10 Feb Pfizer
There are over 10,000 known rare diseases that affect approximately 400 million people worldwide. 80% of these diseases have genetic origins and 50% affect children. Collectively, people living with a rare disease represent one of the largest underserved patient communities in the world, with less than 10% of known rare diseases having one or more approved treatments. At Pfizer, we believe that people living with a rare disease, along with the untold number of family members and caregivers who support them, deserve more. For more than thirty years, we have provided critical treatment options for patients with rare diseases. Our current focus spans four therapeutic areas, including 1) rare hematology, 2) rare endocrine/metabolic, 3) rare neurology and 4) rare cardiology. Ten Pfizer Rare Disease medicines have received regulatory approval, and we are committed to deliver a new generation of breakthrough treatments for rare diseases across a diverse range of therapeutic modalities – small molecule, large molecule/monoclonal antibodies (mAbs), gene therapies and even devices. Pfizer’s Rare Disease pipeline includes five new molecular entities in Phase 3 clinical programs, including three gene therapy programs. With our late-stage pipeline and industry-leading global commercialization and development capabilities, Pfizer has the potential to launch a new rare disease medicine every year from 2023 to 2027.