Keros Announces U.S. FDA Orphan Drug Designation Granted to KER-065 for the Treatment of Duchenne Muscular Dystrophy
Keros Therapeutics (Nasdaq: KROS) has received Orphan Drug designation from the FDA for KER-065, its investigational therapy for Duchenne muscular dystrophy (DMD). This milestone comes as the company prepares to advance KER-065 into Phase 2 clinical trials for DMD patients.
The Orphan Drug designation provides significant benefits, including tax credits for clinical testing, potential FDA fee waivers, and seven years of market exclusivity upon approval. This designation is granted to therapies targeting rare diseases affecting fewer than 200,000 people in the United States.
Keros Therapeutics (Nasdaq: KROS) ha ottenuto la designazione di Farmaco Orfano dalla FDA per KER-065, il suo trattamento sperimentale per la distrofia muscolare di Duchenne (DMD). Questo traguardo arriva mentre la società si prepara a far avanzare KER-065 nella Fase 2 degli studi clinici per pazienti con DMD.
La designazione di Farmaco Orfano offre vantaggi rilevanti, tra cui crediti d'imposta per la sperimentazione clinica, possibili esenzioni dalle tariffe FDA e sette anni di esclusività di mercato in caso di approvazione. Tale designazione è riservata a terapie per malattie rare che colpiscono meno di 200.000 persone negli Stati Uniti.
Keros Therapeutics (Nasdaq: KROS) ha recibido la designación de Medicamento Huérfano por parte de la FDA para KER-065, su terapia experimental para la distrofia muscular de Duchenne (DMD). Este hito se produce mientras la compañía se prepara para avanzar KER-065 a los ensayos clínicos de Fase 2 en pacientes con DMD.
La designación de Medicamento Huérfano aporta beneficios importantes, como créditos fiscales para las pruebas clínicas, posibles exenciones de tasas de la FDA y siete años de exclusividad en el mercado tras la aprobación. Esta designación se concede a terapias dirigidas a enfermedades raras que afectan a menos de 200.000 personas en Estados Unidos.
Keros Therapeutics (Nasdaq: KROS)가 듀센 근이영양증(DMD) 치료 후보물질 KER-065에 대해 FDA로부터 희소의약품(Orphan Drug) 지정을 받았습니다. 이 성과는 회사가 KER-065를 DMD 환자를 대상으로 하는 2상 임상시험으로 진행할 준비를 하는 가운데 이루어졌습니다.
희소의약품 지정은 임상시험 세액공제, FDA 수수료 면제 가능성, 승인 시 7년간의 시장 독점권 등 중요한 혜택을 제공합니다. 이 지정은 미국 내 20만 명 미만의 환자가 있는 희귀질환을 표적하는 치료제에 부여됩니다.
Keros Therapeutics (Nasdaq: KROS) a obtenu la désignation de médicament orphelin de la FDA pour KER-065, son traitement expérimental contre la dystrophie musculaire de Duchenne (DMD). Cette étape intervient alors que la société se prépare à faire progresser KER-065 vers des essais cliniques de phase 2 chez les patients atteints de DMD.
La désignation de médicament orphelin confère des avantages importants, notamment des crédits d'impôt pour les essais cliniques, d'éventuelles exonérations de frais FDA et sept ans d'exclusivité commerciale en cas d'approbation. Cette désignation est accordée aux thérapies ciblant des maladies rares touchant moins de 200 000 personnes aux États-Unis.
Keros Therapeutics (Nasdaq: KROS) hat von der FDA die Orphan-Drug-Zulassung für KER-065 erhalten, seine Untersuchungs- therapie gegen Duchenne-Muskeldystrophie (DMD). Dieser Meilenstein fällt in die Vorbereitungsphase des Unternehmens zur Weiterführung von KER-065 in Phase-2-Studien bei DMD-Patienten.
Die Orphan-Drug-Zulassung bringt bedeutende Vorteile, darunter Steuergutschriften für klinische Tests, mögliche Befreiungen von FDA-Gebühren und sieben Jahre Marktexklusivität im Falle einer Zulassung. Diese Zulassung wird Therapien für seltene Erkrankungen gewährt, die in den USA weniger als 200.000 Menschen betreffen.
