This section highlights FDA-related milestones and regulatory updates for drugs developed by Fortress Biotech (FBIO).
Over the past two years, Fortress Biotech has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ZYCUBO, CUTX-101, Dotinurad, MB-106, MB-101, MB-107, and Triplex. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
ZYCUBO FDA Regulatory Events
ZYCUBO is a drug developed by Fortress Biotech for the following indication: Treatment for Menkes Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ZYCUBO
- Announced Date:
- January 13, 2026
- Indication:
- Treatment for Menkes Disease
Announcement
Fortress Biotech, Inc and its majority-owned subsidiary, Cyprium Therapeutics, Inc. announced that the U.S. Food and Drug Administration ("FDA") has approved ZYCUBO® (copper histidinate, formerly known as CUTX-101) for the treatment of Menkes disease in pediatric patients.
Read Announcement
CUTX-101 FDA Regulatory Timeline and Events
CUTX-101 is a drug developed by Fortress Biotech for the following indication: Menkes disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- CUTX-101
- Announced Date:
- December 15, 2025
- Indication:
- Menkes disease
Announcement
Fortress Biotech, Inc announced that the U.S. Food and Drug Administration ("FDA") has accepted the resubmission of the New Drug Application ("NDA") for CUTX-101 (copper histidinate), intended to treat Menkes disease in pediatric patients.
AI Summary
Fortress Biotech and its majority-owned subsidiary Cyprium announced that the U.S. Food and Drug Administration has accepted the resubmission of the New Drug Application for CUTX-101 (copper histidinate) to treat Menkes disease in pediatric patients. The resubmission was accepted as a Class 1 filing, and the new PDUFA target action date is January 14, 2026. Sentynl Therapeutics is handling the development and commercialization of CUTX-101 and submitted the revised NDA.
Clinical data supporting CUTX-101 show significant improvement in overall survival for patients who receive early treatment. If the drug is approved, Sentynl will transfer a Rare Pediatric Disease Priority Review Voucher to Cyprium, and Cyprium would be eligible for royalties on net sales and up to $129 million in development and sales milestones. CUTX-101 is intended to address an unmet need for a rare, serious pediatric copper‑transport disorder with high morbidity and mortality.
Read Announcement- Drug:
- CUTX-101
- Announced Date:
- October 1, 2025
- Indication:
- Menkes disease
Announcement
Fortress Biotech, Inc. announced that the U.S. Food and Drug Administration ("FDA") has issued a Complete Response Letter ("CRL") relating to the New Drug Application ("NDA") for CUTX-101 (copper histidinate), intended to treat Menkes disease in pediatric patients.
AI Summary
Fortress Biotech and its subsidiary Cyprium Therapeutics announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the New Drug Application (NDA) of CUTX-101 (copper histidinate), intended to treat Menkes disease in pediatric patients. CUTX-101 had received Priority Review based on positive topline clinical results showing improved survival with early treatment.
The CRL identified current good manufacturing practice (cGMP) deficiencies at the facility where CUTX-101 is produced. That site has since provided detailed responses to the FDA’s September 2025 re-inspection report, and Cyprium’s partner plans to address all inspection observations swiftly.
Sentynl Therapeutics, which now leads CUTX-101 development, expects to request a meeting with the FDA soon. They will discuss the CRL findings and outline a timeline for correcting issues and resubmitting the NDA promptly.
No other approvability concerns were cited, and the FDA raised no questions about CUTX-101’s safety or efficacy data.
Read Announcement- Drug:
- CUTX-101
- Announced Date:
- March 31, 2025
- Indication:
- Menkes disease
Announcement
Fortress Biotech, Inc. announced recent corporate highlights for the full-year ended December 31, 2024.
AI Summary
Fortress Biotech, Inc. recently announced its corporate highlights for the full year ended December 31, 2024. The company described the fourth quarter as transformational, driven by key milestones that align with its strategy to enhance long‐term shareholder value through innovative product revenue, equity holdings, and royalty income. Fortress highlighted progress with its late-stage pipeline, including significant developments related to CUTX-101, with a PDUFA target set for September 30, 2025. Emphasis was placed on the continued commercial success of its dermatology portfolio and a strategic transaction where Fortress’ subsidiary Checkpoint Therapeutics is on track to be acquired by Sun Pharma. This deal is expected to generate approximately $28 million on closing, plus additional contingent value, further supporting the company’s mission to deliver novel treatments while optimizing revenue streams and advancing critical clinical programs.
Read Announcement- Drug:
- CUTX-101
- Announced Date:
- January 6, 2025
- Indication:
- Menkes disease
Announcement
Fortress Biotech, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for filing and Priority review Sentynl's New Drug Application (NDA) for CUTX-101, the product candidate for the treatment of Menkes disease.
