Fortress Biotech and Cyprium Therapeutics Announce FDA Acceptance of CUTX-101 NDA Resubmission

Rhea-AI Summary

Fortress Biotech (Nasdaq: FBIO) and majority-owned Cyprium Therapeutics announced FDA acceptance of the CUTX-101 NDA resubmission as a Class 1 resubmission with a new PDUFA target action date of January 14, 2026. Sentynl Therapeutics resubmitted the NDA on November 14, 2025 after a September 30, 2025 complete response letter that cited observations about the manufacturing site's cGMP compliance but did not identify safety or efficacy deficiencies. Clinical topline data reportedly show significant improvement in overall survival for Menkes disease patients treated early. Under the Sentynl transaction, Sentynl will transfer a Rare Pediatric Disease PRV to Cyprium if approval is issued, and Cyprium is eligible for royalties plus up to $129 million in development and sales milestones.

Positive

• FDA accepted NDA resubmission as a Class 1 resubmission
• New PDUFA date set for January 14, 2026
• Clinical topline shows significant improvement in overall survival
• Potential transfer of a Rare Pediatric Disease PRV to Cyprium upon approval
• Cyprium eligible for royalties and up to $129 million in milestones

Negative

• FDA CRL dated September 30, 2025 cited manufacturing site cGMP compliance observations
• Approval contingent on resolving manufacturing cGMP issues noted in the CRL
• Development and commercialization responsibility transferred to Sentynl (dependency risk)

Key Figures

New PDUFA date January 14, 2026 Target action date for CUTX-101 NDA

NDA resubmission date November 14, 2025 Sentynl resubmitted revised CUTX-101 NDA

CRL date September 30, 2025 FDA Complete Response Letter citing cGMP observations

Milestone potential $129 million Aggregate development and sales milestones from Sentynl

Class 1 resubmission Class 1 Regulatory classification of CUTX-101 NDA resubmission

Priority Review Voucher Rare Pediatric Disease PRV Voucher to be transferred to Cyprium if CUTX-101 is approved

Transaction effective date December 2023 Sentynl assumed development and commercialization of CUTX-101

Market Reality Check

$2.93 Last Close

Volume Volume 185,289 is below the 20-day average of 312,357 ahead of this news. low

Technical Shares at $2.93 are trading above the 200-day MA of $2.23, but 30.24% below the 52-week high.

Peers on Argus 1 Up 1 Down

FBIO’s modest -0.34% move came as close peers were mixed: CRBP up 1.57%, while KALA, MGNX and IOBT fell between about -2.78% and -6.09%. Momentum scanner names like SEER (+5.11%) and KALA (-5.21%) showed no clear sector-wide direction.

Historical Context

Date	Event	Sentiment	Move	Catalyst
Dec 10	Phase 1 results	Positive	+3.5%	Phase 1 Emrosi safety and microbiome data published with no major issues.
Nov 14	Q3 earnings	Positive	+5.2%	Q3 2025 revenue growth, swing to net income, and CUTX-101 CRL update.
Nov 12	Q3 earnings	Positive	-2.2%	Journey Medical Q3 revenue growth and Emrosi launch metrics reported.
Nov 05	Earnings date notice	Neutral	+0.4%	Announcement of upcoming Journey Medical Q3 2025 results and call.
Oct 24	Phase 3 efficacy data	Positive	-0.4%	Pooled Emrosi Phase 3 data with superior IGA success and lesion reductions.

Pattern Detected

Recent FBIO/affiliate news skewed positive, with earnings and clinical updates often met by modest price gains, though some strong clinical data saw muted or negative reactions.

Recent Company History

Over the last few months, Fortress and its affiliates have reported several key milestones. On Oct 24, pooled Phase 3 Emrosi data in 653 subjects showed strong efficacy yet the stock dipped slightly. Positive Journey Medical earnings on Nov 12 and Fortress Q3 results on Nov 14 highlighted improving revenues and profitability, with FBIO rising 5.22%. Today’s CUTX-101 NDA resubmission acceptance by the FDA follows the prior CRL citing cGMP issues and formalizes a new Jan 14, 2026 PDUFA date.

Market Pulse Summary

The stock is surging +12.3% following this news. A strong positive reaction aligns with the clearly de-risking tone of this update. The FDA’s acceptance of the CUTX-101 NDA resubmission and assignment of a new January 14, 2026 PDUFA date reframed the prior CRL as mainly a cGMP issue. Past news, such as Q3 2025 earnings with a 5.22% gain, showed investors rewarding concrete regulatory and financial progress.

