PR-M12-19-NI-018
FDA Grants Orphan Drug Designation to Epidermolysis Bullosa Treatment
APR Applied Pharma Research s.a.
PA1853Dec 12, 2019
Orphan Drug Designation
Dynacure Receives Orphan Drug Designation from the US FDA for DYN101, an Antisense Medicine to Treat Rare Disease 'Centronuclear Myopathies'
Dynacure
PR-M08-19-NI-039Aug 14, 2019
Orphan Drug Development
An Expanding Orphan Drug Market
Cynthia A. Challener, Ph.D.; Emilie Branch; David Alvaro, Ph.D.
Pharma's Almanac
PAP-Q2-2019-NI-001May 24, 2019
Orphan Drug Development
Navigating the Road to Successful Orphan Drug Commercialization and Launch
Cynthia A. Challener, Ph.D.; Emilie Branch; David Alvaro, Ph.D.
Pharma's Almanac
PAP-Q2-2019-NI-003May 24, 2019
Orphan Drug
Cholangiocarcinoma Drug Receives Orphan Drug Designation from FDA
Cynthia A. Challener, Ph.D.
Pharma's Almanac
PAO-M03-19-NI-003Mar 05, 2019
Multiple Myeloma
Sutro's STRO-001 Receives U.S. FDA Orphan Drug Designation for Treatment of Multiple Myeloma
Sutro Biopharma, Inc.
PR-M10-18-NI-049Oct 15, 2018
Orphan Drug Designation
OBI Pharma Granted FDA Orphan Drug Designation for OBI-3424 for the Treatment of Acute Lymphoblastic Leukemia (ALL)
OBI Pharma
PR-M09-18-NI-082Sep 26, 2018
FDA Approval
Sirnaomics Oncology IND Receives Green Light from US FDA to Initiate Study for Treatment of Cholangiocarcinoma
Sirnaomics, Inc.
PR-M09-18-NI-062Sep 20, 2018
Drug Designation
Martin Pharmaceuticals’ LIVANTRA™ Receives FDA Orphan Drug Designation in Second Indication
Martin Pharmaceuticals
PR-M09-18-NI-033Sep 13, 2018
Drug Designation
Alector Receives Orphan Drug Designation from the U.S. FDA for AL001 for the Treatment of Frontotemporal Dementia
Alector
PR-M08-18-NI-080Aug 21, 2018
FDA Approval
Alteogen Gets FDA Approval for Orphan Drug Designation with an Antibody-Drug Conjugate for Gastric Cancer
Alteogen
PR-M08-18-NI-052Aug 14, 2018