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Regulatory

A total of 24 posts are filed under Regulatory
Ionis Submits Application to FDA to Market Antisense Drug for hATTR
Rare Daily Staff Ionis Pharmaceuticals said it submitted a new drug application to the U.S. Food and Drug Administration for inotersen, an… Continue Reading
FDA Grants Rare Pediatric Disease Designation to ArQule’s Miransertib for Proteus Syndrome
Rare Daily Staff The U.S. Food and Drug Administration granted ArQule Rare Pediatric Disease Designation for its experimental therapy… Continue Reading
FDA 2017 Orphan Drug Approvals
The following is a year-to-date list of U.S. Food and Drug Administration Orphan Drug Approvals Last update November 3, 2017… Continue Reading
U.S. Food and Drug Administration Orphan Drug Designations in October 2017
The following is a list of FDA Orphan Drug designation made in October 2017… Continue Reading
European Commission Says Regs Helped Drive Pediatric Drug Development, But Failed to Drive Rare Disease Drug Development for Kids
Daniel S. Levine Ten years ago, regulators in Europe set out to address a problem. Drug companies often weren’t pursuing pediatric… Continue Reading
Health Canada Kills Planned Orphan Drug Framework for Broader Review
Daniel S. Levine Durhane Wong-Rieger has long been working to get regulators in Canada to create an orphan drug designation similar to what… Continue Reading
FDA Says It Won’t Approve PTC’s DMD Therapy
Rare Daily Staff The U.S. Food and Drug Administration told PTC Therapeutics it would not approve its experimental therapy Translarna for… Continue Reading