FDA Grants Orphan Drug Designation to Rezolute’s Ersodetug (RZ358) for the Treatment of Hypoglycemia Due to Tumor Hyperinsulinism
Rezolute (Nasdaq: RZLT) announced that the FDA has granted Orphan Drug Designation to ersodetug for treating hypoglycemia due to tumor hyperinsulinism (HI). The company plans to initiate a Phase 3 registrational trial for ersodetug in tumor HI patients in 2025. The FDA's designation offers seven years of market exclusivity and potential expedited approval pathways. Ersodetug aims to provide a universal treatment for hypoglycemia caused by all forms of HI, addressing a serious unmet medical need and enabling patients to continue cancer treatments while managing hypoglycemia.
Rezolute (Nasdaq: RZLT) ha annunciato che la FDA ha concesso la Designazione di Farmaco Orfano a ersodetug per il trattamento dell'ipoglicemia dovuta all'iperinsulinismo tumorale (HI). L'azienda prevede di avviare un trial registrativo di Fase 3 per ersodetug nei pazienti con HI tumorale nel 2025. La designazione della FDA offre sette anni di esclusività di mercato e potenziali percorsi di approvazione accelerata. Ersodetug mira a fornire un trattamento universale per l'ipoglicemia causata da tutte le forme di HI, affrontando una grave esigenza medica insoddisfatta e consentendo ai pazienti di continuare i trattamenti oncologici mentre gestiscono l'ipoglicemia.
Rezolute (Nasdaq: RZLT) anunció que la FDA ha otorgado la Designación de Medicamento Huérfano a ersodetug para el tratamiento de la hipoglucemia causada por el hiperinsulinismo tumoral (HI). La empresa planea iniciar un ensayo registrativo de Fase 3 para ersodetug en pacientes con HI tumoral en 2025. La designación de la FDA ofrece siete años de exclusividad en el mercado y posibles vías de aprobación acelerada. Ersodetug busca proporcionar un tratamiento universal para la hipoglucemia causada por todas las formas de HI, abordando una necesidad médica grave no satisfecha y permitiendo que los pacientes continúen tratamientos contra el cáncer mientras manejan la hipoglucemia.
레졸루트 (Nasdaq: RZLT)는 FDA가 종양성 하이퍼인슐린증(HI)으로 인한 저혈당 치료를 위해 ersodetug에 희귀의약품 지정를 부여했다고 발표했습니다. 이 회사는 2025년에 종양 HI 환자를 위한 ersodetug의 3상 등록시험을 시작할 계획입니다. FDA의 지정은 7년의 시장 독점권과 잠재적인 신속 승인 경로를 제공합니다. Ersodetug는 모든 형태의 HI로 인한 저혈당에 대한 보편적인 치료를 제공하는 것을 목표로 하며, 중대한 unmet medical need를 해결하고 환자들이 저혈당을 관리하면서 암 치료를 계속할 수 있도록 합니다.
Rezolute (Nasdaq: RZLT) a annoncé que la FDA a accordé la désignation de médicament orphelin à ersodetug pour le traitement de l'hypoglycémie due à l'hyperinsulinisme tumoral (HI). L'entreprise prévoit de lancer un essai d'enregistrement de phase 3 pour ersodetug chez les patients atteints de HI tumoral en 2025. La désignation de la FDA offre sept ans d'exclusivité sur le marché et des voies d'approbation potentielles accélérées. Ersodetug vise à fournir un traitement universel pour l'hypoglycémie causée par toutes les formes de HI, répondant à un besoin médical non satisfait et permettant aux patients de poursuivre leurs traitements contre le cancer tout en gérant l'hypoglycémie.
Rezolute (Nasdaq: RZLT) gab bekannt, dass die FDA das Orphan Drug Designation für ersodetug zur Behandlung von Hypoglykämie aufgrund von tumoralen Hyperinsulinismus (HI) erteilt hat. Das Unternehmen plant, 2025 eine Phase-3-Zulassungsstudie für ersodetug bei Patienten mit tumoralem HI zu beginnen. Die Auszeichnung der FDA bietet sieben Jahre Marktexklusivität und potenzielle beschleunigte Genehmigungspfade. Ersodetug soll eine universelle Behandlung für Hypoglykämie bieten, die durch alle Formen von HI verursacht wird, und eine schwerwiegende unbefriedigte medizinische Notwendigkeit angehen und es Patienten ermöglichen, ihre Krebsbehandlungen fortzusetzen, während sie die Hypoglykämie managen.
- FDA granted Orphan Drug Designation, providing 7 years market exclusivity
- Potential expedited approval pathway through FDA designation
- Phase 3 trial planned for 2025, advancing clinical development
- Phase 3 trial not starting until 2025, indicating extended timeline to potential market
Insights
The FDA's Orphan Drug Designation (ODD) for ersodetug represents a significant regulatory milestone for Rezolute. The ODD brings multiple strategic advantages: 7 years of market exclusivity, potential tax credits for clinical trials and possible expedited review pathways. This designation specifically validates the drug's potential in addressing tumor hyperinsulinism, a rare condition with treatment options.
