FDA Responds to Bitopertin Application with Complete Response Letter
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 2 days ago
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Should l Buy IRON?
Source: Newsfilter
- FDA Response: The FDA acknowledged that AURORA and BEACON trials provided sufficient evidence that Bitopertin significantly lowers PPIX, yet it requires results from the ongoing APOLLO study, indicating a cautious approach towards accelerated approval.
- Clinical Trial Progress: The APOLLO study is underway, with topline data expected in Q4 2026, and Disc Medicine plans to respond to the CRL post-study completion, which could impact the traditional approval pathway for Bitopertin.
- Financial Position: As of December 31, 2025, Disc Medicine reported approximately $791 million in cash and cash equivalents, indicating financial resilience to support operations through 2029 amidst FDA challenges.
- Market Reaction: Although the CRL delays Bitopertin's potential approval, Disc Medicine remains confident in the APOLLO trial, which is expected to garner increased enthusiasm from patients and physicians, thereby strengthening its market position in EPP treatment.
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Analyst Views on IRON
Wall Street analysts forecast IRON stock price to rise
13 Analyst Rating
13 Buy
0 Hold
0 Sell
Strong Buy
Current: 71.650
Low
100.00
Averages
120.20
High
154.00
Current: 71.650
Low
100.00
Averages
120.20
High
154.00
About IRON
Disc Medicine, Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases. The Company's pipeline includes bitopertin for the treatment of erythropoietic porphyrias (Eps), including erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), and Diamond-Blackfan Anemia (DBA); DISC-0974 for the treatment of anemia of myelofibrosis (MF), and anemia of chronic kidney disease (CKD), and DISC-3405 (formerly MWTX-003) for the treatment of polycythemia vera (PV) and other hematologic disorders. In addition, its preclinical programs also include DISC-0998, for the treatment of anemia associated with inflammatory diseases. Bitopertin is the lead product candidate in the Company's heme biosynthesis modulation portfolio. It is developing DISC-3405, a monoclonal antibody against Transmembrane Serine Protease 6 that it licensed from Mabwell Therapeutics, Inc.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Disc Medicine Shares Plunge 22% After FDA Complete Response Letter
- FDA Complete Response Letter: Disc Medicine received a Complete Response Letter from the US FDA regarding its bitopertin application, causing shares to plummet approximately 22% on Friday, highlighting the direct impact of regulatory uncertainty on the company's market performance.
- Clinical Trial Requirements: The FDA stated that it needs to see results from the phase 3 APOLLO trial before making a final decision, with topline data expected in Q4, which will directly influence the market outlook for bitopertin.
- Trial Data Context: Disc's NDA included data from the phase 2 AURORA and BEACON trials, with the FDA noting that these results indicated bitopertin significantly lowered levels of PPIX, the molecule in the skin causing erythropoietic protoporphyria, suggesting its potential efficacy.
- Accelerated Approval Program: Bitopertin is part of the FDA Commissioner's National Priority Voucher pilot program for accelerated approval review, indicating its significance and potential market opportunities in the treatment of rare diseases despite facing challenges.
See More
Disc Medicine's Drug Approval Denied, Shares Plunge 21%
- FDA Denial: Disc Medicine's bitopertin oral tablets were denied FDA approval, causing a 21% drop in share price on Friday, reflecting market concerns over the sufficiency of clinical data and impacting investor confidence.
- Clinical Trial Requirements: The FDA has mandated that the company provide evidence of clinical benefit from 'adequate and well-controlled trials' before approval, with Disc planning to resubmit its application after completing the APOLLO trial, expecting a new decision by mid-2027.
- Fast Track Review: Bitopertin is one of 18 drugs under the FDA's National Priority Voucher program initiated in June 2025, which aims to reduce review times from the standard 10-12 months to 1-2 months, indicating the FDA's recognition of the drug's potential.
- Market Sentiment Shift: Despite the FDA's rejection, retail sentiment on Stocktwits for IRON stock shifted from 'bearish' to 'bullish', with message volume increasing from 'normal' to 'high', indicating investor optimism regarding potential future approval.
See More
FDA Responds to Bitopertin Application with Complete Response Letter
- FDA Response: The FDA acknowledged that AURORA and BEACON trials provided sufficient evidence that Bitopertin significantly lowers PPIX, yet it requires results from the ongoing APOLLO study, indicating a cautious approach towards accelerated approval.
