The Food and Drug Administration has granted Breakthrough Therapy designation to danicopan (Achillion Pharmaceuticals), an oral factor D inhibitor, for use in combination with a C5 monoclonal antibody, in the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

The designation was based on positive safety and efficacy data from an ongoing phase 2 study evaluating the effectiveness of danicopan in improving anemia, as measured by blood hemoglobin, when given with eculizumab in patients with PNH (N=11). Eculizumab (Soliris; Alexion) is currently approved for the treatment of PNH to reduce hemolysis.

Results showed a mean increase in hemoglobin of 2.6g/dL in patients who completed 24 weeks of treatment, as well as a reduction in blood transfusions (34 transfusions [total 58 units] in the 24 weeks prior to screening vs 1 transfusion [2 units] during treatment with danicopan). Moreover, improvements in bilirubin, reticulocyte count, and Functional Assessment of Chronic Illness Therapy (FACIT) fatigue scores were also noted.

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“Danicopan, with its demonstrated ability to limit both intravascular and extravascular hemolysis with oral administration, has the potential to benefit a significant number of patients with PNH that continue to have an unmet medical need on standard of care,” said Joe Truitt, President and Chief Executive Officer at Achillion. “We appreciate the review and decision by the FDA and plan to work closely with the Agency in advancing the development of danicopan into phase 3 in early 2020.”

For more information visit achillion.com.