Key Terms

pdufa regulatory

"the new Prescription Drug User Fee Act (PDUFA) target action date for the NDA"

PDUFA, short for the Prescription Drug User Fee Act, is a law that allows drug companies to pay fees to the government to speed up the review process for new medicines. This helps bring important drugs to market more quickly, which can impact their availability and pricing. For investors, PDUFA timelines can influence the timing of a drug’s approval and potential market success.

new drug application regulatory

"has accepted the resubmission of the New Drug Application (“NDA”) for CUTX-101"

A new drug application is a formal request submitted to government regulators seeking approval to market a new medicine. It is like a detailed proposal that shows the drug has been tested for safety and effectiveness. For investors, receiving approval signals that the drug may soon become available for sale, potentially leading to revenue growth and impacting the company's value.

nda regulatory

"has accepted the resubmission of the New Drug Application (“NDA”) for CUTX-101"

An NDA, or nondisclosure agreement, is a legal contract that keeps certain information private between parties. It’s like a promise not to share sensitive details, helping protect business ideas, strategies, or data from being leaked or used without permission. For investors, NDAs help ensure that confidential information remains secure, enabling trust and open communication during business discussions.

complete response letter regulatory

"after receiving a complete response letter (“CRL”) from the FDA on September 30, 2025"

A complete response letter is an official communication from a drug or medical-device regulator, such as the U.S. Food and Drug Administration (FDA), telling a company that a marketing application cannot be approved in its current form and listing the specific deficiencies to be fixed. For investors it matters because it pauses or delays a product’s path to market—like a building inspector issuing a list of repairs before a certificate of occupancy—affecting revenue timing, costs and stock value.

crl regulatory

"after receiving a complete response letter (“CRL”) from the FDA on September 30, 2025"

A CRL, or Complete Response Letter, is a formal notice from a drug regulator saying a drug application cannot be approved in its current form and lists what problems must be fixed. Think of it like a building inspector issuing a list of required repairs before a certificate of occupancy is granted. For investors, a CRL can delay or reduce the commercial value of a drug, affecting a company’s timeline, costs and stock outlook.

cGMP technical

"which CRL cited observations regarding the manufacturing site’s cGMP compliance"

cGMP (current Good Manufacturing Practice) are government-enforced quality standards that manufacturers must follow to ensure drugs, medical devices, and related products are made consistently, safely, and meet specified quality tests. For investors, cGMP compliance is like a restaurant passing health inspections: it reduces the risk of product recalls, regulatory fines, or production stoppages that can hurt revenue and company value, and it supports market access and long-term trust.

AI-generated analysis. Not financial advice.

New PDUFA Target Action Date of January 14, 2026 set by FDA

MIAMI, Dec. 15, 2025 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) and its majority-owned subsidiary, Cyprium Therapeutics, Inc. (“Cyprium”), today 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.

The resubmission has been accepted as a Class 1 resubmission and as a result, the new Prescription Drug User Fee Act (PDUFA) target action date for the NDA is January 14, 2026.

In December 2023, Sentynl Therapeutics, Inc. (“Sentynl”), a U.S.-based biopharmaceutical company wholly owned by Zydus Lifesciences, Ltd. (“Zydus Group”), assumed full responsibility for the development and commercialization of CUTX-101 from Cyprium. On November 14, 2025, Sentynl resubmitted the revised NDA after receiving a complete response letter (“CRL”) from the FDA on September 30, 2025, which CRL cited observations regarding the manufacturing site’s cGMP compliance. The CRL did not cite any other approvability concerns, nor did it identify any deficiencies in CUTX-101’s efficacy and safety data, which demonstrate significant improvement in overall survival for Menkes disease subjects who received early treatment with the therapy.

Pursuant to the transaction with Sentynl, Sentynl will transfer to Cyprium, if issued upon approval, a Rare Pediatric Disease Priority Review Voucher (“PRV”), and Cyprium will also be eligible to receive royalties on net sales of CUTX-101 and up to $129 million in aggregate development and sales milestones from Sentynl.

The CUTX-101 NDA was initially granted Priority Review by the FDA and is supported by positive topline clinical efficacy results for CUTX-101, demonstrating significant improvement in overall survival for Menkes disease subjects who received early treatment with CUTX-101.

About Menkes Disease
Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. The minimum birth prevalence for Menkes disease is believed to be 1 in 34,810 live male births, and potentially as high as 1 in 8,664 live male births, based on recent genome-based ascertainment (Kaler SG, Ferreira CR, Yam LS. Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome Aggregation Database (gnomAD). Molecular Genetics and Metabolism Reports 2020 June 5;24:100602). The condition is characterized by distinctive clinical features, including sparse and depigmented hair (“kinky hair”), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, failure to thrive, and neurodevelopmental delays. Mortality is high in untreated Menkes disease, with many patients dying between 2-3 years of age. Milder versions of ATP7A mutations are associated with other conditions, including Occipital Horn Syndrome and ATP7A-related Distal Motor Neuropathy. Currently, there is no FDA-approved treatment for Menkes disease and its variants.