- Clinical Trial Progress: The APOLLO study is underway, with topline data expected in Q4 2026, and Disc Medicine plans to respond to the CRL post-study completion, which could impact the traditional approval pathway for Bitopertin.
- Financial Position: As of December 31, 2025, Disc Medicine reported approximately $791 million in cash and cash equivalents, indicating financial resilience to support operations through 2029 amidst FDA challenges.
- Market Reaction: Although the CRL delays Bitopertin's potential approval, Disc Medicine remains confident in the APOLLO trial, which is expected to garner increased enthusiasm from patients and physicians, thereby strengthening its market position in EPP treatment.
See More
Pomerantz LLP Investigates Securities Fraud Claims Against Disc Medicine
- Securities Fraud Investigation: Pomerantz LLP is investigating claims of securities fraud against Disc Medicine, Inc., focusing on whether the company and its executives engaged in unlawful business practices that could lead to significant investor losses.
- FDA Review Delay: On January 15, 2026, it was reported that a drug under development by Disc faced a delay in its review timeline due to safety and efficacy concerns raised by FDA scientists, directly impacting market confidence in the company.
- Significant Stock Drop: Following the FDA news, Disc's stock price fell by $6.04, or 7.84%, closing at $71.04 per share on January 15, 2026, reflecting investor concerns about the company's future prospects.
- Potential Legal Action: Pomerantz LLP is advising affected investors to reach out, indicating the possibility of a class action lawsuit, which could further exacerbate Disc's legal and financial pressures in the market.
See More
Disc Medicine's Drug Approval Delayed to Feb 10, Stock Rises 2.59%
- Approval Delay: The FDA has postponed its decision on Disc Medicine's experimental treatment bitopertin to February 10, yet the stock rose 2.59%, indicating market optimism about its future prospects.
- Efficacy Concerns: FDA reviewers questioned the statistical reliability of the secondary endpoint “pain-free time in the sun,” which could impact the drug's approval process and add regulatory uncertainty.
- Internal Review: The FDA has requested an evaluation of bitopertin's potential for abuse, complicating the review process, although analysts believe this may just be noise and unlikely to significantly affect the outcome.
- Market Expectations: H.C. Wainwright maintains a “Buy” rating on Disc Medicine with a price target of $118, reflecting strong market confidence in the company's future.
See More
FDA Delays Disc Medicine's Bitopertin Drug Review, Analysts See Minor Impact
- Review Delay: The FDA has postponed its decision on Disc Medicine's rare disease drug bitopertin, raising review questions; however, analysts generally believe this setback will have a limited impact on the company.
- Market Reaction: Analysts indicate that while the FDA's delay may affect short-term market expectations, it does not pose a substantial threat to Disc Medicine's long-term outlook, with investor sentiment remaining stable.
- Drug Potential: If approved, bitopertin, aimed at treating rare diseases, could open new revenue streams for the company, and analysts believe the market demand for this drug remains strong.
- Strategic Adjustment: Disc Medicine may need to reassess its marketing strategy to address the uncertainties brought by the FDA review delay, ensuring it maintains a competitive edge in the future.
See More
Disc Medicine Shares Plunge 22% After FDA Complete Response Letter
- FDA Complete Response Letter: Disc Medicine received a Complete Response Letter from the US FDA regarding its bitopertin application, causing shares to plummet approximately 22% on Friday, highlighting the direct impact of regulatory uncertainty on the company's market performance.
- Clinical Trial Requirements: The FDA stated that it needs to see results from the phase 3 APOLLO trial before making a final decision, with topline data expected in Q4, which will directly influence the market outlook for bitopertin.
- Trial Data Context: Disc's NDA included data from the phase 2 AURORA and BEACON trials, with the FDA noting that these results indicated bitopertin significantly lowered levels of PPIX, the molecule in the skin causing erythropoietic protoporphyria, suggesting its potential efficacy.
- Accelerated Approval Program: Bitopertin is part of the FDA Commissioner's National Priority Voucher pilot program for accelerated approval review, indicating its significance and potential market opportunities in the treatment of rare diseases despite facing challenges.
