Bitget App
Trade smarter
Buy cryptoMarketsTradeFuturesEarnAISquareMore
Disc Medicine Receives Complete Response Letter from FDA for Bitopertin for the Treatment of EPP

Disc Medicine Receives Complete Response Letter from FDA for Bitopertin for the Treatment of EPP

FinvizFinviz2026/02/13 20:09
By:Finviz
  • FDA acknowledged that AURORA and BEACON provided sufficient evidence that bitopertin significantly lowers PPIX and that there is a strong mechanistic and biological plausibility supporting the use of the PPIX biomarker in protoporphyria
  • FDA indicated a need to see the results of the ongoing Phase 3 APOLLO study before making a decision
  • Ongoing Phase 3 APOLLO study potential to serve as basis for traditional approval; topline data anticipated Q4 2026

WATERTOWN, Mass., Feb. 13, 2026 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, announced that the U.S. Food and Drug Administration (FDA) today issued a Complete Response Letter (CRL) for the New Drug Application (NDA) for bitopertin as a treatment for patients with erythropoietic protoporphyria (EPP). Bitopertin has been under review for accelerated approval and as part of the Commissioner’s National Priority Voucher (CNPV) pilot program.

Accelerated approval relies on (1) whether there is evidence of an effect on the proposed surrogate endpoint (% change in whole blood metal-free PPIX) and (2) whether the proposed surrogate endpoint, including the magnitude of change, is reasonably likely to predict a clinical benefit. On the first point, the FDA agreed that AURORA and BEACON provided sufficient evidence that bitopertin significantly lowers whole blood metal-free PPIX. On the second, based on review of AURORA and BEACON results, the FDA concluded that the trials did not show evidence of association between percent change in PPIX and sunlight exposure-based endpoints, as measured in the trials, despite the strong mechanistic and biological plausibility supporting the use of the PPIX biomarker in protoporphyria. The FDA indicated results of the APOLLO study could serve as evidence to support traditional approval.

“We are committed to delivering bitopertin to patients, knowing how critical this potentially disease-modifying therapy is to the EPP community. While our efforts at utilizing expedited pathways to get bitopertin to patients quickly have not come to fruition, we are continuing to pursue all avenues in support of FDA approval,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc Medicine. “The CRL will delay the potential approval of bitopertin, but we have confidence in the ongoing APOLLO trial, for which we are seeing incredible enthusiasm from the EPP community. Confidence in our product and program guides our approach, and we will continue working closely with the FDA to support their review.”

Disc believes the issue raised is readily addressable, given the APOLLO study is already well underway with topline data expected in Q4. Disc plans to request a Type A meeting to review our approach with the FDA. A blinded sample size re-estimation of the APOLLO study was conducted in January and no modifications to sample size were needed based on statistical analysis. There has been significant patient and physician enthusiasm around the APOLLO trial, allowing Disc to complete trial enrollment in March 2026, several months earlier than expected. Upon completion of APOLLO, Disc would then file a response to the CRL and expect an updated FDA decision by mid-2027. Disc has approximately $791 million at December 31, 2025 in unaudited cash, cash equivalents, and marketable securities and maintains guidance of providing runway into 2029.

Disc Medicine will host a call for investors at 8 am ET on Tuesday, February 17

th
to discuss this outcome. Please register for the event on the Events and Presentations page of Disc’s website ( ).

A copy of the CRL will be included in a Form 8-K to be filed with the Securities and Exchange Commission, which will be accessible on ir.discmedicine.com.

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 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 .

0
0

Disclaimer: The content of this article solely reflects the author's opinion and does not represent the platform in any capacity. This article is not intended to serve as a reference for making investment decisions.

Understand the market, then trade.
Bitget offers one-stop trading for cryptocurrencies, stocks, and gold.
Trade now!

You may also like

The Capital Trends Behind the AI Computing Power Rebound: JPMorgan Fund Flows Reveal Retail Buy-In "Shrinking," Pouring Into Nvidia, SanDisk and Other Computing Power Core Companies

What has been revealed is not a "complete withdrawal of retail investors from AI," but rather a significant slowdown in overall market entry pace under macroeconomic pressure, with stock selections becoming more concentrated. In response to the Federal Reserve's unanimous decision to raise interest rates by 25 basis points, increasing the policy rate to 3.75%–4.00%, JPMorgan's assessment is: if this is simply a withdrawal of last year's "insurance-style rate cuts" during a shallow rate hike cycle—and if corporate earnings remain strong and the Middle East situation does not further spiral out of control—the stock market is still capable of absorbing rising interest rates.

智通财经2026/09/18 03:36

Vote Result 7-2! Bank of Japan Raises Interest Rates at Fastest Pace Since 1990, Does Not Signal a Clearly More Hawkish Stance

The Bank of Japan has raised interest rates to 1.25%, marking the highest level since 1995 and the sixth increase since exiting the negative interest rate policy in March 2024. Out of the nine committee members, Asada and Sato voted against the hike, citing the current economic situation, reflecting ongoing internal disagreements over further tightening. In its statement, the Bank of Japan indicated it will continue to raise rates and adjust the degree of monetary easing, but the forward guidance language showed limited changes from the July statement, without sending notably more hawkish signals.

华尔街见闻2026/09/18 03:36