NASHVILLE, Tenn., Nov. 6, 2024
/PRNewswire/ -- Cumberland Pharmaceuticals Inc. (NASDAQ:
CPIX), a specialty pharmaceutical company, announced today that
the United States (U.S.) Food and
Drug Administration (FDA) granted Orphan Drug Designation and Rare
Pediatric Disease Designation to Ifetroban for the treatment of
cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
Cumberland is completing the FIGHT
DMD™ trial, a multicenter, double-blind, placebo-controlled Phase
II study investigating the pharmacokinetics, safety and efficacy of
once daily oral Ifetroban in patients with DMD. Results will be
announced later this year.
"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 U.S. FDA grants Rare Pediatric Disease Designation to
incentivize drug development for life-threatening diseases
affecting less than 200,000 children in the U.S. As these diseases
pose unique challenges to drug development, special focus is needed
to provide therapeutic options to these children. Companies that
receive approval for a drug or biologic with this designation may
be eligible for a voucher which may be redeemed for priority review
of a different product. Additionally, this voucher may be
transferred or sold to another sponsor.
Orphan Drug Designation is granted by the U.S. FDA to encourage
development of new therapies for rare diseases or conditions
affecting fewer than 200,000 people in the U.S. This designation
offers sponsors multiple incentives, which include exemption from
user fees, tax credits for qualified clinical trials and potential
market exclusivity for seven years following approval of the
product.
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 Nashville, Tennessee, provides laboratory
space, equipment and infrastructure to early-stage life sciences
companies.
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.