CUMBERLAND PHARMACEUTICALS RECEIVES FDA ORPHAN DRUG AND RARE PEDIATRIC DISEASE DESIGNATIONS FOR NEW TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Cumberland Pharmaceuticals (NASDAQ: CPIX) announced that the FDA has granted both Orphan Drug Designation and Rare Pediatric Disease Designation to Ifetroban for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The company is completing the FIGHT DMD™ trial, a Phase II study investigating Ifetroban in DMD patients, with results expected later this year. DMD affects approximately 1 in 3,300 male births worldwide and is characterized by damage to skeletal and cardiac muscle, with cardiomyopathy being the primary cause of death. These FDA designations could provide various benefits including tax credits, user fee exemptions, and potential market exclusivity.
Cumberland Pharmaceuticals (NASDAQ: CPIX) ha annunciato che la FDA ha conferito sia la Designazione di Farmaco Orfano che la Designazione di Malattia Pediatrica Rara a Ifetroban per il trattamento della cardiomiopatia associata alla distrofia muscolare di Duchenne (DMD). L'azienda sta completando il trial FIGHT DMD™, uno studio di Fase II che indaga l'uso di Ifetroban nei pazienti affetti da DMD, con risultati attesi entro la fine di quest'anno. La DMD colpisce circa 1 su 3.300 nati maschi in tutto il mondo ed è caratterizzata da danni ai muscoli scheletrici e cardiaci, con la cardiomiopatia che rappresenta la principale causa di morte. Queste designazioni della FDA potrebbero fornire vari benefici, inclusi crediti d'imposta, esenzioni dalle tasse d'uso e potenziale esclusività di mercato.
Cumberland Pharmaceuticals (NASDAQ: CPIX) anunció que la FDA ha otorgado tanto la Designación de Medicamento Huérfano como la Designación de Enfermedad Pediátrica Rara a Ifetroban para el tratamiento de la cardiomiopatía asociada con la distrofia muscular de Duchenne (DMD). La compañía está finalizando el ensayo FIGHT DMD™, un estudio de Fase II que investiga el uso de Ifetroban en pacientes con DMD, con resultados esperados para finales de este año. La DMD afecta aproximadamente a 1 de cada 3,300 nacimientos masculinos en todo el mundo y se caracteriza por daño a los músculos esqueléticos y cardíacos, siendo la cardiomiopatía la principal causa de muerte. Estas designaciones de la FDA podrían proporcionar varios beneficios, incluidos créditos fiscales, exenciones de tarifas y potencial exclusividad en el mercado.
컴럼블랜드 제약 (NASDAQ: CPIX)은 FDA가 두창 근육 위축증(DMD)과 관련된 심근병 치료를 위해 Ifetroban에 대해 고아약 지정 및 희귀 소아 질환 지정을 부여했다고 발표했습니다. 회사는 DMD 환자에서 Ifetroban을 조사하는 2상 연구인 FIGHT DMD™ 시험을 완료하고 있으며, 결과는 올해 말에 나올 것으로 예상됩니다. DMD는 전 세계적으로 남아 3,300명당 약 1명에게 영향을 미치며, 근골격계와 심장 근육 손상을 특징으로 하며, 심근병증은 주요 사망 원인입니다. 이러한 FDA 지정은 세금 공제, 사용료 면제 및 잠재적인 시장 독점권 등 다양한 혜택을 제공할 수 있습니다.
Cumberland Pharmaceuticals (NASDAQ: CPIX) a annoncé que la FDA a accordé à Ifetroban les deux désignations de Médicament Orphelin et de Maladie Pédiatrique Rare pour le traitement de la cardiomyopathie associée à la dystrophie musculaire de Duchenne (DMD). L'entreprise est en train de finaliser l'essai FIGHT DMD™, une étude de Phase II qui examine Ifetroban chez des patients atteints de DMD, avec des résultats attendus plus tard cette année. La DMD touche environ 1 naissance masculine sur 3 300 dans le monde et se caractérise par des dommages aux muscles squelettiques et cardiaques, la cardiomyopathie étant la principale cause de décès. Ces désignations de la FDA pourraient offrir divers avantages, y compris des crédits d'impôt, des exonérations de frais et une exclusivité potentielle sur le marché.
