Achillion Pharmaceuticals Inc. (NASDAQ: ACHN) has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for danicopan (ACH-4471) in combination with a C5 monoclonal antibody for patients with paroxysmal nocturnal hemoglobinuria (PNH) who are suboptimal responders to a C5 inhibitor alone.

The FDA’s decision was based on positive safety and efficacy data from the ongoing danicopan Phase 2 PNH combination trial. Interim data was reported at the New Era of Aplastic Anemia and PNH Meeting in May 2019. The top-line data from this combination trial is expected in the fourth quarter of 2019.

This designation is designed to expedite the development and review of medicines for serious or life-threatening conditions.

Joe Truitt, president and CEO at Achillion, commented:

The FDA’s granting of Breakthrough Therapy designation for our lead oral factor D inhibitor, danicopan, underscores the urgent need for new treatment options for patients living with PNH. 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. 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.

Shares of Achillion traded up over 6% on Wednesday to $3.84, in a 52-week range of $1.29 to $5.03. The consensus price target is $8.00.

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