Disc Medicine Receives FDA Orphan Drug Designation for DISC-3405 for the Treatment of Polycythemia Vera

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Disc Medicine, Inc. (NASDAQ:IRON) receives Orphan Drug Designation from the FDA for DISC-3405 in treating polycythemia vera, a rare hematologic disease. The designation signifies potential benefits and support for the ongoing Phase 1 trial.
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The Orphan Drug Designation (ODD) granted by the FDA to DISC-3405 is a significant regulatory milestone for Disc Medicine, Inc. This status not only underlines the therapeutic potential of DISC-3405 for treating polycythemia vera (PV) but also provides a range of incentives designed to facilitate the drug's development path. PV, characterized by an overproduction of red blood cells, is a rare condition and a form of myeloproliferative neoplasm. Current treatment options are limited, often focusing on reducing hematocrit levels to prevent thrombosis.

From a research perspective, the incentives accompanying ODD — such as tax credits and exemption from certain FDA fees — can materially lower the financial burden of clinical trials, which are typically a substantial cost center for biopharmaceutical companies. Moreover, the seven-year marketing exclusivity post-approval is a powerful competitive advantage, potentially leading to a stronger market positioning should DISC-3405 reach commercialization. It's essential to monitor the upcoming Phase 1 trial results as they will provide initial efficacy and safety data, which are critical determinants of the drug's future prospects.

The FDA's Orphan Drug Designation for DISC-3405 is likely to be positively received by investors, as it not only suggests a recognition of the drug's potential but also aligns with strategic financial benefits. For a clinical-stage company like Disc Medicine, such milestones can have a material impact on stock valuation due to the increased probability of the drug's success and the financial incentives that reduce development costs.

Investors will closely watch the initial data release from the Phase 1 trial. Positive results could lead to increased investor confidence and upward pressure on the stock price. Conversely, any negative outcomes could significantly impact the company's valuation given its current stage of development. The long-term exclusivity period post-approval could also be factored into valuation models, potentially increasing the net present value of future cash flows from DISC-3405 sales.

The Orphan Drug Designation for DISC-3405 positions Disc Medicine within a specific niche in the hematologic disease market. PV has a relatively small patient population and the ODD suggests that DISC-3405 may address an unmet medical need within this group. The rarity of the disease and lack of treatment options could allow for premium pricing strategies if the drug is approved.

Market analysts will be evaluating the potential market size for DISC-3405, considering the prevalence of PV and competing treatments. The exclusivity period could provide a temporary monopoly, which is particularly valuable in the orphan drug market. The successful development and approval of DISC-3405 could also set a precedent for the company's capabilities in developing treatments for rare diseases, potentially impacting its future portfolio strategy and market perception.

WATERTOWN, Mass., Feb. 09, 2024 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to DISC-3405 for the treatment of patients with polycythemia vera (PV).

“Orphan drug designation is an important milestone that highlights the potential of DISC-3405 in PV, a rare disease with few treatment options,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “We look forward to sharing initial data from our ongoing Phase 1 trial of DISC-3405 in healthy volunteers in the first half of 2024.”

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.

About DISC-3405

DISC-3405 is an investigational, anti-TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) monoclonal antibody designed to increase hepcidin production and suppress serum iron. Disc in-licensed DISC-3405 from Mabwell Therapeutics in January 2023. Preclinical studies of DISC-3405 have demonstrated an increase in hepcidin production and suppression of serum iron levels in animal models of beta-thalassemia and polycythemia vera. Disc initiated a Phase 1 study of DISC-3405 in healthy volunteers in October 2023 and plans to develop DISC-3405 initially as a treatment for polycythemia vera as well as other hematologic conditions.

DISC-3405 is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide.

About Polycythemia Vera (PV)

Polycythemia vera (PV) is a chronic and rare myeloproliferative neoplasm characterized by the abnormal proliferation of red blood cells. PV affects approximately 150,000 patients in the U.S. and has a similar prevalence in Europe. The overproduction of red blood cells alters the viscosity of blood, causing it to thicken and placing patients at an elevated risk of cardiovascular and thromboembolic events, such as heart attack and stroke. Patients also experience complications such as enlarged spleen and symptoms of their disease such as fatigue, pruritis, difficulty concentrating and others. Current therapy involves phlebotomy to physically remove blood and iron to limit erythropoiesis or treatment with cytoreductive agents, with the goal of reducing red blood cell count and managing symptoms.

About Disc Medicine

Disc Medicine (NASDAQ:IRON) is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit

Disc Cautionary Statement Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to DISC-3405 and its clinical development. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.

Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: Disc’s expectations regarding the clinical development and commercialization of DISC-3405; Disc’s expectations regarding future growth and innovation; the other risks and uncertainties described in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2022, Quarterly Reports on Form 10-Q for the quarters ended March 31, 2023, June 30, 2023, and September 30, 2023 and other documents filed by Disc from time to time with the Securities and Exchange Commission (SEC), as well as discussions of potential risks, uncertainties, and other important factors in Disc’s subsequent filings with the SEC. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law.

Media Contact

Peg Rusconi
Verge Scientific Communications

Investor Relations Contact

Christina Tartaglia
Stern Investor Relations

The FDA's Orphan Drug Designation highlights the potential of DISC-3405 in treating polycythemia vera, a rare disease with limited treatment options.

Orphan Drug status offers benefits such as assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees, and seven years of post-approval marketing exclusivity.

Disc Medicine, Inc. plans to share initial data from the Phase 1 trial of DISC-3405 in healthy volunteers in the first half of 2024.
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About IRON

disc medicine is a hematology company harnessing new insights in hepcidin biology to address ineffective red blood cell production (erythropoiesis) in hematologic diseases. focused on the hepcidin pathway, the master regulator of iron metabolism, disc is advancing first-in-class therapies to transform the treatment of hematologic diseases.