AI Summary
Fortress Biotech, Inc. announced that the FDA has accepted and granted Priority Review for Sentynl Therapeutics’ New Drug Application (NDA) for CUTX-101, an investigational treatment for Menkes disease. The FDA’s acceptance means the agency will formally review the submitted data, with a Prescription Drug User Fee Act (PDUFA) target action date set for June 30, 2025. CUTX-101 is a copper histidinate formulation aimed at addressing the critical needs of patients with Menkes disease, a rare genetic condition that currently has no approved therapies. Sentynl, which took over full responsibility for the development and commercialization of CUTX-101 in December 2023, completed the NDA submission and will lead commercialization efforts upon approval. This milestone represents a significant step in advancing potential treatments for Menkes disease, offering hope to patients and families affected by this devastating pediatric disorder.
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Dotinurad FDA Regulatory Events
Dotinurad is a drug developed by Fortress Biotech for the following indication: To address other conditions associated with hyperuricemia such as chronic kidney disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Dotinurad
- Announced Date:
- October 21, 2025
- Indication:
- To address other conditions associated with hyperuricemia such as chronic kidney disease.
Announcement
Fortress Biotech, Inc announced that Crystalys Therapeutics, Inc. ("Crystalys"), in which Urica maintains an equity position, dosed first patients in its two randomized, double-blind, multicenter global Phase 3 trials, the RUBY study (NCT07089875) and the TOPAZ study (NCT07089888), evaluating dotinurad, a next-generation, once daily oral, URAT1 inhibitor with potential for best-in-class safety and efficacy for the treatment of gout.
AI Summary
Urica Therapeutics said Crystalys Therapeutics, in which Urica holds an equity interest, has dosed the first patients in two Phase 3 trials—RUBY and TOPAZ—testing dotinurad, a once-daily oral URAT1 inhibitor for gout.
The randomized, double-blind, multicenter RUBY study will compare dotinurad against physician-determined stable allopurinol in about 500 adults with hyperuricemia associated with gout over 64 weeks. The TOPAZ study will enroll around 250 adults with tophaceous gout for up to 76 weeks under a similar design. Dotinurad is a next-generation therapy with potential best-in-class safety and efficacy. Urica, a Fortress Biotech subsidiary, is eligible for a 3% royalty on future dotinurad sales and owns a minority equity position in Crystalys. Executives say these pivotal trials mark key steps toward potential U.S. and European approvals that could benefit millions of people living with gout.
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MB-106 FDA Regulatory Timeline and Events
MB-106 is a drug developed by Fortress Biotech for the following indication: Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- MB-106
- Announced Date:
- August 27, 2025
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Moleculin Biotech, Inc., announced the completion of its Phase 1B/2 (MB-106) clinical trial evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as AnnAraC) for the treatment of subjects with acute myeloid leukemia (AML).
AI Summary
Moleculin Biotech, Inc. announced the completion of its Phase 1B/2 (MB-106) clinical trial evaluating Annamycin combined with Cytarabine (AnnAraC) in patients with acute myeloid leukemia (AML).
The study enrolled 22 subjects across first to seventh treatment lines. Database lock is expected by the end of September, and a final clinical study report is projected for Q1 2026. Updated overall survival (OS) results show median OS of 15 months for patients achieving complete remission (n=8), 12 months in the second-line evaluable group (n=9), and 9 months in the intent-to-treat population (n=22). These outcomes exceed typical relapsed AML survival of 4–6 months.
The trial recorded a 36% complete remission rate overall and 50% in second-line patients. Median remission durability was 10 months, with no cardiotoxicity observed.
Moleculin continues to use these findings to inform its pivotal MIRACLE Phase 3 trial design.
Read Announcement- Drug:
- MB-106
- Announced Date:
- June 17, 2024
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Mustang Bio, Inc. announced that updated data from the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T-cell therapy, show a favorable safety and efficacy profile in patients with Waldenstrom macroglobulinemia ("WM"), a rare form of blood cancer.
AI Summary
Mustang Bio, Inc. announced encouraging new data from its ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T-cell therapy. The treatment showed a favorable safety and efficacy profile in patients with Waldenstrom macroglobulinemia (WM), a rare blood cancer. In this small study, 90% of the patients responded to treatment, with three achieving complete responses, including one patient who has remained in complete remission for 31 months. The patients, all of whom had been heavily pretreated and refractory to BTK inhibitors, exhibited durable responses. Only one patient required additional anti-WM treatment after MB-106, and the therapy was feasible for outpatient administration. These promising findings highlight MB-106’s potential as a treatment option for WM, a field where no FDA-approved CAR T-cell treatments currently exist.
