Today we announced an important development regarding FDA approval of our gene therapy. Watch to hear a message from our CEO, Doug Ingram:
This drug was rejected by every FDA scientific advisory review: it was found not to be curative nor alter the course of this tragic disease; it met no clinical endpoints; it led to only small increases in surrogate endpoint, which is expression of dystrophin minigene protein that bears minor resemblance to the Becker’s gene product; serious adverse effects including reactions to adenovirus vector, which precludes therapy from future, efficacious gene therapy; and serious liver damage and other concerns. Meanwhile, it costs millions of dollars for treatment. (from report): “…Three FDA review teams and two top officials recommended Sarepta’s application be rejected due to insufficient and conflicting clinical data. They were overruled by Marks, head of the FDA center that reviews gene therapies, who found the results supportive enough to broaden Elevidys’ label. It’s now cleared for Duchenne patients over the age of 4 with mutations to a specific gene, regardless of whether they can still walk.”
Fantastic news! It is wonderful to see that the range of patients who can benefit has expanded, bringing more hope to even more families! ❤️
Wonderful news for patients and a hearty congratulations to the dedication of so many that made this possible! Keep shining on! ❤️
“Today is a good day” Precisely. Congrats again, such incredible progress.
Today is definitely a good day! Thank you and congrats to all who were involved with this!
I am overjoyed to hear this news! So many lives will be changed. Congrats!
This is wonderful news! Congratulations to affected families and the entire Sarepta team.
Congratulations!!! A great day for patients!!!
Senior Talent Acquisition Specialist for Diagnostic & Genomics Segment at Bio-Techne.
1moExciting to see how far Sarepta has come from a mom and pop shop, based out of Washington State, to being the leader in DMD care. #Thankful