pipeline-prospector-insert
X

Find Approved Drugs for Genetic Disease in Clinical Development

Loading...

All Data

Filters Filter refresh
×
FILTER DATA :
Country filter
    News Type filter
      Company filter
        Product Type filter
          Deal Size filter
            refresh

            Product Type Full Screen

            Companies Full Screen

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Albutrepenonacog Alfa

            Therapeutic Area: Genetic Disease Product Name: Idelvion

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 03, 2020

            Details:

            EMA has approved the new Summary of Product Characteristics, which includes information about new, extended dosing options for IDELVION® CSL Behring's novel, long-acting recombinant albumin fusion protein for treating haemophilia B.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Efmoroctocog alfa

            Therapeutic Area: Genetic Disease Product Name: Elocta

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: National Health Service (NHS)

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement June 29, 2020

            Details:

            The National Health Service (NHS) and Sobi UK have come to an agreement that will give people living with haemophilia A in the UK increased access to Elocta (efmoroctocog alfa), by lifting prior volume restrictions for the drug.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Ataluren

            Therapeutic Area: Genetic Disease Product Name: Translarna

            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 June 29, 2020

            Details:

            Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended by a majority of votes to remove the statement "efficacy has not been demonstrated in non-ambulatory patients" from the SmPC for Translarna™ (ataluren).

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): L-Glutamine

            Therapeutic Area: Genetic Disease Product Name: Endari

            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 June 29, 2020

            Details:

            Endari® is approved in Israel as in the U.S. as an amino acid indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients five years of age and older.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            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 June 24, 2020

            Details:

            An oral, once daily therapy, Oxbryta directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. Based on recent discussions with the EMA, GBT intends to seek full marketing authorization of Oxbryta.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Antihemophilic factor recombinant PEGylated-aucl

            Therapeutic Area: Genetic Disease Product Name: Jivi

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 15, 2020

            Details:

            Bayer presented data comparing the pharmacokinetic profiles of two extended half-life recombinant factor VIII (FVIII) therapies, Jivi® (antihemophilic factor [recombinant] PEGylated-aucl) and Adynovate® in people living with hemophilia A.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Betibeglogene autotemcel

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 June 12, 2020

            Details:

            89% of evaluable patients (17/19) with transfusion-dependent β-thalassemia who do not have a β0/β0 genotype achieved transfusion independence with 11.9 g/dL median weighted average total hemoglobin (Hb) level in HGB-207.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            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 June 08, 2020

            Details:

            GBT intends to submit a New Drug Application for Oxbryta, which will include a new age-appropriate formulation, for the treatment of SCD in children ages 4 to 11 years. The NDA will include clinical data from the ongoing Phase 2a HOPE-KIDS 1 study.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Lanadelumab

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 06, 2020

            Details:

            Data from the Phase 3 HELP Study™ Open-label Extension suggest that TAKHZYRO® (lanadelumab) is well-tolerated and can prevent hereditary angioedema (HAE) attacks over an extended treatment period, with sustained and consistent reduction in monthly attack rates.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Mexiletine

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Exeltis Healthcare

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement June 03, 2020

            Details:

            Under the agreements, Exeltis Healthcare will commercialize NaMuscla® in Spain and Portugal, Cresco Pharma B.V will commercialize NaMuscla® in the Netherlands and Macure Pharma ApS in the Nordic countries. Lupin will continue commercialization of NaMuscla® in Germany and UK.

            PharmaCompass