceutical has been selected as one of a very limited number of distribution partners for VYEPTI™ (eptinezumab-jjmr).
Lead Product(s): Eptinezumab
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: ApprovedProduct Type: Large molecule
Recipient: H. Lundbeck AS
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement April 22, 2020
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