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Sarepta Therapeutics, Inc.

Latest From Sarepta Therapeutics, Inc.

Top FDA Staff Are Pushing Review Teams To Be Comfortable With Regulatory Flexibility

Senior leadership in the FDA’s drugs, biologics and devices centers want reviewers to feel more comfortable taking risks in product approvals, but may need more concrete examples of regulatory flexibility's success to convince them.

Drug Approval Standards Rare Diseases

First Four Members Of US FDA’s Newest Advisory Committee Are Mostly Rookies

Most of the new Genetic Metabolic Diseases Advisory Committee members have little FDA or advisory committee experience, except the new industry representative, with six more voting slots still to fill before its first meeting.

Rare Diseases Advisory Committees

Boehringer Looks To Revitalise Its Pipeline

With sales of its big sellers Jardiance and Ofev to start shrinking in the next few years, the German group is teeing up a suite of new drugs.

Sales & Earnings Companies

Rare Diseases Rule, But Some Common Conditions Stand Out In Pending US Applications

Half of the FDA’s upcoming 2024 user fee goals target rare diseases, but highly prevalent conditions like uncomplicated urinary tract infections and atopic dermatitis are also up for agency action in the second half of the year.

Rare Diseases Drug Approval Standards
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Company Information

  • Industry
  • Pharmaceuticals
    • Vaccines
  • Biotechnology
    • Antisense, Oligonucleotides
    • Gene Therapy, Cell Therapy
    • Large Molecule
  • Other Names / Subsidiaries
    • AVI BioPharma, Inc.
    • Ercole Biotech, Inc.
    • Myonexus Therapeutics, Inc.