Alpha-1 proteinase inhibitor (human) is under clinical development by CSL and currently in the Phase III 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 Alpha-1 proteinase inhibitor (human)’s likelihood of approval (LoA) and phase transition for Graft Versus Host Disease (GVHD) took place on 19 Jul 2021, which decreased the likelihood that the drug progresses to the next phase in its clinical pathway and decreased the likelihood of final approval for this indication.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Alpha-1 proteinase inhibitor (human) Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

Alpha-1 proteinase inhibitor (human) overview

Alpha1-proteinase inhibitor, human (Zemaira/ Respreeza) also known as alpha1-antitrypsin, belongs to the serpin superfamily prepared from human plasma via Cohn alcohol fractionation followed by PEG and zinc chloride fractionation. It is formulated as injectable, powder, lyophilized, for solution for intravenous route of administration. Zemaira is indicated for chronic augmentation and maintenance therapy in adults with A1-PI deficiency and clinical evidence of emphysema. Respreeza is indicated for maintenance treatment, to slow the progression of emphysema in adults with documented severe alpha1-proteinase inhibitor deficiency (eg.genotypes PiZZ, PiZ(null), Pi(null,null), PiSZ).

It is under development for the treatment of steroid refractory acute graft vs host disease and eosinophilic esophagitis.

CSL overview

CSL discovers, develops, manufactures, commercializes and distributes biopharmaceuticals and related products. Its portfolio encompasses plasma-derived products for the treatment of hemophilia, hereditary angioedema, von Willebrand disease, inherited primary and secondary immune deficiencies, respiratory disease, neurological disorders and protein-based medicines for treating serious human illnesses. The company also provides products for the prevention of hemolytic disease in newborns, infection in solid organ transplant recipients and to treat specific infections for victims of trauma and burns. CSL markets an array of vaccines, anti-venoms and pharmaceutical products of various other manufacturers. The company sells its products in Australia, Germany, Switzerland, the US, the UK and other countries. CSL is headquartered in Parkville, Victoria, Australia.

Quick View Alpha-1 proteinase inhibitor (human) LOA Data

Report Segments
  • Innovator (NME)
Drug Name
  • Alpha-1 proteinase inhibitor (human)
Administration Pathway
  • Intravenous
Therapeutic Areas
  • Gastrointestinal
  • Genetic Disorders
  • Immunology
  • Respiratory
Key Developers
  • Sponsor Company: CSL
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.