J&J's Nipocalimab Receives Fast Track Designation from FDA for Rare Disease
Developed by J&J, nipocalimab reduces the risk of FNAIT in alloimmunized pregnant individuals
On March 26, 2024, Johnson & Johnson (J&J) shared that the FDA has given fast track designation to nipocalimab. This is an investigational monoclonal antibody (mAb) that aims to reduce the risk of fetal neonatal alloimmune thrombocytopenia (FNAIT) in pregnant adults who have been alloimmunized. As per a press release from the company, nipocalimab is the sole therapy currently being studied to target this specific indication. In December 2023, the FDA granted orphan drug designation to Nipocalimab for the FNAIT indication.
J&J stated in its press release that the mAb is designed to target a specific receptor and decrease the levels of certain antibodies in the body. This could potentially have an impact on various conditions. The company states that Nipocalimab is being studied in various areas of autoantibody diseases. These include rare autoantibody diseases such as generalized myasthenia gravis, chronic inflammatory demyelinating polyneuropathy, warm autoimmune hemolytic anemia, and idiopathic inflammatory myopathies. It is also being investigated for maternal fetal diseases mediated by maternal alloantibodies like hemolytic disease of the fetus and newborn, and fetal and neonatal alloimmune thrombocytopenia.
Additionally, it is being explored for prevalent rheumatology conditions like rheumatoid arthritis, Sjögren’s disease, and systemic lupus erythematosus.
FNAIT is a condition that is both severe and rare. It happens when the immune system of the pregnant person mistakenly targets the platelets of a developing fetus. This can affect the ability to form clots and result in internal bleeding, which poses a serious threat to the fetus or newborn. By blocking FcRn, it is possible to decrease the levels of IgG, which includes pathogenic alloantibodies.
Simultaneously, the obstruction can maintain immune function without leading to a more widespread suppression of the immune system. Preventing the binding of IgG to FcRn in the placenta could potentially stop the transfer of maternal alloantibodies to the fetus, as stated in the press release.
The fast track designation for nipocalimab in FNAIT highlights the importance of finding safe, effective, and targeted treatments to address the urgent need for preventing FNAIT. This condition can have severe health consequences and even be fatal for the fetus or newborn.
Nipocalimab is currently being researched as a potential treatment for hemolytic disease of fetus and newborn (HDFN), which is another alloimmune disease associated with pregnancy. HDFN shares a disease mechanism with FNAIT and is commonly known as the red-blood-cell counterpart to FNAIT. J&J has decided to move forward with Phase III trials that will specifically target HDFN, building upon the positive safety and efficacy findings from the Phase II trial.
Big biopharmaceutical companies are currently placing a significant emphasis on the rare disease sector. As an example, in January 2024, Sanofi took steps to acquire Inhibrx, a biopharmaceutical company in the clinical stage. The agreement is worth around $1.7 billion. Sanofi will acquire several assets, including INBRX-101, a human recombinant protein with the potential to regulate serum alpha-1-antitrypsin (AAT) levels in patients who have AAT deficiency. AAT deficiency is a relatively uncommon genetic disorder characterized by reduced levels of AAT protein. The lungs are primarily affected by symptoms, leading to a gradual decline in tissue health.
Furthermore, AstraZeneca has recently obtained FDA approval for Ultomiris (ravulizumab-cwvz), a therapeutic designed to treat rare diseases. Ultomiris was approved by the FDA on March 25, 2024 for the treatment of adult patients in the United States who have anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a relatively uncommon autoimmune condition that can have a significant impact on a person's health, particularly on their central nervous system, which includes the spine and optic nerves.
Source: Johnson & Johnson; Sanofi; AstraZeneca
