Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50

FDA Grants Second Orphan Designation For VAL-083

By Drug Discovery Trends Editor | March 16, 2016

DelMar Pharmaceuticals, Inc. announced today that the FDA Office of Orphan Products Development (OOPD) has granted orphan drug designation for its lead product candidate, VAL-083, in the treatment of medulloblastoma. The investigational drug candidate previously received an orphan designation for glioblastoma in the United States and in Europe.
 
VAL-083 is a “first-in-class,” small-molecule chemotherapeutic. In more than 40 Phase I and II clinical studies sponsored by the U.S. National Cancer Institute, VAL-083 demonstrated clinical activity against a range of cancers including lung, brain, cervical, ovarian tumors and leukemia both as a single-agent and in combination with other treatments.
 
“We are pleased to achieve this important regulatory milestone and to continue a collaborative relationship with the FDA and the OOPD as we continue to expand the development of VAL-083,” commented Jeffrey Bacha, chairman and CEO of DelMar.  “Orphan designation is a major step toward expediting this promising therapy to an additional patient population with few treatment options.”
 
DelMar has been conducting clinical trials with VAL-083 as a potential new treatment for glioblastoma multiforme (GBM), the most common and aggressive form of brain cancer.  In September 2015, DelMar announced completion of enrollment in a Phase II clinical trial in refractory GBM.  The Company anticipates top-line overall survival data from this trial in the first half of 2016.   
 
Through its research, DelMar has also been exploring the unique cytotoxic mechanism of VAL-083 in order to identify additional indications where VAL-083 may address modern unmet medical needs in the treatment of cancer.  In November 2015, DelMar presented new pre-clinical data in a poster entitled, “Dianhydrogalactitol (VAL-083) Offers Potential Therapeutic Alternatives in the Treatment of Pediatric Brain Tumors,” at the American Association for Cancer Research (AACR) Advances in Pediatric Research: From Mechanisms and Models to Treatment and Survivorship Conference.
 
Medulloblastoma is the most common malignant pediatric brain tumor, accounting for 15-30% of all childhood intracranial neoplasms. Although multidisciplinary treatment has improved the 5-year survival rates in children significantly, the prognosis for certain subtypes of medulloblastoma and for recurrent disease remains poor with a median overall survival of less than one (1) year. 
 
In historical NCI-sponsored clinical studies, VAL-083 demonstrated clinical activity against medulloblastoma. In these studies VAL-083 was investigated both as a stand-alone therapy and in combination with other chemotherapeutic regimens.  DelMar’s recent pre-clinical research demonstrates that VAL-083 is active against medulloblastoma cells with difficult to treat sonic hedgehog (SHH) characteristics and p53 mutations; and VAL-083 in combination with temozolomide completely inhibits self-renewal of pediatric brain cancer stem cells (CSCs).
 
“Taken together, we believe these data will serve as a basis for our clinical development strategy with VAL-083 in pediatric brain tumors,” continued Mr. Bacha. “We plan to continue our discussions with leading clinical investigators in order to undertake the necessary steps to advance VAL-083 into clinical studies as a potential treatment for children suffering from recurrent and difficult-to-treat medulloblastoma subtypes.”
 
Source: DelMar Pharmaceuticals, Inc. 

Filed Under: Drug Discovery

 

Related Articles Read More >

Roche in Drug Discovery & Development Pharma 50
Roche’s bispecific antibody Lunsumio wins priority review from FDA for non-Hodgkin lymphoma
Tiffany Hurd
CB Therapeutics biz dev lead sees upside for psychedelics in mental health and beyond
Pharmacovigilance
A new era of pharmacovigilance: Worldwide master key for drug safety monitoring
Takeda Pharmaceutical in the Drug Discovery & Development Pharma 50
Takeda’s Takhzyro fares well in pediatric hereditary angioedema study

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.

Need Drug Discovery news in a minute?

We Deliver!
Drug Discovery & Development Enewsletters get you caught up on all the mission critical news you need. Sign up today.
Enews Signup
Drug Discovery and Development
  • MASSDEVICE
  • DEVICETALKS
  • Medical Design & Outsourcing
  • MEDICAL TUBING + EXTRUSION
  • MEDTECH 100
  • Medical Design Sourcing
  • Subscribe to our Free E-Newsletter
  • Contact Us
  • About Us
  • Advertise With Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2022 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
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50