The planned Phase 3 trial in 2025 gains additional support through this designation, potentially accelerating the development timeline and reducing regulatory hurdles. The company's existing Expanded Access Program data showing real-world benefits strengthens the clinical development strategy. This regulatory win positions Rezolute favorably in the rare disease space, particularly for hypoglycemia management in tumor HI patients.
This ODD significantly enhances Rezolute's market position in the rare disease space. With market exclusivity protection and potential expedited pathways, the company gains competitive advantages in commercializing ersodetug. The tumor HI market, while niche, represents a high-value opportunity due to treatment options and serious unmet medical needs.
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Phase 3 registrational trial for ersodetug in patients with tumor hyperinsulinism (HI) expected to commence in 2025; real-world patient benefit demonstrated in Expanded Access Program
REDWOOD CITY, Calif., Dec. 03, 2024 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a late-stage biopharmaceutical company dedicated to developing transformative therapies for rare diseases with serious unmet needs, today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ersodetug for the treatment of hypoglycemia due to tumor HI.
"FDA's granting of Orphan Drug Designation is a recognition of the serious unmet need patients with tumor hyperinsulinism face and validates the potential significant benefit that ersodetug can provide," said Susan Stewart, J.D, Chief Regulatory Officer of Rezolute. "Tumor HI requires directed treatments for hypoglycemia to prevent serious adverse outcomes and improve patients’ quality of life, as well as to enable patients to continue cancer treatments. We are thrilled with FDA’s designation, which allows us to continue developing a potential universal treatment for hypoglycemia caused by all forms of HI.”
The FDA's Orphan Drug Designation is intended to encourage the development of treatments for rare diseases. Orphan Drug Designation offers benefits including seven years of market exclusivity protection and may shorten the clinical development path through potential qualification for expedited pathways to approval.
About Tumor Hyperinsulinism (HI)
Tumor HI is a rare disease that may be caused by two distinct types of tumors: islet cell tumors (ICTs) and non-islet cell tumors (NICTs), both of which lead to hypoglycemia as a result of excessive activation of the insulin receptor. Insulinomas are the most common type of ICT and may cause hypoglycemia by stimulating the over production of insulin. A variety of different NICTs, particularly hepatocellular carcinoma, can cause hypoglycemia by producing and secreting insulin-like paraneoplastic substances such as IGF-2 that bind to and activate the insulin receptor. With high morbidity and mortality rates within tumor HI, there remains a significant unmet need for new therapies directed at hypoglycemia treatment. Ersodetug has shown real-world benefit in patients with insulinoma and preclinical studies have shown that ersodetug can similarly blunt IGF-2 and insulin-mediated insulin-receptor signaling.
About Ersodetug
Ersodetug is a fully human monoclonal antibody that binds to a unique allosteric site on insulin receptors to counteract the effects of insulin receptor over-activation by insulin and related substances (such as IGF-2), thereby improving hypoglycemia in the setting of hyperinsulinism (HI). Because ersodetug acts downstream from the pancreas, it has the potential to be universally effective at treating hypoglycemia due to any form of HI.
About Rezolute, Inc.
Rezolute is a late-stage rare disease company focused on significantly improving outcomes for individuals with hypoglycemia caused by hyperinsulinism (HI). The Company’s antibody therapy, ersodetug, is designed to treat all forms of HI and has shown substantial benefit in clinical trials and real-world use for the treatment of congenital HI and tumor HI. For more information, visit www.rezolutebio.com.
Forward-Looking Statements
This release, like many written and oral communications presented by Rezolute and our authorized officers, may contain certain forward-looking statements regarding our prospective performance and strategies within the meaning of Section 27A of the Securities Act and Section 21E of the Securities Exchange Act of 1934, as amended. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and are including this statement for purposes of said safe harbor provisions. Forward-looking statements, which are based on certain assumptions and describe future plans, strategies, and expectations of Rezolute, are generally identified by use of words such as "anticipate," "believe," "estimate," "expect," "intend," "plan," "project," "seek," "strive," "try," “potential,” or future or conditional verbs such as "could," "may," "should," "will," "would," or similar expressions. These forward-looking statements include but are not limited to statements regarding the ersodetug Expanded Access Program, the ability of ersodetug to become an effective treatment for congenital hyperinsulinism, the effectiveness or future effectiveness of ersodetug for the treatment of congenital hyperinsulinism, statements regarding clinical trial timelines for ersodetug, the timing of the Phase 3 registration trial, and the FDA’s Orphan Drug Designation as it relates to ersodetug. Our ability to predict results or the actual effects of our plans or strategies is inherently uncertain. Accordingly, actual results may differ materially from anticipated results. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release. Except as required by applicable law or regulation, Rezolute undertakes no obligation to update these forward-looking statements to reflect events or circumstances that occur after the date on which such statements were made. Important factors that may cause such a difference include any other factors discussed in our filings with the SEC, including the Risk Factors contained in the Rezolute’s Annual Report on Form 10-K and Quarterly Reports on Form 10-Q, which are available on the SEC’s website at www.sec.gov. You are urged to consider these factors carefully in evaluating the forward-looking statements in this release and are cautioned not to place undue reliance on such forward-looking statements, which are qualified in their entirety by this cautionary statement.
Contact:
Rezolute, Inc.
Christen Baglaneas
cbaglaneas@rezolutebio.com
508-272-6717
FAQ
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