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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 September 25, 2020
Details:
Safety data from a cohort of the Phase 3 ARRIVAL study support treatment with KALYDECO in children ages four to <6 months with eligible mutations.
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Lead Product(s): CTX001
Therapeutic Area: Genetic Disease Product Name: CTX001
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Recipient: CRISPR Therapeutics
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 22, 2020
Details:
European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD).
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Lead Product(s): LNP based mRNA Therapeutics
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Discovery Product Type: Cell and Gene therapy
Recipient: Moderna Therapeutics
Deal Size: $455.0 million Upfront Cash: $75.0 million
Deal Type: Collaboration September 16, 2020
Details:
The three-year research collaboration initially will focus on the discovery and optimization of novel LNPs and mRNAs that can deliver gene-editing therapies to cells in the lungs, enabling functional cystic fibrosis transmembrane conductance regulator protein to be produced.
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Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor
Therapeutic Area: Genetic Disease Product Name: Trikafta
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 September 10, 2020
Details:
The primary endpoint of the study was safety and tolerability, and the results showed that TRIKAFTA was generally well tolerated and the safety data were consistent with those observed in previous Phase 3 studies.
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Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor
Therapeutic Area: Genetic Disease Product Name: Trikafta
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 September 01, 2020
Details:
U.S. Food and Drug Administration (FDA) accepted three supplemental New Drug Applications (sNDAs) for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor).
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Lead Product(s): Elexacaftor,Tezacaftor,Ivacaftor
Therapeutic Area: Genetic Disease Product Name: Kaftrio
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 August 21, 2020
Details:
E.C has granted marketing authorization for KAFTRIO® in a combination regimen with ivacaftor to treat people with cystic fibrosis ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the CFTR gene.
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Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor
Therapeutic Area: Genetic Disease Product Name: Trikafta
Highest Development Status: Phase IV Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 20, 2020
Details:
The results of this study demonstrate that the triple combination provides significant additional benefit compared to existing CFTR modulator therapy for F/G and F/RF patients and supports the benefit of the medicine for patients with at least one F508del mutation.
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Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor
Therapeutic Area: Genetic Disease Product Name: Kaftrio
Highest Development Status: Phase IV Product Type: Small molecule
Recipient: NHS England
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement June 30, 2020
Details:
As part of the agreement with NHS England, Vertex has committed to submit ORKAMBI®, SYMKEVI® and KAFTRIO® to the National Institute for Health and Care Excellence (NICE) within an agreed upon timetable, allowing for a period of real-world data collection on the medicines.
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Lead Product(s): AAV capsid-gene therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Recipient: Affinia Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration April 27, 2020
Details:
Affinia Therapeutics’ proprietary AAV vector technology to be used in Vertex’s genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis.
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Lead Product(s): Lumacaftor,Ivacaftor
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: FSIO
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement April 21, 2020
Details:
Agreement enables the possibility of rapid patient access to future triple combination regimen (elexacaftor/tezacaftor/ivacaftor and ivacaftor) once approved in Switzerland .
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