Disc Medicine Announces Receipt of FDA Commissioner’s National Priority Voucher (CNPV) for Bitopertin in Erythropoietic Protoporphyria (EPP)

Rhea-AI Summary

Disc Medicine (NASDAQ:IRON) received a Commissioner’s National Priority Voucher (CNPV) from the FDA for bitopertin in erythropoietic protoporphyria (EPP) on Oct 16, 2025.

Disc submitted a New Drug Application (NDA) in September 2025 seeking accelerated approval for patients aged 12 and older. Bitopertin holds Orphan Drug and Rare Pediatric Disease designations. The CNPV program can shorten FDA review to 1–2 months and provides enhanced communications and rolling review benefits.

Positive

• CNPV awarded enabling potential 1–2 month FDA review
• NDA for bitopertin submitted in September 2025
• Bitopertin has Orphan Drug and Rare Pediatric Disease designations

Negative

• None.

Insights

Regulatory Strategy Expert

Receipt of an FDA Commissioner’s National Priority Voucher (CNPV) materially accelerates review timing for bitopertin in EPP.

Disc Medicine secured a CNPV for bitopertin and filed a New Drug Application under the FDA’s accelerated approval pathway for patients aged 12 years and older. The voucher grants benefits including enhanced communications, rolling review, and a reduced target review window of 1-2 months, materially shortening standard review timelines.

The practical dependency is clear: successful accelerated approval still requires the Agency’s evaluation of the NDA dossier and any confirmatory data obligations tied to accelerated pathways. Risks include potential information requests, need for post-approval confirmatory studies, and review outcomes that can remain uncertain until the FDA completes its shortened review. Watch for the FDA’s substantive review milestones and any communication on outstanding data requests in the coming weeks to months. This change in review cadence shortens the timeline to a regulatory decision and therefore is a near-term, monitorable event for stakeholders.

• Disc is seeking accelerated approval of bitopertin for patients aged 12 years and older with EPP
• The CNPV program is designed to reduce the drug application review process to 1-2 months

WATERTOWN, Mass., Oct. 16, 2025 (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 it received a Commissioner’s National Priority Voucher (CNPV) from the U.S. Food and Drug Administration (FDA) for bitopertin in erythropoietic protoporphyria (EPP), including X-linked protoporphyria (XLP). Disc submitted a New Drug Application (NDA) to the FDA for bitopertin for patients aged 12 years and older with EPP following the FDA’s accelerated approval pathway in September 2025. Bitopertin has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA.

“We are honored to have received this voucher which underscores the potential impact of bitopertin and reinforces the immense need EPP patients have for a new therapy that may address the underlying cause of their disease,” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. “We are grateful to the FDA for implementing this innovative new voucher program and we look forward to working closely with the Agency to bring bitopertin to patients as soon as possible. All of this would not be possible without collaboration from the EPP patient and caregiver community, and we thank them for their contributions.”

The Commissioner’s National Priority Voucher program, announced in June 2025, is designed to accelerate the development and review of certain drugs aligned with US national health priorities by offering the opportunity to reduce drug application review times to 1-2 months. Additionally, companies selected for the program will be issued a voucher entitling them to benefits including enhanced communications and rolling review to allow for shortened review time.

Erythropoietic protoporphyria is a rare, debilitating and potentially life-threatening disease caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate called protoporphyrin IX (PPIX). This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensations and potential blistering and disfigurement. PPIX also accumulates in the hepatobiliary system and can result in complications including gallstones, cholestasis, and liver damage in 20-30% of patients and in extreme cases liver failure. Current standard of care involves extreme measures to avoid sunlight, including restricting outdoor activities to nighttime, use of protective clothing and opaque shields, and pain management. This has a significant impact on the psychosocial development, quality of life, and daily activities of patients, particularly in young children and families.

About Bitopertin

Bitopertin is an investigational, clinical-stage, orally administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is developing bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Bitopertin has been studied in multiple clinical trials in patients with EPP, including the Phase 2 open-label BEACON trial, the Phase 2 double-blind, placebo-controlled AURORA trial, an open-label extension HELIOS trial, and the confirmatory Phase 3 double-blind, placebo-controlled APOLLO trial.

Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021.

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 www.discmedicine.com.

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 the registrational pathway for bitopertin, including the timeline for the FDA’s review of the NDA submission and the potential benefits of the Commissioner’s National Priority Voucher program; and the potential of bitopertin as a therapeutic drug. 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: the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; the content and timing of decisions made by the FDA and other regulatory authorities; and the other risks and uncertainties described in Disc’s filings with the SEC, including in the “Risk Factors” section of Disc’s Annual Report on Form 10-K for the year ended December 31, 2024, and in subsequent Quarterly Reports on Form 10-Q. 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,

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Precision AQ
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FAQ

What does the CNPV mean for Disc Medicine (IRON) and bitopertin timelines?

CNPV may reduce the FDA review clock to 1–2 months and provides enhanced communications and rolling review.

When did Disc submit the NDA for bitopertin (IRON) in EPP?

Disc submitted the NDA in September 2025 seeking accelerated approval for patients aged 12 and older.

Has bitopertin (IRON) received any FDA designations that affect review?

Yes. Bitopertin has Orphan Drug and Rare Pediatric Disease designations from the FDA.

What patient population is the bitopertin NDA (IRON) seeking approval for?

The NDA seeks accelerated approval for patients aged 12 years and older with erythropoietic protoporphyria (EPP).

What benefits come with the Commissioner’s National Priority Voucher for IRON?

The voucher offers shortened review time (1–2 months), enhanced communications, and rolling review capabilities.

How does the CNPV program align with US health priorities for IRON’s drug?

The CNPV is designed to accelerate development and review of drugs aligned with national health priorities, qualifying bitopertin for expedited review benefits.