Danicopan is under clinical development by Alexion Pharmaceuticals and currently in Phase I for Atypical Hemolytic Uremic Syndrome (Nondiarrhea – Associated Hemolytic Uremic Syndrome). According to GlobalData, Phase I drugs for Atypical Hemolytic Uremic Syndrome (Nondiarrhea – Associated Hemolytic Uremic Syndrome) have a 100% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how Danicopan’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Danicopan overview

Danicopan (Voydeya) is a first-in-class oral proximal, complement alternative pathway factor D inhibitor. It is formulated as film coated tablets for oral route of administration. It is indicated for the treatment of paroxysmal nocturnal hemoglobinura (PNH).

Danicopan (ACH-4471) is under development for the treatment of coronavirus disease 2019 (COVID-19), paroxysmal nocturnal hemoglobinura (PNH), atypical hemolytic uremic syndrome (aHUS), geographic atrophy and age related macular degeneration. It is administered by oral route. The drug candidate acts by targeting complement factor D.

It was under development for the treatment of C3G including C3 glomerulopathy or immune-complex membranoproliferative glomerulonephritis, dense deposit disease (DDD) and  atypical hemolytic uremic syndrome (aHUS).

Alexion Pharmaceuticals overview

Alexion Pharmaceuticals (Alexion), a subsidiary of AstraZeneca Plc, discovers, develops and markets therapies for severe and life-threatening diseases. The company’s medicinal brands comprise Kanuma, Koselug, Soliris, StrensIQ, Ultomiris and Voydeya. It develops medicines to treat conditions such as atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), hypophosphatasia (HPP), lysosomal acid lipase de?ciency (LAL-D), neurofibromatosis type 1 plexiform neurofibromas (NF1 PN), neuromyelitis optica spectrum disorder (NMOSD) and paroxysmal nocturnal hemoglobinuria (PNH). Alexion’s therapeutic areas include hematology, nephrology, neurology, metabolics, cardiology and others. Alexion collaborates with various research organizations and pharmaceutical companies. The company operates in Europe and the Middle East, North America, Latin America and Asia Pacific. Alexion is headquartered in Boston, Massachusetts, the US.

For a complete picture of Danicopan’s drug-specific PTSR and LoA scores, buy the report here.

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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 clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.