US FDA extends orphan drug designation for Race Oncology’s lead asset

US FDA extends orphan drug designation to Race’s re-formulated RC220 bisantrene drug product for treatment of Acute Myeloid Leukemia
Orphan drug designation has a range of commercial advantages including seven years market exclusivity in the US
Designation enables Race to work closely and constructively with the FDA on all its RC220 bisantrene clinical programs

Special Report: The US FDA extends orphan drug designation (ODD) to Race Oncology (ASX:RAC) bisantrene RC220 for AML treatment.

The ODD was originally granted to Update Pharma Inc in 2014 for the RC110 formulation of bisantrene for the treatment of acute myeloid leukemia (AML).

The designation was transferred to RAC in 2017 and has been maintained through annual reporting to the FDA on clinical and non-clinical activities involving bisantrene.

The ODD program was established by the US FDA under The Orphan Drug Act of 1983 to encourage the development of drugs, biologics, devices, or medical foods for rare diseases or conditions affecting fewer than 200,000 people within the US.

This legislation has resulted in the development and approval of 1240 new treatments and drugs.

Being granted ODD offers various benefits to sponsors of new treatments for orphan diseases.

These include seven-year US marketing exclusivity, a 25% federal tax credit for clinical research expenses incurred in the US and waiver of fees under the Prescription Drug User Fee Act (PDUFA).

RAC says the waiver of fees amount to more than US$4m in FY24.

Additionally, an ODD makes recipients eligible to receive research grants from the Office of Orphan Products Development (OOPD) and allows access to further regulatory assistance and guidance from the FDA.

Race looks for European ODD

RAC says the European Medicines Agency (EMA) has a similar ODD scheme to the FDA to help aid the development of new drugs and treatments for rare diseases.

The company says the major difference between the EMA and US FDA ODD programs is that the EMA ODD provides for up to 10-years post-approval marketing exclusivity.

The EMA ODD requires a separate application process, but this is broadly similar to the US FDA ODD application process.

RAC says it has begun the EMA ODD process for bisantrene.

At heart of cancer care

Bisantrene is an anthracene-based chemotherapy, which was initially created by small French pharmaceutical firm Lederle Laboratories during the 1970s and 1980s.

Research confirms bisantrene’s ability to lower the risk of cardiotoxicity and provide cardio-protection when used in conjunction with anthracyclines, a group of drugs used in cancer treatment.

Although effective in patients and approved for treating AML in France, bisantrene’s complex administration prevented commercialisation.

However, RAC has reformulated the drug to enable easy clinical use through standard infusion via arm or leg veins.

RAC is developing bisantrene to meet the significant unmet needs of patients across multiple oncology conditions, initially focusing on metastatic breast cancer and AML.

The biotech is exploring its anticancer and cardio-protective properties alongside known standards of care.

When tested against a panel of 143 cancer cell lines representing more than 20 human tumour types, bisantrene has previously been shown to kill more than 79% of the cells at clinically achievable drug concentrations.

Further, when the cancer cells were treated with mixtures containing bisantrene and the widely used, standard-of-care chemotherapy drug doxorubicin, greater cell-killing was seen in 86% of tumour cells relative to doxorubicin alone.

RAC also recently announced bisantrene and another standard-of-care drug decitabine used together offer significantly improved cancer cell-killing across 143 tumour cell lines than either drug used alone.

The results from recent preclinical work performed under contract at Oncolines B.V. (Netherlands) support use of decitabine in combination with bisantrene as a potential treatment for many cancers, including solid tumours such as lung, prostate, pancreas, breast, head, and neck cancer.

Race welcomes extra FDA guidance

RAC CEO Dr Daniel Tillett says continuing to have ODD for its lead drug asset is important for the company.

He says the additional support and guidance the FDA provides for orphan drugs is particularly valuable as the company advances RC220 bisantrene as a cardioprotective anticancer agent outside of AML.

The FDA ODD is in addition to IP protection for the RC220 bisantrene formulation obtainable via the patent processes which is expected to extend to 2044.

“Race continues to advance bisantrene as a novel treatment for AML, however being able to leverage the additional regulatory and guidance support from the FDA that ODD provides is very welcome,” Tillett says.

RAC chief medical officer Dr Michelle Rashford agrees the close guidance from the FDA will be important for RAC moving forward.

“Orphan Drug Designation is a major asset beyond AML as it enables Race to work closely and constructively with the FDA on all of our RC220 bisantrene clinical programs as we progress towards opening an FDA IND in 2025,” she says.

This article was developed in collaboration with Race Oncology, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.