Achillion Pharmaceuticals

Patients and Clinicians

Achillion Pharmaceuticals, Inc. is a complement focused, patient-driven company. Our mission is to transform the lives of patients and families affected by rare diseases of the complement system. We are committed to delivering on the promise of complement-based therapeutics, including our portfolio of factor D inhibitor compounds, as well as educating and supporting the patient and healthcare communities. Achillion is currently conducting global, Phase 2 clinical trials to investigate oral, factor D inhibitors as a potential treatment strategy for patients with C3 glomerulopathy and paroxysmal nocturnal hemoglobinuria (PNH). Additional information on the ongoing clinical trials and available resources are provided below. Learn more about Achillion.

C3 GLOMERULOPATHY (C3G)

For information on Achillion’s C3G clinical trials, please visit:

  • Proof of Concept Study for a 12 Month Treatment in Patients With C3 Glomerulopathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN): NCT03459443
  • Proof of Concept Study for 6 Month Treatment in Patients With C3 Glomerulopathy (C3G): NCT03369236
  • Proof-of-Mechanism Study to Determine the Effect of ACH-0144471 on C3 Levels in Patients With C3G or IC-MPGN: NCT03124368

WeC3G Patient Support Initiative

  • Unites voices of community impacted by disease
  • Connects patients and caregivers to each other and to information, support and resources that can shine the light on C3G
  • Raises awareness and understanding

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Natural History Study

  • Ongoing study conducted by Imperial College of London in up to 400 patients globally
  • Retrospective and prospective data collection to track natural course of disease over 4 years

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C3G Patient-Focused Drug Development

  • First PFDD meeting focused on a renal disease led by the NKF and FDA
  • Sponsored by Achillion with a goal of understanding the C3G patient and caregiver experience and perspective

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PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)

For additional information on Achillion’s PNH clinical trials, please visit:

  • A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to Eculizumab (PNH): NCT03472885
  • A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)): NCT03053102
  • A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH): NCT03181633