Unmet Needs

In which orphan spaces or unmet medical needs do you think we will see the next treatment innovations?

Pharma's Almanac

Parenterals

Trends Shaping the Dynamic Market for Parenterals

Marga Viñes

Grifols

Commercial Manufacturing

Advancing Commercial Manufacturing from Orphan Drugs to Blockbusters

Tyler Ewald

UPM Pharmaceuticals

Ophthalmology

Innovative Therapies to Treat Rare and Underserved Eye Diseases

Shankar Musunuri, Ph.D.

Ocugen

Orphan Drugs

Customer Engagement and Information Sharing are Essential for Success in Orphan Drug Outsourcing

Marga Viñes

Grifols

Parenteral Manufacturing

Catering to a Growing Demand for Small-Volume Parenteral Manufacturing

Oriol Prat

Grifols

Nice Insight Overview

Where Do 
Orphan Drugs 
Go From Here?

Nigel Walker

Nice Insight

Orphan Drug Development

An Expanding Orphan Drug Market

Cynthia A. Challener, Ph.D.; Emilie Branch; David Alvaro, Ph.D.

Pharma's Almanac

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

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

Clinical Trial Logistics

Facilitating Complex Clinical Trials For Rare Diseases

Ariette van Strien

Marken

Orphan Drug

Cholangiocarcinoma Drug Receives Orphan Drug Designation from FDA

Cynthia A. Challener, Ph.D.

Pharma's Almanac

FDA

BioInvent Receives FDA Orphan Designation for BI-1206 for Mantle Cell Lymphoma

BioInvent

Orphan Drug

Arrowhead Receives Positive EMA Opinion on Orphan Designation for ARO-AAT for Treatment of Congenital Alpha-1 Antitrypsin Deficiency

Arrowhead Pharmaceuticals

Orphan Drug

Samus Therapeutics Announces PU-H71 Granted Orphan Drug Designation and First Patient Dosed in Phase 1b Study in Myelofibrosis

Samus Therapeutics

FDA

Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy

Scholar Rock

Orphan Drug

Kazia Receives FDA Orphan Designation for GDC-0084

Kazia Therapeutics Limited

Orphan Drug

Selumetinib Granted Orphan Drug Designation by the U.S. FDA for Neurofibromatosis Type 1

Merck

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.

FDA

Unjamming the Orphan Drug Backlog

Nigel Walker

Nice Insight