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Fibrocell Up on FDA Designation for Gene Therapy Candidate

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Fibrocell Science, Inc. (FCSC - Free Report) announced that the FDA has granted Regenerative Medicine Advanced Therapy (“RMAT”) designation to its gene therapy candidate, FCX-007. The candidate is being developed for the treatment of recessive dystrophic epidermolysis bullosa ("RDEB"), a rare, genetic skin disorder.

The RMAT designation was created under the 21st Century Cures Act and is granted to speed up the development and review of gene therapies that target serious or life-threatening conditions. The designation also indicates that FCX-007 can be eligible for accelerated approval and priority review depending upon certain criteria. The designation is similar to Breakthrough Therapy designation granted to drugs.

The candidate already enjoys Orphan Drug, Rare Pediatric Disease and Fast Track designations in the United States.

Shares of Fibrocell rallied 10.7% on May 29 following the announcement. However, so far this year, Fibrocell’s shares have gained 25% compared with the industry’s increase of 0.8%.

 

 

Currently there are no approved treatments for RDEB. FCX-007 can be a potentially transformative treatment option for the given skin disorder if it wins approval. In March, the FDA provided guidance on the design aspects of Fibrocell’s proposed phase III study on FCX-007 named DEFI-RDEB in a Type-B meeting. The company expects to initiate the evaluation in second-quarter 2019.

We remind investors that Fibrocell is developing the candidate in collaboration with Intrexon Corporation (XON - Free Report) under its exclusive channel collaboration. Moreover, Fibrocell collaborated with privately held Castle Creek Pharmaceuticals to develop/commercialize FCX-007. Fibrocell is eligible to receive upfront as well as milestone payments from Creek Pharmaceuticals.

Fibrocell is developing another gene therapy candidate, FCX-013, for the treatment of moderate-to-severe localized scleroderma. The company is currently enrolling in the phase I portion of a phase I/II program on FCX-013 and expects to complete the task in the third quarter of 2019.

Zacks Rank & Key Picks

Fibrocell currently carries a Zacks Rank #2 (Buy). A couple of other top-ranked stocks from the biotech sector include Anika Therapeutics Inc. (ANIK - Free Report) and Axovant Sciences Ltd. (AXGT - Free Report) , both sporting a Zacks Rank #1 (Strong Buy).You can see the complete list of today’s Zacks #1 Rank stocks here.

Anika’s earnings estimates have been revised 4.9% upward for 2019 and 9.9% for 2020 over the past 60 days. The stock has gained 13.3% so far this year.

Axovant’s loss estimates have narrowed 3.5% for 2019 and 12.2% for 2020 over the past 60 days.

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