Cumberland Pharmaceuticals (NASDAQ: CPIX) gab bekannt, dass die FDA sowohl die Orphan Drug Designation als auch die Rare Pediatric Disease Designation für Ifetroban zur Behandlung der mit der Duchenne-Muskeldystrophie (DMD) assoziierten Kardiomyopathie vergeben hat. Das Unternehmen schließt gerade die FIGHT DMD™-Studie ab, eine Phase-II-Studie, die Ifetroban bei DMD-Patienten untersucht, deren Ergebnisse noch in diesem Jahr erwartet werden. DMD betrifft weltweit etwa 1 von 3.300 männlichen Geburten und ist durch Schäden an Skelett- und Herzmuskulatur gekennzeichnet, wobei die Kardiomyopathie die Hauptursache für den Tod darstellt. Diese FDA-Auszeichnungen könnten verschiedene Vorteile bieten, darunter Steuererleichterungen, Gebührenbefreiungen und potenzielle Marktexklusivität.
- Received FDA Orphan Drug Designation providing potential 7-year market exclusivity
- Obtained Rare Pediatric Disease Designation enabling eligibility for priority review voucher
- Phase II clinical trial completion approaching with results expected this year
- Multiple regulatory incentives including tax credits and user fee exemptions
- Phase II trial results still pending and success not guaranteed
- Product still in development phase with no immediate revenue potential
- Significant competition in rare disease market
Insights
The FDA's dual designation for Ifetroban represents a significant milestone for Cumberland Pharmaceuticals' DMD program. The Orphan Drug Designation provides important benefits including tax credits and 7-year market exclusivity, while the Rare Pediatric Disease Designation offers potential for a valuable priority review voucher that could be worth
The ongoing Phase II FIGHT DMD™ trial's results, expected this year, could be transformative for Cumberland. DMD affects approximately 1 in 3,300 male births, creating a significant market opportunity despite the small patient population. Ifetroban's unique mechanism targeting cardiomyopathy - the primary cause of death in DMD - addresses an critical unmet need in treatment.
The company's parallel development of Ifetroban for systemic sclerosis and pulmonary fibrosis demonstrates broader potential applications, significantly expanding the drug's market opportunity beyond DMD.
"For Duchenne muscular dystrophy, a devastating genetic disorder affecting young boys, securing both Orphan Drug and Rare Pediatric Disease Designations for Ifetroban from the FDA is a critical step forward," said A.J. Kazimi, chief executive officer of Cumberland Pharmaceuticals. "These designations not only recognize the urgent need for effective treatments but also provide vital support to accelerate research and development. These important regulatory milestones represent hope for families and a pathway to bring transformative medicines to a vulnerable patient population more quickly and efficiently."
The
Orphan Drug Designation is granted by the
About Duchenne Muscular Dystrophy
DMD is a rare and fatal genetic disorder which affects about 1 in every 3,300 male births worldwide. Characterized by mutations in the gene responsible for producing dystrophin, DMD causes damage to the skeletal and cardiac muscle of DMD patients, with cardiomyopathy recognized as the primary cause of death. Ifetroban is being evaluated to treat the heart disease associated with DMD, which has received limited attention with current therapies.
About Ifetroban
Ifetroban is a potent and selective thromboxane-prostanoid receptor (TPr) antagonist. Ifetroban exhibits high affinity for TPr on many cell types including cardiomyocytes, platelets, vascular and airway smooth muscle, and fibroblasts, and lacks agonistic activity. In several preclinical models of muscular dystrophy, including limb-girdle and Duchenne, ifetroban prevented cardiac fibrosis and cardiac dysfunction and improved mortality.
