FDA 2017 Orphan Drug Approvals

November 3, 2017

The following is a year-to-date list of U.S. Food and Drug Administration Orphan Drug Approvals by the FDA’s Center for Drug Evaluation and Research.
Last update December 30, 2017.

Regulatory

Senate Pressure Grows on Novartis over Gene Therapy Data Manipulation

Rare Daily Staff Senate Finance Committee Chairman Chuck Grassley (R-Iowa) is demanding records and answers from Novartis […]

Regulatory

Sarepta Says DMD Gene Therapy Adverse Event Report Submitted Erroneously

Rare Daily Staff Sarepta Therapeutics said that it was informed that an adverse event report regarding its […]

Regulatory

FDA Updates Guidance on Rare Pediatric Disease Priority Review Vouchers

Rare Daily Staff The U.S. Food and Drug Administration issued updated draft guidance on Rare Pediatric Disease […]

Regulatory

FDA Orphan Drug Approvals First Half 2019

Regulatory

FDA Orphan Drug Designations in Q2 2019

Regulatory

FDA Tells Acer It Won’t Approve vEDS Therapy without Additional Trial

The U.S. Food and Drug Administration rejected rare disease drug developer Acer Therapeutics’ application for marketing approval […]

Regulatory

Catalyst Sues FDA Over Approval of Competing LEMS Drug

Catalyst Pharmaceuticals has filed a lawsuit against the U.S. Food and Drug Administration demanding that it vacate […]

Regulatory

Getting Real

In the world of drug development, when things are working as they should, data drives decisions. […]

Regulatory

FDA Places Clinical Hold on Translate Bio’s Application to Begin Trials for mRNA Therapy

Rare Daily Staff The U.S. Food and Drug Administration has placed a clinical hold on Translate Bio’s […]

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FDA 2017 Orphan Drug Approvals

Related

Senate Pressure Grows on Novartis over Gene Therapy Data Manipulation

Sarepta Says DMD Gene Therapy Adverse Event Report Submitted Erroneously

FDA Updates Guidance on Rare Pediatric Disease Priority Review Vouchers

FDA Orphan Drug Approvals First Half 2019

FDA Orphan Drug Designations in Q2 2019

FDA Tells Acer It Won’t Approve vEDS Therapy without Additional Trial

Catalyst Sues FDA Over Approval of Competing LEMS Drug

Getting Real

FDA Places Clinical Hold on Translate Bio’s Application to Begin Trials for mRNA Therapy

Stay Connected