Paroxysmal Nocturnal Hemoglobinuria Investigative Therapy Moving to Phase 3 Trial in 2020

The EMA has just announced that Danicopan has been accepted into its PRIority MEdicines (PRIME) program. Danicopan is being developed for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. PRIME designation will help to accelerate development of this therapy. PRIME designation means the EMA has recognized this medicines potential benefit for a rare disease with a high unmet need.


Danicopan is being developed by Achillion Pharmaceuticals. Specifically, it has been created for PNH patients who have not responded to a C5 inhibitor. A C5 inhibitor is the current standard of care. Currently however, approximately 75% of patients treated with this therapy remain anemic. Essentially, their symptoms are only partially controlled. Researchers believe this new therapy could be more effective.

The therapy has already been granted Breakthrough Therapy Designation and Orphan Drug Designation by the FDA. Additionally, it has received Orphan Drug Status by the EMA.

The PRIME decision was the result of promising data from a Phase 2 combination trial. Full results from this 24-week trial will be presented at the American Society of Hematology Annual Meeting. This event will occur in Orlando, Florida on December 9th, 2019. The hope is that with the help of this new program, Danicopan will be in the hands of patients sooner.

Looking Forward 

Achillion will now work with the EMA to continue to develop Danicopan through their program. They hope that in early 2020 they will be able to launch a Phase 3 trial for the treatment. This trial will be global in scope.

Additionally, Achillion is working to develop this therapy for other diseases for which they believe it could be effective. These include immune complex membranoproliferazive glomerulonephritis (IC-MPGN) and C3 Glomerulopathy (C3G).

You can read more about this new development and Danicopan’s potential benefits  here.

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