Cumberland previously announced the acquisition of the ifetroban program in collaboration with Vanderbilt University and Cumberland Emerging Technologies.
Cumberland is also evaluating ifetroban for systemic sclerosis and pulmonary fibrosis. The ongoing FIGHTING FIBROSIS Trial is a multicenter, randomized placebo-controlled Phase II study in patients with idiopathic pulmonary fibrosis (IPF), a progressive interstitial lung disease. IPF is marked by inflammation and fibrosis of the lungs, resulting in rapidly declining lung function and reduced survival within five years of diagnosis. The study is evaluating the safety and efficacy of once daily oral Ifetroban for 52 weeks.
About Cumberland Pharmaceuticals
Cumberland Pharmaceuticals Inc. is a specialty pharmaceutical company focused on the delivery of high-quality prescription brands to improve patient care. The Company develops, acquires, and commercializes brands for the hospital acute care, gastroenterology and rheumatology market segments. The Company's portfolio of FDA-approved brands includes:
- Acetadote® (acetylcysteine) Injection, for the treatment of acetaminophen poisoning;
- Caldolor® (ibuprofen) Injection, for the treatment of pain and fever;
- Vaprisol® (conivaptan) Injection, to raise serum sodium levels in hospitalized patients with euvolemic and hypervolemic hyponatremia;
- Vibativ® (telavancin) Injection, for the treatment of certain serious bacterial infections including hospital-acquired and ventilator-associated bacterial pneumonia, as well as complicated skin and skin structure infections;
- Kristalose® (lactulose) for Oral Solution, a prescription laxative, for the treatment of chronic and acute constipation; and
- Sancuso® (granisetron) Transdermal delivery system, for the treatment of nausea and vomiting associated with chemotherapy.
For more information on Cumberland's approved products, including full prescribing information, please visit the individual product websites, links to which can be found on the Company's website www.cumberlandpharma.com.
About Cumberland Emerging Technologies
Cumberland Emerging Technologies, Inc. (www.cet-fund.com) is a joint initiative between Cumberland Pharmaceuticals Inc., Vanderbilt University, Launch Tennessee and WinHealth Pharma. The mission of CET is to bring biomedical technologies and products conceived at Vanderbilt and other regional research centers to the marketplace. CET helps manage the development and commercialization process for select projects and provides expertise on intellectual property, regulatory, manufacturing and marketing issues that are critical to successful new biomedical products. CET's Life Sciences Center, located in
Forward-Looking Statements
This press release contains forward-looking statements, which are subject to certain risks and reflect Cumberland's current views on future events based on what it believes are reasonable assumptions. No assurance can be given that these events will occur. As with any business, all phases of Cumberland's operations are subject to factors outside of its control, and any one or combination of these factors could materially affect Cumberland's results of operations. These factors include market conditions, competition, an inability of manufacturers to produce Cumberland's products on a timely basis or failure of manufacturers to comply with regulations applicable to pharmaceutical manufacturers, maintaining an effective sales and marketing infrastructure, natural disasters, public health epidemics, and other events beyond our control, as more fully discussed in the Company's most recent Form 10-K and subsequent 10-Qs as filed with the SEC. There can be no assurance that results anticipated by the Company will be realized or that they will have the expected effects. Readers are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date hereof. The Company does not undertake any obligation to publicly revise these statements to reflect events after the date hereof.
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SOURCE Cumberland Pharmaceuticals Inc.
FAQ
What FDA designations did Cumberland Pharmaceuticals (CPIX) receive for Ifetroban?
When will Cumberland Pharmaceuticals (CPIX) announce the FIGHT DMD trial results?
What is the market size for Cumberland's (CPIX) Ifetroban DMD treatment?
What benefits does the Orphan Drug Designation provide to Cumberland (CPIX)?
