NEWS

FDA approves long-acting version of eculizumab-Ravulizumab

Release time:

2019-02-24 23:10

2018Year12Month21day,FDAApprovedAlexion PharmaceuticalsNew drugs developed by the companyULTOMIRIS(Ravulizumab) marketed for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), the drug is the first long-actingC5Complement inhibitors.FDAOriginally scheduled2019Year2Month18Day before.ULTOMIRISThe approval was made two months earlier than expected.

In the past, the rare disease drug eculizumab (trade nameSoliris) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (AHUS) and resistanceAchRAntibody-positive myasthenia gravis (gMG). eculizumab2007First listed in the United States,2018Year09Month04The U.S. patent will be granted in China.2027years due, whileULTOMIRISThe successful listing is expected to extend the patent2035Year.

 

Nocturnal paroxysmal hemoglobinuria (PNH)

Nocturnal paroxysmal hemoglobinuria (Paroxysmal nocturnal hemoglobinuria, PNH) is a rare blood disease characterized by complement-mediated red blood cell destruction (hemolysis), due1One or more hematopoietic stem cells via acquired somatic cellsPIG-AGene (Phosphotidyl inositol glycan complementing group A) non-malignant clonal disease caused by mutation,PIG-AMutations cause glycosylphosphatidylinositol (Glycosyl phosphatidyl inositole,GPI) synthesis anomalies, resulting inGPILoss of a group of membrane proteins anchored to the cell membrane, includingCD16,CD55,CD59The main clinical manifestations are chronic intravascular hemolysis, aplastic anemia and repeated thrombosis, which can cause extensive debilitating symptoms and complications. The disease can occur in the whole body and lead to organ damage and premature death.

 

Long-acting version of eculizumab- Ravulizumab

English name:Ravulizumab

Trade Name:ULTOMIRISTM

Ravulizumab(ALXN1210) is the first and currently the only long-acting complement inhibitor, which inhibits the terminal complement cascade by inhibitingC5protein to prevent the occurrence of hemolysis;C5The protein is part of the body's immune system, and when it is activated uncontrollably, it triggersPNHHemolytic uremic syndrome(AHUS)Anti-acetylcholine receptor(AchR)And antibody positive myasthenia gravis and other serious rare diseases.

InPNHindications in clinical trials,RavulizumabwithSoliriscompared to non-inferiority (allp < 0.0006），PNHpatients from each2Use eculizumab once a week and change it to every8use once a weekRavulizumabThe effect is not reduced, especially.RavulizumabSubcutaneous injection formulations are being developed, which have a significant impact onRavulizumabIt has market development potential.

Except approvedPNHOutside the indications,RavulizumabPhase III clinical trials for hemolytic uremic syndrome are also currently underway. In addition, clinical trials are planned for the treatment of myasthenia gravis and the feasibility of subcutaneous injection.

 

Patent Information

Through the search found that there are7Related U.S. Patents and2Chinese family patents, each of which has1The patent has not been granted. The patent containsFcSegment genetic engineering improvementFc-FcRnInPH 6.0Under the affinity, the half-life of the antibody was extended by the genetic engineering scheme.

Mab, buildermab, pnh