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Orphan Drugs
From Market Failures to Lifesaving Innovations: The Evolution of Orphan Drugs
Cynthia A. Challener, Ph.D.
Pharma's Almanac
PAO-25-30-06
Mar 02, 2025
Unmet Needs
In which orphan spaces or unmet medical needs do you think we will see the next treatment innovations?
Pharma's Almanac
PAO-06-022--RT-01
Jun 24, 2022
Parenterals
Trends Shaping the Dynamic Market for Parenterals
Marga Viñes
Grifols
PAP-Q3-20-CL-018
Sep 29, 2020
Commercial Manufacturing
Advancing Commercial Manufacturing from Orphan Drugs to Blockbusters
Tyler Ewald
UPM Pharmaceuticals
PAP-Q4-19-CL-032
Dec 06, 2019
Ophthalmology
Innovative Therapies to Treat Rare and Underserved Eye Diseases
Shankar Musunuri, Ph.D.
Ocugen
PAP-Q4-19-CL-025
Dec 06, 2019
Orphan Drugs
Customer Engagement and Information Sharing are Essential for Success in Orphan Drug Outsourcing
Marga Viñes
Grifols
PAP-Q3-19-CL-013
Oct 29, 2019
Parenteral Manufacturing
Catering to a Growing Demand for Small-Volume Parenteral Manufacturing
Oriol Prat
Grifols
PAO-M04-19-CL-001
May 28, 2019
Nice Insight Overview
Where Do Orphan Drugs Go From Here?
Nigel Walker
Nice Insight
PAP-Q2-2019-NI-004
May 24, 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-001
May 24, 2019
Orphan Drug Development
Managing the Risks Associated with Orphan Drug Development and Manufacturing
Cynthia A. Challener, Ph.D.; Emilie Branch; David Alvaro, Ph.D.
Pharma's Almanac
PAP-Q2-2019-NI-002
May 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-003
May 24, 2019
Clinical Trial Logistics
Facilitating Complex Clinical Trials For Rare Diseases
Ariette van Strien
Marken
PAP-Q1-2019-CL-003
Mar 12, 2019
Orphan Drug
Cholangiocarcinoma Drug Receives Orphan Drug Designation from FDA
Cynthia A. Challener, Ph.D.
Pharma's Almanac
PAO-M03-19-NI-003
Mar 05, 2019
BioInvent
FDA
BioInvent Receives FDA Orphan Designation for BI-1206 for Mantle Cell Lymphoma
BioInvent
PR-M01-19-NI-088
Jan 30, 2019
Arrowhead Pharmaceuticals
Orphan Drug
Arrowhead Receives Positive EMA Opinion on Orphan Designation for ARO-AAT for Treatment of Congenital Alpha-1 Antitrypsin Deficiency
Arrowhead Pharmaceuticals
PR-M06-18-NI-097
Jun 29, 2018
Samus Therapeutics
Orphan Drug
Samus Therapeutics Announces PU-H71 Granted Orphan Drug Designation and First Patient Dosed in Phase 1b Study in Myelofibrosis
Samus Therapeutics
PR-M06-18-NI-050
Jun 14, 2018
Scholar Rock
FDA
Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy
Scholar Rock
PR-M04-18-NI-003-4472
Apr 02, 2018
Kazia Therapeutics Limited
Orphan Drug
Kazia Receives FDA Orphan Designation for GDC-0084
Kazia Therapeutics Limited
PR-M02-18-NI-107
Feb 28, 2018
Merck & Co.
Orphan Drug
Selumetinib Granted Orphan Drug Designation by the U.S. FDA for Neurofibromatosis Type 1
Merck & Co.
PR-M02-18-NI-76
Feb 21, 2018
MEI Pharma, Inc.
Orphan Drug Designation
Helsinn Group and MEI Pharma Announce that Pracinostat has Received Orphan Drug Designation from the European Medicines Agency for the Treatment of Acute Myeloid Leukemia (AML)
MEI Pharma, Inc.
PR-M01-18-NI-045
Jan 16, 2018
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