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FDA Lifts Clinical Hold on Sarepta's DMD Gene Therapy Program

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Sarepta Therapeutics, Inc. (SRPT - Free Report) announced that the FDA has lifted the clinical hold for the company’s Duchenne muscular dystrophy (DMD) micro-dystrophin gene therapy program. The clinical hold on the phase I/IIa clinical study evaluating the micro-dystrophin gene therapy was put by the FDA in July 2018 due to the presence of trace amounts of DNA fragment in research-grade third-party supplied plasmid.

The company then developed an action plan along with Nationwide Children’s Hospital and submitted the same to the FDA. The plan included an audit of the plasmid supplier used for research and a commitment to use only plasmid produced under good manufacturing practice (GMP) for all future production lots. The company intends to hold a meeting with the FDA so that it can start the study again by end of 2018.

The shares of the company rose 3.7% in response to the news. Year to date, the company’s shares have increased 175.2% against the industry’s decline of 2.7%.

 

 

One of the most common fatal genetic disorders affecting children around the world, DMD is a devastating and incurable muscle-wasting disease. There is a significant unmet need for DMD treatments. Thus, Sarepta is looking to build its DMD pipeline beyond its sole marketed product, Exondys 51, by developing other exon-skipping treatments. In fact, the company has about eight exon-skipping candidates in its pipeline, including golodirsen and casimersen. If approved, these candidates will be eligible to treat 75-80% of the DMD population.

However, we remind investors that, this week, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) upheld its earlier negative opinion for marketing approval of Exondys in EU, which was earlier announced on May 31. The company said it will seek scientific advice from the EMA to get approval for Exondys in Europe. A decision is expected by end of the year. Sarepta is expecting the EC to adopt the CHMP’s opinion.

Sarepta Therapeutics, Inc. Price

Zacks Rank & Stocks to Consider

Sarepta is a Zacks Rank #3 (Hold) stock.

Some better-ranked stocks in the biotech sector are Anika Therapeutics Inc. (ANIK - Free Report) , Ligand Pharmaceuticals Incorporated (LGND - Free Report) and Regeneron Pharmaceuticals Inc. (REGN - Free Report) . While Anika and Ligand sport a Zacks Rank #1 (Strong Buy), Regeneron carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Anika’s earnings per share estimates have increased from 57 cents to 95 cents for 2018 and $1.02 to $1.49 for 2019 over the past 60 days. The company delivered a positive earnings surprise in two of the trailing four quarters with an average beat of 20.84%.

Ligand’s earnings per share estimates have moved up from $5.64 to $6.33 for 2018 and $5.59 to $5.74 for 2019 in the last 30 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 59.54%. Share price of the company has increased 96% year to date.

Regeneron’s earnings per share estimates have increased from $18.99 to $20.38 for 2018 and $21.15 to $21.74 for 2019 over the past 60 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 8.18%. The stock has rallied 3.8% so far this year.

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