CUMBERLAND PHARMACEUTICALS RECEIVES FDA ORPHAN DRUG AND RARE PEDIATRIC DISEASE DESIGNATIONS FOR NEW TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
CPIX 11.06.2024
About Gravity Analytica
"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
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
Cumberland is also evaluating ifetroban for systemic sclerosis and pulmonary fibrosis. The ongoingFIGHTING FIBROSIS Trialis 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
- 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 websitewww.cumberlandpharma.com.
About Cumberland Emerging Technologies
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
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SOURCE
Investor Contact: Shayla Simpson, Cumberland Pharmaceuticals, (615) 255-0068; Media Contact: Molly Aggas, Dalton Agency, (704) 641-6641