RedHill Biopharma's Opaganib Gets FDA Orphan Drug Designation

TL;DR

FDA grants RedHill Biopharma's opaganib Orphan Drug status for bile duct cancer - big win for exclusivity!

AI Summary

RedHill Biopharma Ltd. announced on August 26, 2024, that its drug opaganib has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of cholangiocarcinoma. This designation provides market exclusivity for seven years upon approval and potential tax credits and fee waivers.

Why It Matters

This FDA designation is a significant step for RedHill Biopharma, potentially accelerating the development and commercialization of opaganib for a rare cancer and offering market exclusivity.

Risk Assessment

Risk Level: medium — While the Orphan Drug Designation is positive, the ultimate success of opaganib depends on further clinical trials and regulatory approval, which carry inherent risks.

Key Numbers

• 7 years — Market Exclusivity (Granted by FDA upon approval due to Orphan Drug Designation)

Key Players & Entities

• RedHill Biopharma Ltd. (company) — Registrant
• Opaganib (drug) — Drug receiving designation
• U.S. Food and Drug Administration (FDA) (company) — Regulatory body granting designation
• Cholangiocarcinoma (disease) — Indication for opaganib
• August 26, 2024 (date) — Date of announcement

FAQ

Key Financial Figures

• $1.5 billion — toma market is expected to reach almost $1.5 billion before mid-2030s 3 -- This is the se

Filing Documents

• zk2431921.htm (6-K) — 40KB
• image00001.jpg (GRAPHIC) — 4KB
• 0001178913-24-002762.txt ( ) — 47KB

SIGNATURES

SIGNATURES Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized. REDHILL BIOPHARMA LTD. (the "Registrant") Date: August 26, 2024 By: /s/ Dror Ben-Asher Name: Dror Ben-Asher Title: Chief Executive Officer Press Release RedHill's Opaganib Granted Orphan Drug Designation by the FDA for Childhood Cancer, Neuroblastoma Neuroblastoma is rare but is the most common infancy malignancy with a median age of diagnosis of 17 months. In the U.S., it accounts for up to around 10% of all childhood cancer cases and 15% of pediatric cancer-related deaths 1 ,2 -- Orphan Drug designation provides for seven-years' marketing exclusivity should opaganib be approved in neuroblastoma and may confer additional benefits such as accelerated development and review times, potential grant funding and possible tax credits -- The neuroblastoma market is expected to reach almost $1.5 billion before mid-2030s 3 -- This is the second orphan drug designation by the FDA for opaganib in oncology, after cholangiocarcinoma (bile duct cancer, CCA) -- With multiple U.S. government collaborations ongoing, opaganib is a novel, host-directed, potentially broad acting, orally administered small molecule drug with demonstrated safety & efficacy profiles TEL-AVIV, Israel / RALEIGH, NC, August 26, 2024 /PRNewswire/ -- RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to opaganib for treatment of neuroblastoma, a type of childhood cancer that develops from immature nerve cells and accounts for 15% of all pediatric cancer-related deaths. Orphan Drug designation provides for a seven-year marketing exclusivity period should opaganib be approved in neuroblastoma and may confer additional benefits

Forward Looking Statements

Forward Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 and may discuss investment opportunities, stock analysis, financial performance, investor relations, and market trends. Such statements may be preceded by the words "intends," "may," "will," "plans," "expects," "anticipates," "projects," "predicts," "estimates," "aims," "believes," "hopes," "potential" or similar words and include statements regarding out-licensing of the Company's development pipeline assets, timing of opaganib's development for Acute Radiation Syndrome, non-dilutive development funding from RHB-107 and its inclusion in a key platform study. Forward-looking statements are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company's control and cannot be predicted or quantified, and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation: market and other conditions; the Company's ability to regain compliance with the Nasdaq Capital Market's minimum bid price requirements; the risk that the addition of new revenue generating products or out-licensing transactions will not occur; the risk that acceptance onto the RNCP Product Development Pipeline will not guarantee ongoing development or that any such development will not be completed or successful; the risk that the FDA does not agree with the Company's proposed development plans for opaganib for any indication; the risk that observations from preclinical studies are not indicative or predictive of results in clinical trials; the risk that the FDA pre-study requirements will not be met and/or that the Phase 3 study of RHB-107 in COVID-19 outpatients will not be approved to commence or if approved, will not be completed or, should that be the case, t

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