The FDA has awarded priority review status to Sarepta Therapeutics' eteplirsen. The regulatory go sign means Sarepta is still tucked in just behind BioMarin in the race to bring a therapy for Duchenne muscular dystrophy to market, with analysts tipping the agency to make a decision on both drugs by the end of the year.
Rivals Sarepta Therapeutics and BioMarin Pharmaceutical are in line to become the next recipients of an FDA coupon for a fast review, each potentially picking up a sellable asset that recently brought in $350 million on the open market.
Sarepta, ending a protracted process that may have cost its last CEO his job, submitted its treatment for Duchenne muscular dystrophy to the FDA, setting the stage for a possible approval early next year.
The FDA has given Sarepta Therapeutics the go-ahead to start a rolling submission for its Duchenne muscular dystrophy drug, eteplirsen. The green light comes 7 weeks after Sarepta parted company with its CEO Chris Garabedian--and his interim successor was quick to frame the regulatory breakthrough as the result of the subsequent shift in strategy.
Cambridge, MA-based Catabasis is once again raising cash. According to a Form D filed with the SEC yesterday, the biotech has raised $12.4 million out of a $20.4 million raise.
The U.K.'s Summit Therapeutics is elbowing its way into the race to treat Duchenne muscular dystrophy, pulling off a $34 million U.S. IPO to fund its research.
Investigators at Duke have turned to a new gene-editing tool to come up with a new approach to Duchenne muscular dystrophy that theoretically could apply to a majority of the boys afflicted with the fatal rare disease.
SINGAPORE-- Several Japan drugmakers are making strides toward being major pioneers in the new pharmaceuticals field of nucleic acid-based treartments. A few already have clinical trials in the works for such things as Duchenne muscular dystrophy.
A biotech with scientific roots at the University of Oxford and a new approach to treating Duchenne muscular dystrophy is coming to America with a $40 million IPO.
Scientists say that they have developed a method to use modified messenger RNA to lengthen telomeres, opening a pathway to countering the effects of aging in cells--a process that might apply to a range of diseases from DMD to diabetes and heart disease.