Regen BioPharma Prepares Response For FDA Regarding Its Orphan Drug Application for HemaXellerate
Rhea-AI Summary
Regen BioPharma (OTC:RGBP) is preparing its response to FDA comments regarding their Orphan Drug Application for HemaXellerate, a novel cell-based therapy targeting aplastic anemia. The treatment, utilizing autologous mesenchymal stem cells, aims to restore hematopoietic stem cell function in patients with this rare bone marrow disorder affecting fewer than 200,000 Americans.
If granted, the orphan drug designation would provide significant benefits including seven years of market exclusivity upon approval, tax credits for clinical testing, and FDA application fee exemptions. The company plans to initiate Phase I clinical trials soon and will discuss the FDA submission during their presentation at the Emerging Growth Conference on September 24, 2025.
Positive
- Potential to receive orphan drug designation benefits including 7 years market exclusivity
- Advancing to Phase I clinical trials for HemaXellerate
- Addressing an unmet medical need in rare disease with limited treatment options
Negative
- FDA has raised comments requiring response before potential approval
- Early-stage development with no clinical trial data yet
- Faces regulatory uncertainty and clinical development risks
SAN DIEGO, Sept. 16, 2025 (GLOBE NEWSWIRE) -- Regen BioPharma, Inc. (OTC PINK: RGBP) and (OTC PINK: RGBPP) today announced the Company is in the process of addressing comments received by the U.S. Food and Drug Administration (FDA) in connection with the Company’s recent submission of an Orphan Drug Application (ODA) to the FDA for HemaXellerate, its lead therapeutic candidate for the treatment of aplastic anemia. Aplastic anemia is a rare and potentially life-threatening bone marrow disorder. The Company is confident that it will be able to satisfactorily address any comments received.
HemaXellerate is a novel, cell-based therapeutic designed to stimulate bone marrow activity and restore hematopoietic function. The submission of the ODA marks a key milestone in Regen BioPharma’s mission to develop innovative therapies for patients with limited treatment options.
“This application represents a major step forward in our commitment to advancing regenerative therapies for rare diseases,” said Dr. David Koos, Chairman and CEO of Regen BioPharma. “Receiving orphan drug designation would provide important regulatory and commercial advantages as we continue clinical development of HemaXellerate.”
Orphan drug designation is granted by the FDA to drugs and biologics intended to treat rare diseases affecting fewer than 200,000 people in the United States. If granted, the designation provides benefits such as seven years of market exclusivity upon approval, tax credits for clinical testing, and exemption from certain FDA application fees.
Aplastic anemia is characterized by the failure of the bone marrow to produce sufficient blood cells, leading to fatigue, infections, and uncontrolled bleeding. Current treatment options are limited, and many patients do not respond adequately to standard therapies.
HemaXellerate, an autologous mesenchymal stem cell product, is expected to restore hematopoietic stem cell function and reverse the effects of aplastic anemia-induced bone marrow suppression. Regen BioPharma plans to initiate Phase I clinical trials shortly.
“We believe HemaXellerate has the potential to significantly improve the lives of patients suffering from this debilitating condition,” added Dr. Koos. “We are committed to working closely with the FDA to bring this therapy to patients as quickly and safely as possible.”
We will be covering the submission and FDA’s response during our presentation at the Emerging Growth Conference 4:35 Eastern Time on September 24, 2025 https://goto.webcasts.com/starthere.jsp?ei=1717091&tp_key=c78a55764a&sti=rgbp.
About Regen BioPharma, Inc.
Regen BioPharma, Inc. is a publicly traded biotechnology company (PINK: RGBP) and (PINK: RGBPP). The Company is focused on the immunology and immunotherapy space. The Company is focused on rapidly advancing novel technologies through pre-clinical and Phase I/ II clinical trials. Currently, the Company is focused on mRNA and small molecule therapies for treating cancer and autoimmune disorders. Additional information on Regen BioPharma is available at http://www.regenbiopharmainc.com.
Disclaimer: This news announcement may contain forward-looking statements. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Future events and actual results could differ materially from those set forth in, contemplated by, or underlying the forward-looking statements. The risks and uncertainties to which forward looking statements are subject include, but are not limited to, the effect of government regulation, competition and other material risks.
CONTACT INFORMATION:
Regen BioPharma Inc.
David R. Koos, Ph.D.
Chairman & Chief Executive Officer
+1-619-722-5505 Phone
+1-619-330-2328 Fax
Email: david.koos@regenbiopharmainc.com or
david.koos@regenbiopharmainc.com
X (formerly twitter): https://x.com/TheRegenBio
FAQ
What is Regen BioPharma's HemaXellerate treatment for aplastic anemia?
What benefits would RGBP receive with FDA orphan drug designation?
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