FDA Grants Fast Track Status to Fibrocell’s Gene Therapy FCX-013 for Localized Scleroderma

Alice Melão, MSc avatar

by Alice Melão, MSc |

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TLY012 orphan drug status

Fibrocell Science‘s gene therapy candidate FCX-013 received fast track designation by the U.S. Food and Drug Administration as a treatment for moderate-to-severe localized scleroderma, which usually does not affect internal organs.

The FDA’s latest move adds to its previously granted orphan drug and rare pediatric disease designations, which are all expected to support, facilitate, and expedite the clinical development and approval of FCX-013 as a treatment for localized scleroderma.

“Fast Track designation represents an important milestone in advancing clinical development of FCX-013,” John Maslowski, president and CEO of Fibrocell, said in a press release.

“We are pleased the FDA has awarded this designation to FCX-013 which, we believe, has the potential to be the first gene therapy to treat excessive collagen accumulation in the skin and soft tissue at the site of localized scleroderma lesions, and to bring relief from the severe pain and functional disability associated with the disorder,” Maslowski added.

FCX-013 is a gene therapy that was designed to deliver modified cells that carry the MMP1 gene, which provides instructions to make an enzyme that can break down the collagen accumulated in the skin of patients with scleroderma.

This therapy is administrated by injection under the skin, at the site of the localized fibrotic lesions. At the same time, patients will take an oral medicine called Veledimex that will help enhance the production of the enzyme.

As soon as the fibrotic lesions are resolved, the patient will stop taking Veledimex to restore the enzyme levels back to a lower concentration.

This novel targeted gene therapy is being developed by Fibrocell in collaboration with Precigen, a subsidiary of Intrexon.

The FDA has already approved the start of clinical trials to evaluate FCX-013’s safety and effectiveness as treatment for moderate-to-severe localized scleroderma.

According to Fibrocell, in August the company started enrolling participants in a Phase 1/2 clinical trial evaluating FCX-013. The trial is expected to recruit 10 patients with any subtype of localized scleroderma.

The Phase 1 part of the study will include adult patients, and the Phase 2 part is planned to include pediatric patients. The study will only advance to Phase 2 after the clinical data collected in Phase 1 part has been approved by the FDA and a Data Safety Monitoring Board.

The primary goal of the trial is to assess FCX-013’s safety profile. Secondary analyses will evaluate the impact of the treatment on skin lesions, among other efficacy measures, at different time points during the 16 weeks after FCX-013 administration.