Regen BioPharma Prepares Response For FDA Regarding Its Orphan Drug Application for HemaXellerate
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.
Regen BioPharma (OTC:RGBP) sta preparando la risposta ai commenti FDA riguardo l'Orphan Drug Application per HemaXellerate, una terapia cellulare innovativa mirata all'anemia aplastica. Il trattamento, che utilizza cellule staminali mesenchimali autologhe, mira a ripristinare la funzione delle cellule staminali hematopoietiche nei pazienti con questo raro disturbo del midollo osseo che colpisce meno di 200.000 americani.
Se concesso, la designazione di farmaco orfano fornirebbe benefici significativi tra cui sette anni di esclusività di mercato dopo l'approvazione, crediti d'imposta per i test clinici e esenzioni dalle tariffe di presentazione FDA. L'azienda intende avviare presto studi clinici di Fase I e discuterà la sottomissione FDA durante la presentazione alla Emerging Growth Conference il 24 settembre 2025.
Regen BioPharma (OTC:RGBP) está preparando su respuesta a los comentarios de la FDA sobre su Solicitud de Fármaco Huérfano para HemaXellerate, una novedosa terapia basada en células dirigida a la anemia aplásica. El tratamiento, que utiliza células madre mesenquimales autólogas, tiene como objetivo restablecer la función de las células madre hematopoyéticas en pacientes con este raro trastorno de la médula ósea que afecta a menos de 200,000 estadounidenses.
De ser concedida, la designación de fármaco huérfano proporcionaría beneficios significativos, incluyendo siete años de exclusividad en el mercado tras la aprobación, créditos fiscales para pruebas clínicas y exenciones de tarifas de presentación de la FDA. La empresa planea iniciar ensayos clínicos de Fase I pronto y discutirá la presentación ante la FDA durante su ponencia en la Emerging Growth Conference el 24 de septiembre de 2025.
Regen BioPharma(OTC:RGBP)은 HemaXellerate에 대한 FDA의 고아 의약품 신청에 관한 코멘트에 대한 답변을 준비 중입니다. 자가 생성된 mesenchymal stem cells를 이용한 새로운 세포 기반 치료제로, 희귀 골수 질환인 재생불량성 빈혈 환자에서 조혈모세포 기능 회복을 목표로 합니다. 이 질환은 미국인 중 20만 명 미만에게 영향을 미칩니다.
허가가 내려지면 고아 의약품 지정을 통해 허가 후 7년의 시장 독점, 임상 시험에 대한 세액 공제, FDA 신청 수수료 면제 등의 중요한 혜택이 제공됩니다. 회사는 조만간 1상 임상시험을 시작할 계획이며 2025년 9월 24일 Emerging Growth Conference에서 FDA 제출에 대해 발표 중 논의할 예정입니다.
Regen BioPharma (OTC:RGBP) prépare sa réponse aux commentaires de la FDA concernant sa demande de médicament orphelin pour HemaXellerate, une thérapie cellulaire innovante ciblant l'anémie aplasique. Le traitement, utilisant des cellules souches mésenchymales autologues, vise à restaurer la fonction des cellules souches hématopoïétiques chez les patients atteints de ce trouble rare de la moelle osseuse qui touche moins de 200 000 Américains.
Si l'octroi est accordé, la désignation de médicament orphelin offrirait des avantages significatifs, notamment sept ans d'exclusivité sur le marché après l'agrément, des crédits d'impôt pour les essais cliniques et des exemptions des frais de soumission à la FDA. L'entreprise prévoit de lancer prochainement des essais cliniques de Phase I, et discutera de la soumission à la FDA lors de sa présentation à la Emerging Growth Conference le 24 septembre 2025.
Regen BioPharma (OTC:RGBP) bereitet seine Antwort auf FDA-Kommentare zu ihrem Orphan-Drug-Antrag für HemaXellerate vor, eine neuartige zellbasierte Therapie, die auf aplastische Anämie abzielt. Die Behandlung nutzt autologe mesenchymale Stammzellen und zielt darauf ab, die Funktion der Blutstammzellen bei Patienten mit dieser seltenen Knochenmarkserkrankung wiederherzustellen, die weniger als 200.000 Amerikaner betrifft.
Wird sie genehmigt, würde die Orphan-Drug-Zuordnung erhebliche Vorteile bieten, darunter sieben Jahre Marktexklusivität nach der Zulassung, Steueranreize für klinische Tests und Ausnahmen von FDA-Antragsgebühren. Das Unternehmen plant, bald Phase-I-Studien zu beginnen, und wird die FDA-Einreichung während ihrer Präsentation bei der Emerging Growth Conference am 24. September 2025 besprechen.
Regen BioPharma (OTC:RGBP) تستعد للرد على تعليقات هيئة الغذاء والدواء الأمريكية (FDA) بخصوص طلب دواء ودّي جاهز للأدوية اليتيمة لـ HemaXellerate، وهو علاج خلايا جديد يستهدف فقر الدم اللاتيغي. العلاج، الذي uses الخلايا الجذعية الوسيطة ذاتياً، يهدف إلى استعادة وظيفة الخلايا الجذعية المولِّدة للدم لدى المرضى المصابين بهذا الاضطراب النادر في نخاع العظم الذي يؤثر على أقل من 200,000 أمريكي.
إذا مُنِح، ستوفر تسمية الدواء اليتيم مزايا كبيرة تشمل سبع سنوات من الحصرية السوقية بعد الموافقة وامتيازات ضريبية للاختبارات السريرية وإعفاءات من رسوم تقديم FDA. تخطط الشركة لبدء تجارب المرحلة I قريباً وستناقش تقديم FDA أثناء عرضها في مؤتمر Emerging Growth بتاريخ 24 سبتمبر 2025.
Regen BioPharma (OTC:RGBP) 正在为其 HemaXellerate 的孤儿药申请准备回应 FDA 的意见，这是一种针对再生性贫血的新型细胞治疗。该治疗利用自体间充质干细胞，旨在在这个罕见的骨髓疾病患者中恢复造血干细胞功能，该疾病影响的美国人少于 20 万人。
如果获批，孤儿药资格将带来显著利益，包括获批后七年的市场排他性、临床试验税收抵免以及 FDA 申请费豁免。公司计划很快启动 I 期临床试验，并将在 2025 年 9 月 24 日的 Emerging Growth Conference 上讨论 FDA 的提交情况。
- 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
- 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?
When will Regen BioPharma begin clinical trials for HemaXellerate?
How many people are affected by aplastic anemia in the US?
When will RGBP present updates on the FDA submission?