See More
Disc Medicine's Drug Approval Denied, Shares Plunge 21%
- FDA Denial: Disc Medicine's bitopertin oral tablets were denied FDA approval, causing a 21% drop in share price on Friday, reflecting market concerns over the sufficiency of clinical data and impacting investor confidence.
- Clinical Trial Requirements: The FDA has mandated that the company provide evidence of clinical benefit from 'adequate and well-controlled trials' before approval, with Disc planning to resubmit its application after completing the APOLLO trial, expecting a new decision by mid-2027.
- Fast Track Review: Bitopertin is one of 18 drugs under the FDA's National Priority Voucher program initiated in June 2025, which aims to reduce review times from the standard 10-12 months to 1-2 months, indicating the FDA's recognition of the drug's potential.
- Market Sentiment Shift: Despite the FDA's rejection, retail sentiment on Stocktwits for IRON stock shifted from 'bearish' to 'bullish', with message volume increasing from 'normal' to 'high', indicating investor optimism regarding potential future approval.
See More
FDA Responds to Bitopertin Application with Complete Response Letter
- FDA Response: The FDA acknowledged that AURORA and BEACON trials provided sufficient evidence that Bitopertin significantly lowers PPIX, yet it requires results from the ongoing APOLLO study, indicating a cautious approach towards accelerated approval.
- Clinical Trial Progress: The APOLLO study is underway, with topline data expected in Q4 2026, and Disc Medicine plans to respond to the CRL post-study completion, which could impact the traditional approval pathway for Bitopertin.
- Financial Position: As of December 31, 2025, Disc Medicine reported approximately $791 million in cash and cash equivalents, indicating financial resilience to support operations through 2029 amidst FDA challenges.
- Market Reaction: Although the CRL delays Bitopertin's potential approval, Disc Medicine remains confident in the APOLLO trial, which is expected to garner increased enthusiasm from patients and physicians, thereby strengthening its market position in EPP treatment.
See More
Pomerantz LLP Investigates Securities Fraud Claims Against Disc Medicine
- Securities Fraud Investigation: Pomerantz LLP is investigating claims of securities fraud against Disc Medicine, Inc., focusing on whether the company and its executives engaged in unlawful business practices that could lead to significant investor losses.
- FDA Review Delay: On January 15, 2026, it was reported that a drug under development by Disc faced a delay in its review timeline due to safety and efficacy concerns raised by FDA scientists, directly impacting market confidence in the company.
- Significant Stock Drop: Following the FDA news, Disc's stock price fell by $6.04, or 7.84%, closing at $71.04 per share on January 15, 2026, reflecting investor concerns about the company's future prospects.
- Potential Legal Action: Pomerantz LLP is advising affected investors to reach out, indicating the possibility of a class action lawsuit, which could further exacerbate Disc's legal and financial pressures in the market.
See More
Disc Medicine's Drug Approval Delayed to Feb 10, Stock Rises 2.59%
- Approval Delay: The FDA has postponed its decision on Disc Medicine's experimental treatment bitopertin to February 10, yet the stock rose 2.59%, indicating market optimism about its future prospects.
- Efficacy Concerns: FDA reviewers questioned the statistical reliability of the secondary endpoint “pain-free time in the sun,” which could impact the drug's approval process and add regulatory uncertainty.
- Internal Review: The FDA has requested an evaluation of bitopertin's potential for abuse, complicating the review process, although analysts believe this may just be noise and unlikely to significantly affect the outcome.
- Market Expectations: H.C. Wainwright maintains a “Buy” rating on Disc Medicine with a price target of $118, reflecting strong market confidence in the company's future.
See More
FDA Delays Disc Medicine's Bitopertin Drug Review, Analysts See Minor Impact
- Review Delay: The FDA has postponed its decision on Disc Medicine's rare disease drug bitopertin, raising review questions; however, analysts generally believe this setback will have a limited impact on the company.
- Market Reaction: Analysts indicate that while the FDA's delay may affect short-term market expectations, it does not pose a substantial threat to Disc Medicine's long-term outlook, with investor sentiment remaining stable.
- Drug Potential: If approved, bitopertin, aimed at treating rare diseases, could open new revenue streams for the company, and analysts believe the market demand for this drug remains strong.
- Strategic Adjustment: Disc Medicine may need to reassess its marketing strategy to address the uncertainties brought by the FDA review delay, ensuring it maintains a competitive edge in the future.
See More
