Conestat alfa is under clinical development by Pharming Group and currently in the Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect Conestat alfa’s likelihood of approval (LoA) and phase transition for Delayed Graft Function (DGF) took place on 26 Dec 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway and increased the likelihood of final approval for this indication.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Conestat alfa Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

Conestat alfa overview

Conestat alfa (Ruconest/Rhucin) is the first recombinant analogue of the human C1 esterase inhibitor (rhC1INH) produced by recombinant DNA technology in the milk of transgenic rabbits.The amino acid sequence of conestat alfa is identical to that of endogenous C1INH. It is formulated as a powder for solution injection for intravenous administration. It is indicated for the treatment of acute attacks of angioedema in adults and in children with hereditary angioedema (HAE) due to deficiency of c1 esterase inhibitor.

Conestat alfa is under development for the treatment of prophylaxis of hereditary angioedema, acute kidney injury, prevention of acute ischemic cerebral and renal events after transcatheter aortic valve implantation, delayed graft function (kidney transplantation rejection), contrast-induced nephropathy, hemorrhagic shock and cardiac protection pre-eclampsia and coronavirus disease 2019 (COVID-19) associated acute respiratory distress syndrome and covid-19 pneumonia. The drug candidate is administered through intravenous, intramuscular, intradermal and subcutaneous routes. It was under development for the treatment of acute pancreatitis.

Pharming Group overview

Pharming Group (Pharming) develops precision medicines and innovative protein replacement therapies for the treatment of rare diseases and unmet medical needs. The company’s marketed product includes, Ruconest (conestat alfa), a recombinant human C1 inhibitor is used for the treatment of acute hereditary angioedema (HAE) and has been approved in the US, the UK, Europe, Israel, and South Korea. Its product pipeline consists of rhC1INH for the treatment of pre-eclampsia, COVID-19 and acute kidney injury; leniolisib for treatment of activated phosphoinositide 3-kinase delta syndrome (APDS); a-Glucosidase for the treatment of Pompe and Fabry’s disease. The company markets its products through own sales force and network of distributors and partners. Pharming is headquartered in Leiden, the Netherlands.

Quick View Conestat alfa LOA Data

Report Segments
  • Innovator (NME)
Drug Name
  • Conestat alfa
Administration Pathway
  • Intradermal
  • Intramuscular
  • Intravenous
  • Subcutaneous
Therapeutic Areas
  • Cardiovascular
  • Gastrointestinal
  • Genito Urinary System And Sex Hormones
  • Immunology
  • Infectious Disease
Key Developers
Highest Development Stage
  • Marketed

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.