Shire plc has announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation for the investigation of Cinryze (C1 esterase inhibitor [human]) for intravenous administration in subjects with antibody mediated rejection (AMR) in renal transplant recipients.
Cinryze is being studied as an adjunct treatment to donor specific antibodies (DSA) reduction therapy in kidney transplant patients with acute AMR.
“There are currently no approved therapies for antibody mediated rejection, a life-threatening and debilitating condition which can manifest in patients receiving kidney transplants,” said Philip J. Vickers, Ph.D., head of research and development at Shire.
"The Fast Track designation represents an understanding of the significant unmet medical need for this condition. Shire looks forward to working closely with the FDA as we continue to study Cinryze as a potential treatment option for these patients.”
Shire is planning a phase 3 multi-center, multi-national, randomized, double-blind, placebo-controlled study (SHP616-302) to evaluate the efficacy of Cinryze (intravenous administration) as an adjunct to DSA reduction therapy (plasmapheresis, plasma exchange, and/or immune adsorption treatments and IVIG) for the treatment of acute AMR in kidney transplant recipients. The trial will be conducted in the United States, Europe and Canada and the study will open for enrollment by the end of October 2015.
The FDA's Fast Track programme is designed to facilitate the development and expedite the review of drugs that address serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Fast Track designation may provide increased opportunities to interact and meet with FDA, and potentially increases the likelihood of being eligible for priority review if relevant criteria are met.
Cinryze is currently only approved for, and should only be used to treat, routine prophylaxis against angioedema attacks in adolescent and adult patients with Hereditary Angioedema (HAE).
AMR is a condition that sometimes occurs in patients following solid organ transplantation and is initiated by the presence of DSA in the transplant recipient. DSA identified prior to transplant (pre-formed) may be a contraindication to transplantation due to the risk of AMR, however DSA can also form after a transplant takes place. AMR can be life threating and debilitating to the transplant recipient, often times resulting in declining organ function and reduced graft survival.
Cinryze is currently approved for routine prophylaxis against angioedema attacks in adolescent and adult patients with HAE. HAE is a rare genetic disease characterized by recurrent sudden attacks of swelling of the skin or the mucous membranes which can be disfiguring, painful and potentially life-threatening in the case of laryngeal attacks.