Horizon’s FA Therapy Granted FDA Fast Track Designation

Specialty biopharmaceutical Horizon Pharma plc has been awarded Fast Track status from the U.S. Food and Drug Administration (FDA) for the company’s ACTIMMUNE (interferon gamma-1b) to be used in patients with Friedreich’s ataxia (FA). ACTIMMUNE is a biologically developed protein resembling one produced naturally by the body to prevent infection, which is already approved for two other indications.

The FDA has granted the Fast Track designation to ACTIMMUNE with the purpose of giving the company more direct access to the administration in order to accelerate the treatment’s development process. The status is awarded to experimental therapies that may be able to effectively treat severe or life-threatening conditions, and offer great potential in addressing unmet medical needs.

As a result of the designation, Horizon will now work closely with the FDA, which may expedite the approval of ACTIMMUNE as a treatment for FA. The status also gives the company the ability to submit sections of the registration dossier for the target indication on a rolling basis, as well as to be eligible for priority review after the submission process and forthcoming clinical data.

“We commend the FDA for granting Fast Track designation for ACTIMMUNE in Friedreich’s ataxia to potentially help treat patients suffering from this debilitating disorder, for which there are no FDA approved treatments available,” stated the chairman, president and CEO of Horizon Pharma plc, Timothy P. Walbert, in a press release. “We look forward to continuing our ongoing clinical development program as we evaluate ACTIMMUNE in this patient population.”

The biological protein ACTIMMUNE has been shown to be effective in decreasing the frequency and severity of serious infections and has already been approved by the FDA to treat both the genetic conditions Chronic Granulomatous Disease (CGD) and malignant osteoporosis (SMO). Horizon is studying the use of the treatment in patients with FA, a debilitating, life-shortening and degenerative neuro-muscular disease diagnosed in one in every 50,000 people in the US alone.

Horizon has submitted an Investigational New Drug (IND) application for ACTIMMUNE for FA patients last February and is now planning on starting phase 3 clinical studies during the second quarter of the year. The studies will be undertaken in partnership with the Friedreich’s Ataxia Research Alliance (FARA) and a sub-set of FARA’s Collaborative Clinical Research Network (CCRN).

The FDA has already agreed on the primary endpoint for the phase 3 study dedicated to the potential FA therapy, approving the alteration from baseline after 26 weeks in the Friedreich’s Ataxia Rating Scale-modified neurological exam score (FARS-mNeuro) for patients treated with ACTIMMUNE compared to placebo, as a primary endpoint for the phase 3 study.