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
    • Voices
    • Views
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE

European Agency Recommends Orphan Medicinal Product Designation For Dicerna’s DCR-PHXC

By Dicerna Pharmaceuticals, Inc. | July 12, 2018

Dicerna Pharmaceuticals announced that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has recommended designating DCR-PHXC, the Company’s lead GalXC product candidate, as an orphan medicinal product for the treatment of primary hyperoxaluria (PH) in the European Union (EU). The European Commission (EC) is expected to review the COMP opinion and issue a final ruling within 30 days of receipt.

Separately, Dicerna recently announced that the U.S. FDA granted Orphan Drug Designation to DCR-PHXC for the treatment of PH.

Dicerna is investigating DCR-PHXC for the treatment of all forms of PH, a family of severe, rare, inherited disorders of the liver that often result in kidney failure. The Company initiated the PHYOX Phase 1 clinical trial of DCR-PHXC in normal healthy volunteers in the fourth quarter of 2017 and dosed the first patient with PH in May 2018, with clinical proof-of-concept data anticipated in the second half of 2018.

“We are gratified to see regulators recognize the urgent need for a safe and effective therapy for primary hyperoxaluria, as well as the encouraging preclinical data for DCR-PHXC in various mouse models of PH,” said Ralf Rosskamp, M.D., chief medical officer of Dicerna. “This positive recommendation, as well as the Orphan Drug Designation from the FDA, acknowledges the needs of this underserved patient population.”

The EMA grants orphan medicinal product designation to investigational drugs intended to treat, prevent or diagnose a life-threatening or chronically debilitating disease affecting fewer than five in 10,000 people in the EU, and for which no satisfactory treatment is available. Orphan medicinal product designation provides regulatory and financial incentives for companies to develop and market therapies, including market exclusivity, protocol assistance, fee reductions, and EU-funded research.

In examining the orphan medicinal product application for DCR-PHXC, the COMP concluded that Dicerna established the following:

  • The intention to treat PH with DCR-PHXC is justified based on reduction of urine oxalate concentration and kidney oxalate crystal deposits in a non-clinical model of the condition;
  • The condition is chronically debilitating and life-threatening, in particular because of nephrolithiasis (presence of kidney stones) and nephrocalcinosis (calcification in the renal parenchyma, the functional part of the kidney) leading to renal damage. The majority of patients with PH reach end-stage renal disease (ESRD) during the third to fifth decade of life;
  • The condition was estimated to affect fewer than five per 10,000 persons in the EU at the time the application was submitted.
About DCR-PHXC

DCR-PHXC is an investigational drug in development for the treatment of all forms of primary hyperoxaluria (PH), and the most advanced product candidate utilizing Dicerna’s GalXC technology. GalXC is a proprietary platform invented by Dicerna scientists to discover and develop next-generation RNAi-based therapies designed to silence disease-driving genes in the liver. In animal models of PH, DCR-PHXC selectively silences LDHA in the liver, blocking the excess production of oxalate, a hallmark of the disease. In preclinical studies of DCR-PHXC, the compound was well tolerated with no adverse effects in the liver.

Studies have shown that people who are completely deficient in LDHA show no liver dysfunction and can lead normal lives. LDHA deficiency in the liver might be beneficial for patients with PH, as the LDHA enzyme is implicated in the abnormal production of oxalate in PH, which in turn is responsible for the severe damage to kidneys and other organ systems in patients with PH.

(Source: Dicerna Pharmaceuticals, Inc.)


Filed Under: Drug Discovery

 

Related Articles Read More >

Swissmedic approves first malaria treatment for infants
Korean team reports all-in-one cancer nanomedicine in pre-clinical studies
Nektar’s Phase 2b atopic dermatitis win triggers 1,746% analyst target surge, but legal tussle with ex-partner Lilly could complicate path forward
Dupixent approved to treat bullous pemphigoid
“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
    • Voices
    • Views
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE