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Lead Product(s): Berotralstat
Therapeutic Area: Genetic Disease Product Name: Orladeyo
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Royalty Pharma
Deal Size: $325.0 million Upfront Cash: $125.0 million
Deal Type: Funding December 07, 2020
Details:
BioCryst plans to invest the combined proceeds to support the launch of ORLADEYO in the U.S. and Europe and to advance the development of BCX9930 into clinical trials in multiple complement-mediated diseases.
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Lead Product(s): Lumasiran
Therapeutic Area: Genetic Disease Product Name: Oxlumo
Highest Development Status: Approved Product Type: Large molecule
Partner/Sponsor/Collaborator: Alnylam Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership December 03, 2020
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.
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Lead Product(s): Lumasiran
Therapeutic Area: Genetic Disease Product Name: Oxlumo
Highest Development Status: Approved Product 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 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.
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Lead Product(s): Lumasiran
Therapeutic Area: Genetic Disease Product Name: Oxlumo
Highest Development Status: Approved Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 03, 2020
Details:
Alnylam has achieved full patient enrollment in its ILLUMINATE-C Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1– the gene encoding glycolate oxidase (GO) – for the treatment of adults and children with advanced primary hyperoxaluria type 1.
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Lead Product(s): Voxelotor
Therapeutic Area: Genetic Disease Product Name: Oxbryta
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 02, 2020
Details:
Through the voxelotor early access program, physicians in countries with an early access regulatory and legal pathway may be able to request voxelotor for eligible SCD patients who do not have access to the medicine as part of a clinical trial.
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Lead Product(s): Lonafarnib
Therapeutic Area: Genetic Disease Product Name: Zokinvy
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: The Progeria Research Foundation
Deal Size: $95.0 million Upfront Cash: Undisclosed
Deal Type: Divestment November 23, 2020
Details:
The PRV was granted in conjunction with the recent approval by the U.S. Food and Drug Administration of ZokinvyTM (lonafarnib) for treatment of Progeria and processing-deficient Progeroid Laminopathies.
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Lead Product(s): Lonafarnib
Therapeutic Area: Genetic Disease Product Name: Zokinvy
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 20, 2020
Details:
Zokinvy is a disease-modifying agent that has demonstrated a statistically significant survival benefit in children and young adults with Progeria. In patients with Progeria, Zokinvy reduced the incidence of mortality by 60% and increased average survival time by 2.5 years.
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Lead Product(s): Lumasiran
Therapeutic Area: Genetic Disease Product Name: Oxlumo
Highest Development Status: Approved Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 19, 2020
Details:
OXLUMO is the first therapeutic approved for the treatment of PH1, and the only therapy proven to lower harmful oxalate levels that drive the progression of PH1 disease.
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Lead Product(s): Ivacaftor
Therapeutic Area: Genetic Disease Product Name: Kalydeco
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 05, 2020
Details:
KALYDECO® (ivacaftor) will be now available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland.
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Lead Product(s): Ivacaftor
Therapeutic Area: Genetic Disease Product Name: Kalydeco
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Royalty Pharma
Deal Size: $650.0 million Upfront Cash: $575.0 million
Deal Type: Acquisition November 02, 2020
Details:
Funding will support the CF Foundation’s work to fund research and drug development and advance high-quality, specialized CF care. KALYDECO® (ivacaftor) as First and Only CFTR Modulator, indicate to treat Eligible Infants With CF as Early as Four Months of Age.
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