Oncoheroes Biosciences, a biotech focused on advancing new therapies for childhood cancer, recently announced that the Unites States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to volasertib, one of its drugs.
Volasertib is a small molecular inhibitor that has been identified as an anti-cancer agent. Receiving ODD and RPDD qualifies Oncoheroes to receive fast-track review, partial tax credits for clinical trial expenditures, and a priority review voucher at the time of marketing approval of volasertib.
This is fantastic news in the realm of childhood cancer; however, many individuals are a bit uncertain about what this actually means. To keep the community informed, National Foundation for Cancer Research provides the following simple breakdown.
How common is childhood cancer?
There are dozens of cancers and numerous subtypes that occur in children aged 0-14 years. Childhood cancers are often different than adult cancers as pediatric cancers are not strongly linked to risk factors related to lifestyle or the environment. In the US, cancer is the leading cause of death by disease in children – taking the life of nearly 1,200 children each year. It is estimated that one in 389 children will be diagnosed with cancer by the age of 15.
What is RPDD?
RPDD is a voucher system designed to support teams that develop products (such as volasertib) that treat or prevent rare pediatric diseases, including cancer. The voucher system is a type of incentive to encourage the development of these products. Developing a new product is costly and timely – costing millions to billions of dollars and many years to complete. After the lengthy development process, the products then go to the FDA for review. The FDA has 10 months to review each product, further extending the time it takes to move the product to market. The priority review voucher that is awarded with RPDD allows an expedited six-month review instead of the 10-month review. These vouchers can only be awarded by the FDA, demonstrating that the significant potential of volasertib is recognized by government officials.
What is ODD?
First and foremost, an orphan drug is defined as a drug intended for the treatment, prevention, or diagnosis of a rare disease or condition that affects less than 200,000 people in the United States. The FDA awards Orphan Drug Designation to potential therapies addressing the unmet needs of underserved patients with a rare disease. ODD encourages organizations to invest the time and money into a product that will be used by a comparatively small population. The incentives that accompany this status include a tax credit on expenditures incurred in clinical studies, exemption from filing fees such as the user fee, eligibility for a research grant awarded by the FDA, and seven years of US market exclusivity upon approval. Being granted orphan designation does not modify the standard regulatory requirements to obtain marketing approval.
What is volasertib?
Volasertib is an enzyme inhibitor, effecting the enzyme known to be involved in disease progression in a number of cancers. This product was originally discovered and developed by Boehringer Ingelheim for the treatment of Acute Myeloid Leukemia but was discontinued for strategic reasons. Other researchers called for further development of volasertib for rhabdomyosarcoma, a rare type of childhood cancer that forms in the soft tissue. In 2019, Oncoheroes in-licensed volasertib from Boehringer Ingelheim to continue the clinical development of this drug candidate for the benefit of younger cancer patients.
Who is Oncoheroes Biosciences Inc.?
Oncoheroes Biosciences Inc. is a life science company focused on advancing new therapies for childhood cancer. National Foundation for Cancer Research has been supporting Oncoheroes through the AIM-HI Accelerator Fund for over three years in their noble mission of developing new and innovative treatments for childhood cancers. This support has allowed Oncoheroes to set up their discovery lab, launch their first project for the development of a new therapy for medulloblastoma patients, gain support from like-minded organizations, and raise funds to support their research.
Learn more about Oncoheroes and the amazing work supported by NFCR and the AIM-HI Accelerator Fund here.
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