Read Announcement- Drug:
- MB-106
- Announced Date:
- June 14, 2024
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Moleculin Biotech, Inc., reported additional efficacy findings from the Company's ongoing Phase 1B/2 (MB-106) clinical trial evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as AnnAraC) for the treatment of subjects with acute myeloid leukemia (AML).
AI Summary
Moleculin Biotech, Inc. has released new efficacy findings from its ongoing Phase 1B/2 (MB-106) trial testing Annamycin combined with Cytarabine (AnnAraC) for treating acute myeloid leukemia (AML). In the study, 22 subjects were enrolled and 20 completed efficacy evaluations. Of these patients, 45% achieved a composite complete remission (CRc), including both complete remission (CR) and CR with incomplete blood count recovery (CRi). Notably, 89% of the CRc group had poor prognosis cytogenetics or mutations. In second line treatment, AnnAraC achieved an estimated median overall survival of about 6 months, with a similarly rising median durability of response for CRc patients. Importantly, the trial demonstrated no cardiotoxicity, and the combination was generally well tolerated, supporting its potential as a promising treatment option for AML patients.
Read Announcement- Drug:
- MB-106
- Announced Date:
- May 16, 2024
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Moleculin Biotech, Inc., announced its abstract has been accepted for poster presentation at the EHA2024 Hybrid Congress being held June 13-16, 2024 in Madrid, Spain and virtually.
AI Summary
Moleculin Biotech, Inc. recently announced that its abstract has been accepted for a poster presentation at the EHA2024 Hybrid Congress. The event will take place from June 13-16, 2024 in Madrid, Spain, and will also be accessible virtually. The poster, titled “LIPOSOMAL ANNAMYCIN (L-ANN) IN COMBINATION WITH CYTARABINE FOR TREATMENT OF PATIENTS WITH ACUTE MYELOID LEUKAEMIA (AML) REFACTORY TO OR RELAPSED (R/R) AFTER INDUCTION THERAPY (MB-106 STUDY),” will be presented by Dr. Wolfram C. M. Dempke, who serves as Moleculin’s European Chief Medical Officer.
This presentation highlights the Company’s efforts in developing new treatment approaches for challenging blood cancers. Attendees at the congress will have the opportunity to learn about Moleculin’s latest research, which may pave the way for improved therapies for patients with acute myeloid leukemia.
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MB-101 FDA Regulatory Events
MB-101 is a drug developed by Fortress Biotech for the following indication: Leptomeningeal brain tumors.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- MB-101
- Announced Date:
- July 7, 2025
- Indication:
- Leptomeningeal brain tumors
Announcement
Mustang Bio, Inc announced that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation to Mustang for MB-101 (IL13Ra2-targeted CAR T-cells) for the treatment of recurrent diffuse and anaplastic astrocytoma (astrocytomas) and glioblastoma (GBM).
AI Summary
Mustang Bio, Inc. announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its MB-101 therapy. MB-101 is an IL13Ra2-targeted CAR T-cell treatment aimed at recurrent diffuse and anaplastic astrocytomas as well as glioblastoma (GBM). This designation is given for drugs intended to treat rare diseases affecting fewer than 200,000 people in the United States. It offers valuable benefits, including tax credits for clinical trials, waiver of prescription drug user fees, and seven years of market exclusivity if the treatment is approved.
The grant validates Mustang Bio’s scientific approach and supports their ongoing efforts to develop MB-101 as a potential treatment for difficult-to-treat brain tumors. The company is keen on advancing this therapy, which may work even better when paired with their MB-108 oncolytic virus for patients battling malignant glioma and other high-grade astrocytomas.
Read Announcement- Drug:
- MB-101
- Announced Date:
- July 1, 2025
- Indication:
- Leptomeningeal brain tumors
Announcement
Mustang Bio Receives FDA Orphan Drug Designation For MB-101 Treatment Of Malignant Glioma
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MB-107 FDA Regulatory Events
MB-107 is a drug developed by Fortress Biotech for the following indication: Newly diagnosed infants with X-linked severe combined immunodeficiency (XSCID) or bubble boy disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- MB-107
- Announced Date:
- June 5, 2025
- Indication:
- Newly diagnosed infants with X-linked severe combined immunodeficiency (XSCID) or bubble boy disease
Announcement
Moleculin Biotech, Inc., announced the release of its Soft Tissue Sarcoma (STS) Lung Mets KOL Webcast discussing the final data from its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases (MB-107).
