Neurodegenerative
Leriglitazone Marketing Authorization Application Submitted For Treatment of Cerebral Adrenoleukodystrophy Has Been Validated
Minoryx
PR-07-25-NI-2Jul 23, 2025
Orphan Drugs
From Market Failures to Lifesaving Innovations: The Evolution of Orphan Drugs
Cynthia A. Challener, Ph.D.
Pharma's Almanac
PAO-25-30-06Mar 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-01Jun 24, 2022
Parenterals
Trends Shaping the Dynamic Market for Parenterals
Marga Viñes
Grifols
PAP-Q3-20-CL-018Sep 29, 2020
Commercial Manufacturing
Advancing Commercial Manufacturing from Orphan Drugs to Blockbusters
Tyler Ewald
UPM Pharmaceuticals
PAP-Q4-19-CL-032Dec 06, 2019
Ophthalmology
Innovative Therapies to Treat Rare and Underserved Eye Diseases
Shankar Musunuri, Ph.D.
Ocugen
PAP-Q4-19-CL-025Dec 06, 2019
Orphan Drugs
Customer Engagement and Information Sharing are Essential for Success in Orphan Drug Outsourcing
Marga Viñes
Grifols
PAP-Q3-19-CL-013Oct 29, 2019
Parenteral Manufacturing
Catering to a Growing Demand for Small-Volume Parenteral Manufacturing
Oriol Prat
Grifols
PAO-M04-19-CL-001May 28, 2019
Nice Insight Overview
Where Do Orphan Drugs Go From Here?
Nigel Walker
Nice Insight
PAP-Q2-2019-NI-004May 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-001May 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-002May 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
Clinical Trial Logistics
Facilitating Complex Clinical Trials For Rare Diseases
Ariette van Strien
Marken
PAP-Q1-2019-CL-003Mar 12, 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
FDA
BioInvent Receives FDA Orphan Designation for BI-1206 for Mantle Cell Lymphoma
BioInvent
PR-M01-19-NI-088Jan 30, 2019
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-097Jun 29, 2018
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-050Jun 14, 2018
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-003-4472Apr 02, 2018
Orphan Drug
Kazia Receives FDA Orphan Designation for GDC-0084
Kazia Therapeutics Limited
PR-M02-18-NI-107Feb 28, 2018
Orphan Drug
Selumetinib Granted Orphan Drug Designation by the U.S. FDA for Neurofibromatosis Type 1
Merck & Co.
PR-M02-18-NI-76Feb 21, 2018