About Cyprium Therapeutics
Cyprium Therapeutics, Inc. (“Cyprium”) is focused on the development of novel therapies for the treatment of Menkes disease and related copper metabolism disorders. In March 2017, Cyprium entered into a Cooperative Research and Development Agreement with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (“NICHD”), part of the NIH, to advance the clinical development of CUTX-101 (Copper Histidinate injection) for the treatment of Menkes disease. CUTX-101 is an investigational drug currently under FDA review to treat patients with Menkes disease. In 2023, Cyprium completed the transfer of its proprietary rights and assigned its FDA documents pertaining to CUTX-101 to Sentynl Therapeutics, Inc. Cyprium and NICHD also have an ongoing worldwide, exclusive license agreement to develop and commercialize adeno-associated virus (AAV)-based gene therapy, called AAV-ATP7A, to deliver working copies of the copper transporter that is defective in patients with Menkes disease, and to be used in combination with CUTX-101; AAV-ATP7A gene therapy is currently in pre-clinical development and has received FDA Orphan Drug Designation. Cyprium was founded by, and is a majority-owned subsidiary of, Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit www.cypriumtx.com.

About Fortress Biotech
Fortress Biotech, Inc. (“Fortress”) is an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty income. The company has eight marketed prescription pharmaceutical products and multiple programs in development at Fortress, at its majority-owned and majority-controlled partners and subsidiaries and at partners and subsidiaries it founded and in which it holds significant minority ownership positions. Fortress’ portfolio is being commercialized and developed for various therapeutic areas including oncology, dermatology, and rare diseases. Fortress’ model is focused on leveraging its significant biopharmaceutical industry expertise and network to further expand and advance the company’s portfolio of product opportunities. Fortress has established partnerships with some of the world’s leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including AstraZeneca, City of Hope, Nationwide Children’s Hospital, Columbia University, Dana Farber Cancer Center and Sentynl Therapeutics. For more information, visit www.fortressbiotech.com.

Forward-Looking Statements
Statements in this press release that are not descriptions of historical facts are “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. The words “anticipates,” “believes,” “can,” “continue,” “could,” “estimates,” “expects,” “intends,” “may,” “might,” “plans,” “potential,” “predicts,” “should,” or “will” or the negative of these terms or other comparable terminology are generally intended to identify forward-looking statements. These forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include risks relating to: the possibility that the NDA for CUTX-101 is not approved or that such NDA is approved but no PRV is issued in connection therewith; our growth strategy, financing and strategic agreements and relationships; our need for substantial additional funds and uncertainties relating to financings; uncertainty related to the timing and amounts expected to be realized from future milestone, contingent value right, royalty or similar future revenue streams, if at all; our ability to identify, acquire, close and integrate product candidates successfully and on a timely basis; our ability to attract, integrate and retain key personnel; the early stage of products under development; the results of research and development activities; uncertainties relating to preclinical and clinical testing; our ability to obtain regulatory approval for products under development; our ability to successfully commercialize products or other marketable assets for which we receive regulatory approval or receive royalties or other distributions from third parties; our ability to secure and maintain third-party manufacturing, marketing and distribution of our and our partner companies’ products and product candidates; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein.

Company Contact:
Jaclyn Jaffe
Fortress Biotech, Inc.
(781) 652-4500
ir@fortressbiotech.com

Media Relations Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
tplohoros@6degreespr.com

FAQ

What is the new PDUFA target date for CUTX-101 (FBIO/Cyprium)?

The FDA set a PDUFA target action date of January 14, 2026 for the CUTX-101 NDA resubmission.

Why was CUTX-101 resubmitted to the FDA on November 14, 2025?

Sentynl resubmitted the NDA on November 14, 2025 after a September 30, 2025 CRL that cited observations about the manufacturing site's cGMP compliance.

Does the CRL for CUTX-101 cite efficacy or safety deficiencies?

No; the CRL did not identify deficiencies in CUTX-101's efficacy or safety data and topline results show improved overall survival with early treatment.

What commercial consideration will Cyprium receive if CUTX-101 is approved?

If approved, Cyprium will receive a transferred Rare Pediatric Disease PRV, royalties on net sales, and up to $129 million in aggregate milestones from Sentynl.

Who currently holds development and commercialization responsibility for CUTX-101?

Sentynl Therapeutics assumed full responsibility for CUTX-101 development and commercialization in December 2023.