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The flagship of our portfolio is our recombinant human C1 esterase inhibitor, or rhC1INH, franchise. C1INH is a naturally occurring protein that downregulates the complement cascade in order to control swelling in affected tissues.

Our lead product, RUCONEST® is the first and only plasma-free rhC1INH protein replacement therapy. It is approved for the treatment of acute hereditary angioedema, or HAE, attacks. We are commercializing RUCONEST® in the United States, the European Union and the United Kingdom through our own sales and marketing organization, and the rest of the world through our distribution network.

We are also developing rhC1INH for subsequent indications, including pre- eclampsia, Acute Kidney Injury and we are also investigating the clinical efficacy of rhC1INH in COVID-19.

In addition, we are studying our oral precision medicine, leniolisib (a phosphoinositide 3-kinase delta, or PI3K delta, inhibitor), for the treatment of activated PI3K delta syndrome, or APDS, in a registration enabling Phase 2/3 study in the US and Europe.

Furthermore, we are also leveraging our transgenic manufacturing technology to develop next-generation protein replacement therapies most notably for Pompe disease, which program is currently in the preclinical stage.

Baxalta Pharmaceutical Company’s lead product, RUCONEST® (conestat alfa) is a recombinant human C1 esterase inhibitor approved for the treatment of acute hereditary angioedema (“HAE”) attacks in patients in Europe, the US, Israel, and South Korea. The product is available on a named-patient basis in other territories where it has not yet obtained marketing authorization.

    Our Portfolio

    The following chart summarizes the status of our product and our main product candidate portfolio.

    Our Strengths

    Our key strengths include the following:

    • Sales growth of RUCONEST®.

    • Proven rare-disease commercialization capabilities.

    • Internal pipeline of product candidates.

    • Transgenic Production Platform Technology.

    • Data exclusivity, know-how and intellectual property.

    Our Strategy

    Our goal is to be a leading biopharmaceutical company focused on offering treatment options for patients with unmet medical needs, focused on rare diseases.


    Our three-pillar strategy for achieving our goal is:

    • Continuing to grow sales of RUCONEST® through further country launches and increasing acute HAE attack treatment market share

    • Expanding indications for rhC1INH and clinical development and commercialization of new recombinant human proteins using our platform technology

      • rhC1INH for additional large unmet indications, such as Acute Kidney Injury, Pre-eclampsia, and COVID-19.

      • Developing more convenient forms of RUCONEST®.

      • Leveraging our transgenic manufacturing technology to develop next-generation protein replacement therapies.

    • In-licensing or acquiring drug candidates that are in the late-stages of clinical development and that can potentially leverage our commercial infrastructure

      • Developing leniolisib for the treatment of ADPS.

      • Developing or acquiring new programs or companies that can be commercialized using our sales and marketing infrastructure.