Achillion Pharma PNH treatment Danicopan gets European Medicines Agency PRIME Designation

New Delhi: Achillion Pharmaceuticals, Inc. a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients and families affected by complement-mediated diseases, recently announced that the European Medicines Agency (EMA) has granted access to support through the PRIME (PRIority MEdicines) program for Danicopan (ACH-4471) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in patients who are not adequately responding to a C5 inhibitor.

The decision from the EMA was based on Danicopan safety and efficacy data from the Phase 2 PNH combination trial. The top-line 24-week results from this combination trial was accepted for poster presentation at the 61stAmerican Society of Hematology (ASH) Annual Meeting scheduled for December 9th in Orlando, FL.

“We are very pleased that the European Medicines Agency has granted PRIME designation for Danicopan, our lead oral factor D inhibitor,” said Dr. Kevin P. Malobisky, Senior Vice President Regulatory Affairs, Quality & Compliance. “The EMA’s decision further highlights the unmet medical need for PNH patients who are suboptimal responders to a C5 inhibitor, the current standard of care. We appreciate the review and decision by the EMA and plan to continue working closely with the Agency through Scientific Advice to advance the development of Danicopan into a global Phase 3 trial in early 2020.”

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The EMA launched the PRIME program to ensure that promising medicines that may provide a therapeutic advantage over existing treatments have a pathway to accelerate development through early interaction and dialogue. The program is intended to optimize development plans and expedite the review and approval process so that these medicines may reach patients as early as possible. The Agency will confirm eligibility to the centralized procedure at the time of a marketing authorization application (MAA) and will appoint a Rapporteur from the Committee for Medicinal Products for Human Use (CHMP) to provide continuous support and help to build knowledge ahead of a marketing authorization application. To be eligible for PRIME, medicines must target an unmet medical need and show potential benefit for patients based on early clinical data. Danicopan (ACH-4471) has previously received orphan drug designation and Breakthrough Therapy designation from the U.S. Food & Drug Administration (FDA) for the treatment of PNH, and orphan status from the European Medicines Agency.

PNH is a rare, acquired blood disease caused by a somatic mutation resulting in the absence of key receptors, CD55 and CD59, on the surface of red blood cells (RBCs).

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