RARE Daily

FDA Grants Rare Pediatric Disease Designation to Oncoheroes’ Dovitinib for Osteosarcoma

September 23, 2022

The United States Food and Drug Administration has granted Rare Pediatric Disease to Oncoheroes Biosciences’ dovitinib, an investigational treatment for osteosarcoma.

Photo: Ricardo Garcia, Oncoheroes’ co-founder, and CEO

Osteosarcoma (OS) is a rare disease and the most common cause of bone cancer in children and adolescents, diagnosed in approximately 750 to 1,000 new individuals each year in the United States. About 450 are children or adolescents under the age of 20. The demonstration of the utility of chemotherapy for all patients in the early 1980s led to the current survival rate of greater than 65 percent, but this has not changed over the past three decades. The 5-year overall survival rates are approximately 20 percent for patients who develop metastatic disease. Additionally, of patients who experience disease progression or recurrence, less than 30 percent survive.

“This is an acknowledgment of the urgent need for better treatments for children and adolescents with osteosarcoma,” said Ricardo Garcia, Oncoheroes’ co-founder, and CEO.

Dovitinib is a pan-tyrosine kinase inhibitor targeting fibroblast growth factor receptor, vascular endothelial growth factor receptor, and other receptor tyrosine kinases (RTKs). In January 2022, Oncoheroes signed an exclusive pediatric licensing agreement for dovitinib with Allarity Therapeutics, Inc. to continue the clinical development of this drug candidate for the benefit of younger cancer patients. Dovitinib will be developed together with Allarity’s DRP companion diagnostics.

Several RTKs inhibited by dovitinib are involved in the growth, differentiation, and survival of different types of tumor cells as well as in tumor angiogenesis and development and maintenance of the tumor stroma microenvironment that is essential for proliferation of tumor cells and angiogenesis. Many RTKs must be inhibited simultaneously to generate a clinical response. Generally, the efficacy of tyrosine kinase inhibitors (TKIs) in the clinic depends on achieving a wide therapeutic index (vs off-tumor/on-target toxicity). Resistance can quickly occur to single TKI through mutations and therefore resistance is less likely to quickly develop with TKIs as these hit multiple targets simultaneously. Oncoheroes believes dovitinib’s utility in pediatric osteosarcoma is promising. given its multi-target mechanism of action.

The FDA actively supports companies that develop drugs for rare diseases, defined as diseases affecting less than 200,000 Americans. One of the relevant programs created by the agency is for rare diseases primarily affecting individuals younger than 18 years old, called the Rare Pediatric Disease designation (RPDD) which comes with the related priority review voucher opportunity.

Rare Pediatric Disease Designation qualifies Oncoheroes to receive fast track review, and a priority review voucher at the time of marketing approval of dovitinib. The priority review voucher is fully transferable and grants accelerated FDA review of a drug candidate for any indication, reducing the review period to 6 months and potentially gaining early market access. To date, 18 out of 34 vouchers received for pediatric indications have been sold for a cumulative sale price of $2.3 billion. Most recently, Marinus Pharmaceuticals sold its voucher to Novo Nordisk for $110 million.

Author: Rare Daily Staff

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