AI Summary
Moleculin Biotech, Inc. recently released its Soft Tissue Sarcoma (STS) Lung Mets KOL Webcast featuring the final data from its U.S. Phase 1B/2 clinical trial. The webcast highlights the evaluation of Annamycin as a monotherapy for treating soft tissue sarcoma lung metastases (MB-107). During the presentation, key speakers including CEO Walter Klemp and Senior Chief Medical Officer Dr. Paul Waymack, along with several noted experts, discussed the promising results from the trial. The study focuses on the potential effectiveness of Annamycin, a next-generation anthracycline designed to avoid common issues such as multidrug resistance and cardiotoxicity, in managing a difficult-to-treat condition. The webcast is now available on the Moleculin website for a limited time, providing investors and stakeholders with detailed insights on the trial outcomes and the drug's future role in cancer treatment.
Read Announcement- Drug:
- MB-107
- Announced Date:
- June 4, 2025
- Indication:
- Newly diagnosed infants with X-linked severe combined immunodeficiency (XSCID) or bubble boy disease
Announcement
Moleculin Biotech, Inc today reported positive topline efficacy results from its completed U.S. Phase 1B/2 clinical trial evaluating Annamycin for the treatment of soft tissue sarcoma lung metastases ("STS lung mets") (MB-107).
AI Summary
Moleculin Biotech, Inc. today announced encouraging topline efficacy results from its U.S. Phase 1B/2 trial (MB-107) for Annamycin in treating soft tissue sarcoma lung metastases. The trial, which used a single-agent approach, established both the Maximum Tolerable Dose and the Recommended Phase 2 Dose while also evaluating safety and effectiveness. In the study, the clinical benefit rate was 59.4% among participants, with many patients showing stable disease and one achieving a partial response. Notably, patients who responded after two cycles experienced improvements in overall survival and progression-free survival, with some achieving approximately 4 months of progression-free survival and 20 months of overall survival. These results suggest that Annamycin could provide a valuable new treatment option, especially for patients who have undergone multiple prior chemotherapy regimens.
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Triplex FDA Regulatory Events
Triplex is a drug developed by Fortress Biotech for the following indication: Adults Co-Infected with HIV and CMV.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Triplex
- Announced Date:
- January 27, 2025
- Indication:
- Adults Co-Infected with HIV and CMV
Announcement
Fortress Biotech, Inc announced that the first patient was dosed in a multicenter, placebo-controlled and randomized Phase 2 clinical trial to evaluate Triplex, a cytomegalovirus ("CMV") vaccine, when administered to human leukocyte antigen ("HLA") matched related stem cell donors to reduce CMV events in patients undergoing hematopoietic stem cell transplantation ("HSCT").
AI Summary
Fortress Biotech has started a new Phase 2 clinical trial testing Triplex, a cytomegalovirus (CMV) vaccine, in a study aimed at reducing CMV events after stem cell transplants. In this trial, human leukocyte antigen (HLA) matched related donors will receive the Triplex vaccine before donating stem cells. The goal is to boost the donor’s immune response so that the transplant recipient gains protection against CMV, a virus that can cause severe complications after the procedure. This approach may provide transplant patients with earlier immunity to the virus compared to standard treatments. The study is placebo-controlled and randomized, ensuring reliable results, and it is funded by the National Cancer Institute. If successful, Triplex could offer a new way to protect vulnerable transplant patients from a common and dangerous infection.
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Cosibelimab (formerly CK-301) FDA Regulatory Events
Cosibelimab (formerly CK-301) is a drug developed by Fortress Biotech for the following indication: Metastatic cutaneous squamous cell carcinoma (mCSCC).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Cosibelimab (formerly CK-301)
- Announced Date:
- September 16, 2024
- Indication:
- Metastatic cutaneous squamous cell carcinoma (mCSCC)
Announcement
Checkpoint Therapeutics announced the presentation of longer-term data from its pivotal trial of cosibelimab, its anti-programmed death ligand-1 ("PD-L1") antibody, in locally advanced and metastatic cutaneous squamous cell carcinoma ("cSCC") during the European Society for Medical Oncology ("ESMO") Congress 2024, which is taking place in Barcelona, Spain, from September 13 to 17, 2024.
AI Summary
Checkpoint Therapeutics has announced new longer-term data for cosibelimab, its anti-PD-L1 antibody, in treating patients with locally advanced and metastatic cutaneous squamous cell carcinoma (cSCC). The data were presented at the European Society for Medical Oncology (ESMO) Congress 2024 in Barcelona, Spain, from September 13 to 17, 2024. Results from the pivotal trial showed a deepening of response over time, with higher objective response rates and increased complete response rates compared to the initial analysis. Involving 109 patients with advanced cSCC, the study highlighted that extended follow-up led to meaningful improvements in response durability, with median follow-up durations reaching 24.1 and 29.3 months in locally advanced and metastatic cases, respectively. The findings reinforce the potential of cosibelimab as a promising immunotherapy option for advanced cSCC with a sustained safety profile.
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