- In November 2024, Amgen announced the presentation of new data from its rare disease portfolio and pipeline at the American College of Rheumatology (ACR) Convergence 2024 conference in Washington, D.C. The new findings highlight a reduction in disease activity with UPLIZNA® (inebilizumab-cdon) in Immunoglobulin G4-Related Disease (IgG4-RD) and provide support for shorter infusion times of KRYSTEXXA (pegloticase) when co-administered with weekly oral methotrexate at 15 mg
- In October 2024, JCR Pharmaceuticals Co., Ltd. announced the start of patient dosing in Japan for the Phase I clinical trial of JR-441, an investigational enzyme replacement therapy aimed at treating mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A. MPS IIIA is a rare genetic disorder marked by severe central nervous system (CNS) symptoms
- In July 2023, Alexion, AstraZeneca Rare Disease, reached an agreement with Pfizer to acquire a portfolio of preclinical gene therapies for rare diseases. As part of the deal, Alexion will gain access to several novel adeno-associated virus (AAV) capsids. AAV capsids have demonstrated effectiveness in delivering therapeutic gene cargos for both gene therapy and gene editing.
- In November 2022, AstraZeneca announced that Soliris (eculizumab), the first rare disease therapy from its Alexion, AstraZeneca Rare Disease group, is now available in China. This marks a significant expansion of the company's global presence in the rare disease field, as Soliris is now available for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in both adults and children in China
- In September 2020, AbbVie announced that the U.S. Food and Drug Administration (FDA) had granted both Orphan Drug and Fast Track designations to elezanumab (ABT-555), an investigational treatment for patients with spinal cord injury. Orphan Drug Designation is awarded to a drug or biologic intended for the treatment, diagnosis, or prevention of a rare disease or condition
