Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE

Capsules To Fight Rare Metabolic Disorder Approved For Children One-Year-Old And Up

By Horizon Pharma plc | December 28, 2017

Horizon Pharma plc announces FDA approval to expand the indication for Procysbi (cysteamine bitartrate) delayed-release capsules to include children one year of age and older living with nephropathic cystinosis.

The U.S. Food and Drug Administration (FDA) has approved an expansion to the indication for Horizon Pharma plc’s Procysbi (cysteamine bitartrate) delayed-release capsules to include children one year of age and older living with nephropathic cystinosis.

With the update to the indication, the Procysbi prescribing information now includes revised guidance for physicians administering the medicine to pediatric patients, including new clinical evidence and dosing information for very young children. It was previously FDA-approved for adults and children as young as two years of age.

“Data included in the updated label provide further evidence around the unique role of Procysbi in helping physicians manage young children during one of the most crucial periods for growth,” said Craig B. Langman, M.D., lead investigator for the study that led to the label update.

The Procysbi labeling was updated based on a long-term, prospective, open-label study that enrolled 17 people living with nephropathic cystinosis, including 15 children between the ages of 1 and 5 years old, who had not previously been treated with cysteamine therapy.

Children enrolled in the study experienced lowering of white blood cell cystine levels from poor controlled to well controlled at 12 and 18 month measurements. Additionally, they experienced measured improvements in growth milestones including weight and height. 

The most common adverse reactions (>10 percent) in patients treated in clinical trials reflected in the FDA approved product labeling were vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath odor, nausea, electrolyte imbalance, and headache. The study was required by the FDA as a post-marketing commitment after Procysbi was approved. 

(Source: Horizon Pharma plc)


Filed Under: Drug Discovery

 

Related Articles Read More >

Lokavant’s Spectrum v15 uses AI to cut trial-feasibility modeling from weeks to minutes
Prime time for peptide-based drug discovery 
Why smaller, simpler molecular glues are gaining attention in drug discovery
Glass vial, pipette and woman scientist in laboratory for medical study, research or experiment. Test tube, dropper and professional female person with chemical liquid for pharmaceutical innovation
Unlocking ‘bench-to-bedside’ discoveries requires better data sharing and collaboration
“ddd
EXPAND YOUR KNOWLEDGE AND STAY CONNECTED
Get the latest news and trends happening now in the drug discovery and development industry.

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.
Drug Discovery and Development
  • MassDevice
  • DeviceTalks
  • Medtech100 Index
  • Medical Design Sourcing
  • Medical Design & Outsourcing
  • Medical Tubing + Extrusion
  • Subscribe to our E-Newsletter
  • Contact Us
  • About Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2025 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE