Sarepta Therapeutics, Inc. (SRPT) announced that it has dosed the first patient in an open label study, 4658-204, on its lead exon-skipping therapeutic candidate, eteplirsen. Sarepta is evaluating the candidate for the treatment of Duchenne muscular dystrophy (DMD) in non-ambulatory patients or those who have advanced DMD and do not meet a minimum 6-minute walk test score at baseline.
The study will evaluate the safety of eteplirsen in approximately 20 patients suffering from DMD over a span of 96 weeks of dosing. The patients enrolled in the study will be administered once weekly intravenous infusions of 30mg/kg of eteplirsen.
We remind investors that late last month, Sarepta had received updated guidance from the FDA regarding eteplirsen. The FDA stated that results from an independent assessment of dystrophin images as well as 168-week data from Study 202 have to be included in the submission for marketing approval.
Moreover, the FDA pointed out that it requires specific data like a minimum duration of safety in new patients treated with eteplirsen and patient-level natural history data to be obtained by Sarepta from independent academic institutions. Also, MRI data from a recent study conducted by an independent academic group needs to be included apart from other specific data.
Given the FDA’s additional requirements, Sarepta now expects to submit a new drug application (NDA) for eteplirsen by mid-2015.
Currently, Prosensa Holding (RNA) is also developing drisapersen for the treatment of DMD. Last month, the company initiated the process of a rolling NDA submission for drisapersen.
We note that Sarepta currently does not have any approved product. The successful development and commercialization of eteplirsen is crucial for the company. We expect investor focus to remain on updates pertaining to eteplirsen.
Investors looking for well-ranked stocks in the health care sector can consider Illumina Inc. (ILMN) and Celgene Corporation (CELG). While Illumina is a Zacks Rank #1 (Strong Buy) stock, Celgene carries a Zacks Rank #2 (Buy).
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