Sarepta Therapeutics, Inc. (SRPT - Free Report) announced that it will seek rapid approval for its second drug, golodirsen, to treat Duchenne Muscular Dystrophy (DMD), the most common type of muscular dystrophy.
The small biotech said it will file a rolling new drug application (NDA) with the FDA, seeking accelerated approval of golodirsen for DMD in patients who have a confirmed mutation of the DMD gene susceptible to exon 53 skipping. The rolling NDA submission is expected to be completed in late 2018, which means that golodirsen could be approved in 2019, much earlier than expected.
Shares of Sarepta shot up more than 6% on Monday, hitting a 52-week high. Sarepta’s shares have risen 42.1% this year so far, significantly better than the industry’s rise of 3% in the same time frame.
Sarepta met with the FDA Division of Neurology Products in February to get directions on the regulatory pathway for golodirsen while the final minutes of the Type C meeting were received last week
In September last year, data presented from the phase I/II 4053-101 study evaluating golodirsen demonstrated 100% response rate with 10.7 times increase in mean dystrophin protein (muscle protein) from baseline. This positive data coupled with the FDA guidance from the Type C meeting formed the basis for initiating the rolling NDA filing.
Meanwhile, ongoing phases III study 4045-301 (ESSENCE) on golodirsen will be treated as the post-marketing confirmatory study
Sarepta’s only approved drug Exondys 51 for treating DMD witnessed impressive sales trends in 2017. The drug generated revenues of $155 million last year. The company expects the drug’s sales to double in 2018.
Please note that Exondys 51 was approved in the United States in September 2016 as a once-weekly intravenous infusion, specifically targeting patients who have a confirmed mutation of the DMD gene susceptible to exon 51 skipping. However, Exondys 51 is not yet approved in Europe.
DMD is a rare genetic disorder caused by the absence of the muscle protein dystrophin, which is needed for muscles to function properly. There is significant unmet need for DMD treatments, a disease that mostly affects male children. Exondys 51 has the potential to address up to 13% of the total DMD population. If golodirsen gains an earlier-than-expected approval, Sarepta can cater to a larger population of DMD patients.
Sarepta carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the health care sector are Regeneron Pharmaceuticals, Inc. (REGN - Free Report) , Ligand Pharmaceuticals Incorporated (LGND - Free Report) and Horizon Pharma Public Limited Company (HZNP - Free Report) . While Regeneron sports a Zacks Rank #1 (Strong Buy), Ligand and Horizon Pharma carry a Zacks Rank #2 (Buy). You can seethe complete list of today’s Zacks #1 Rank stocks here.
Regeneron’s earnings per share estimates have moved up by 9.37% for 2018 and by 6.3% for 2019 in the last 60 days. The company pulled off a positive earnings surprise in three of the last four quarters with an average beat of 9.15%.
Ligand’s earnings per share estimates have moved up by 9.8% for 2018 and 21% for 2019 in the last 30 days. The company delivered a positive surprise in three of the trailing four quarters with an average beat of 24.88%. Share price of the company has surged 71.1% in the past year.
Horizon Pharma’s earnings per share estimates have moved up by 10% for 2018 and 14.9% for 2019 in the last 30 days. Horizon Pharma came up with a positive surprise in three of the last four quarters with an average beat of 68.9%. Share price of the company has risen 2.2% in the past year.
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