- FDA Orphan Drug designation granted for KER-065
- Seven years of market exclusivity potential if approved
- Tax credits and FDA fee waivers available through designation
- Advancement to Phase 2 clinical trials
- None.
LEXINGTON, Mass., Aug. 20, 2025 (GLOBE NEWSWIRE) -- Keros Therapeutics, Inc. (“Keros”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta (“TGF-ß”) family of proteins, today announced the U.S. Food and Drug Administration (“FDA”) granted Orphan Drug designation for KER-065 for the treatment of Duchenne muscular dystrophy (“DMD”).
“Receiving Orphan Drug designation for KER-065 highlights the significant unmet medical need for patients with DMD,” said Jasbir S. Seehra, President and Chief Executive Officer. “This designation serves as a significant milestone for Keros as we advance KER-065 into a Phase 2 clinical trial in patients with DMD.”
The FDA grants Orphan Drug designation to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. This designation provides certain potential benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved.
About KER-065
KER-065 is a novel ligand trap comprised of a modified ligand-binding domain derived from activin receptor type IIA and activin receptor type IIB that is fused to the portion of the human antibody known as the Fc domain. KER-065 is designed to act as a ligand trap and inhibit the biological effects of myostatin and activin A, two ligands that signal through activin receptors, to increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle and increase bone strength. We are developing KER-065 for the treatment of neuromuscular diseases, with an initial focus on DMD.
About Duchenne Muscular Dystrophy (DMD)
DMD is the most common form of muscular dystrophy and results in muscle degeneration and premature death. DMD results from the lack of functional dystrophin protein that helps promote myofiber stability, caused by a gene mutation. The lack of dystrophin, an important structural component of muscle cells, causes muscle cells to have increased susceptibility to damage and to progressively die. Additionally, the absence of dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and the replacement of muscle with fibrotic and fatty tissue. The replacement of muscle fibers with fatty and fibrotic tissue leads to progressive loss of muscle strength and function leading to immobility and respiratory and cardiac complications. In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD. The National Organization for Rare Disorders estimates that approximately one in every 3,500 male births is affected by DMD worldwide.
About Keros Therapeutics, Inc.
Keros is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the TGF-ß family of proteins. Keros is a leader in understanding the role of the TGF-ß family of proteins, which are master regulators of the growth, repair and maintenance of a number of tissues, including blood, bone, skeletal muscle, adipose and heart tissue. By leveraging this understanding, Keros has discovered and is developing protein therapeutics that have the potential to provide meaningful and potentially disease-modifying benefit to patients. Keros’ lead product candidate, KER-065, is being developed for the treatment of neuromuscular diseases, with an initial focus on DMD. Keros’ most advanced product candidate, elritercept, is being developed for the treatment of cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndrome and in patients with myelofibrosis.
Cautionary Note Regarding Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Words such as “anticipates,” “believes,” “continue,” “expects,” “enable,” “intention,” “potential” and “will” or similar expressions are intended to identify forward-looking statements. Examples of these forward-looking statements include statements concerning: Keros’ expectations regarding its strategy, progress and timing of its clinical trials for KER-065; and the potential benefit of an Orphan Drug designation for KER-065 in DMD. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Keros’ limited operating history and historical losses; Keros’ ability to raise additional funding to complete the development and any commercialization of its product candidates; Keros’ dependence on the success of its product candidates, KER-065 and elritercept; that Keros may be delayed in initiating, enrolling or completing any clinical trials; competition from third parties that are developing products for similar uses; Keros’ ability to obtain, maintain and protect its intellectual property; and Keros’ dependence on third parties in connection with manufacturing, clinical trials and preclinical studies.
These and other risks are described more fully in Keros’ filings with the Securities and Exchange Commission (the “SEC”), including the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q, filed with the SEC on August 6, 2025, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Keros undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Contacts
Investor Contact:
Justin Frantz
jfrantz@kerostx.com
617-221-6042
Media Contact:
Mahmoud Siddig / Adam Pollack / Viveca Tress
Joele Frank, Wilkinson Brimmer Katcher
(212) 355-4449
FAQ
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