FDA Grants Orphan Drug Designation for Ganaxolone in CDKL5 Disorder
June 29 2017 - 2:29PM
Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical
company dedicated to the development of innovative therapeutics to
treat epilepsy and neuropsychiatric disorders, today announced that
the U.S. Food and Drug Administration (FDA) has granted Orphan Drug
Designation to ganaxolone for the treatment of CDKL5 Disorder.
Ganaxolone is currently being evaluated in children with CDKL5
Disorder in a Phase 2 clinical trial.
“CDKL5 Disorder is a severe, rare genetic
disorder that affects children at an early age and causes
difficult-to-control seizures and neuro-developmental impairment,”
remarked Christopher M. Cashman, Chief Executive Officer of Marinus
Pharmaceuticals. “There are no approved therapies for children with
CDKL5 Disorder, and a great need for new treatment options that can
control both the seizures and co-morbidities of the disease to
improve the quality of life for the child and their family.
We are pleased to receive Orphan Drug Designation for ganaxolone in
CDKL5 Disorder and look forward to presenting the data from our
ongoing Phase 2 trial in the upcoming months.”
Orphan Drug Designation is granted by the FDA
Office of Orphan Products Development to novel drugs or biologics
that treat a rare disease or condition affecting fewer than 200,000
patients in the U.S. The designation provides the drug developer
with a seven-year period of U.S. marketing exclusivity, as well as
tax credits for clinical research costs, the ability to apply for
annual grant funding, clinical research trial design assistance and
waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
About CDKL5 Disorder
CDKL5 Disorder is a serious and rare genetic
disorder that is caused by a mutation of the cyclin-dependent
kinase-like 5 (CDKL5) gene, located on the X chromosome. It
predominantly affects girls and is characterized by early-onset,
difficult-to-control seizures and severe neuro‑developmental
impairment. The CDKL5 gene encodes a protein essential for normal
brain function. Most children affected by CDKL5 cannot walk,
talk, or care for themselves. Many also suffer from scoliosis,
visual impairment, gastrointestinal difficulties, and sleeping
disorders. Currently, there are no approved therapies for CDKL5
Disorder.
About Ganaxolone
Ganaxolone, a positive allosteric modulator of
GABAA, is being developed in three different dose forms
(intravenous, capsule, and liquid) intended to maximize therapeutic
reach to adult and pediatric patient populations in both acute and
chronic care settings. Unlike benzodiazepines, ganaxolone exhibits
anti-seizure and anti-anxiety activity via its effects on synaptic
and extrasynaptic GABAA receptors. Ganaxolone has been
studied in more than 1,500 subjects, both pediatric and adult, at
therapeutically relevant dose levels and treatment regimens for up
to two years. In these studies, ganaxolone was generally safe and
well-tolerated. The most commonly reported adverse events were
somnolence, dizziness and fatigue.
About Marinus
Pharmaceuticals
Marinus Pharmaceuticals, Inc. is a
biopharmaceutical company dedicated to the development of
ganaxolone, which offers a new mechanism of action, demonstrated
efficacy and safety, and convenient dosing to improve the lives of
patients suffering from epilepsy and neuropsychiatric disorders.
Ganaxolone is a positive allosteric modulator of GABAA that acts on
a well-characterized target in the brain known to have both
anti-seizure and anti-anxiety effects. Ganaxolone is being
developed in three different dose forms (IV, capsule and liquid)
intended to maximize therapeutic reach to adult and pediatric
patient populations in both acute and chronic care settings.
Marinus is currently evaluating ganaxolone in women with PPD and in
orphan pediatric indications for the treatment of genetic seizure
and behavior disorders, and preparing to initiate Phase 2 studies
in status epilepticus, an orphan indication. For more information
visit www.marinuspharma.com. Please follow us on Twitter:
@MarinusPharma.
Forward-Looking Statements
To the extent that statements contained in this
press release are not descriptions of historical facts regarding
Marinus, they are forward-looking statements reflecting the current
beliefs and expectations of management made pursuant to the safe
harbor provisions of the Private Securities Litigation Reform Act
of 1995. Words such as “may”, “will”, “expect”, “anticipate”,
“estimate”, “intend”, “believe”, and similar expressions (as well
as other words or expressions referencing future events, conditions
or circumstances) are intended to identify forward-looking
statements. Examples of forward-looking statements contained
in this press release include, among others, statements regarding
our interpretation of preclinical studies, development plans for
our product candidate, including the development of dose forms, the
clinical trial testing schedule and milestones, the ability to
complete enrollment in our clinical trials, interpretation of
scientific basis for ganaxolone use, timing for availability and
release of data, the safety, potential efficacy and therapeutic
potential of our product candidate and our expectation regarding
the sufficiency of our working capital. Forward-looking statements
in this release involve substantial risks and uncertainties that
could cause our clinical development programs, future results,
performance or achievements to differ significantly from those
expressed or implied by the forward-looking statements. Such
risks and uncertainties include, among others, the uncertainties
inherent in the conduct of future clinical trials, the timing of
the clinical trials, enrollment in clinical trials, availability of
data from ongoing clinical trials, expectations for regulatory
approvals, and other matters, including the development of
formulations of ganaxolone, that could affect the availability or
commercial potential of our drug candidates. Marinus
undertakes no obligation to update or revise any forward-looking
statements. For a further description of the risks and
uncertainties that could cause actual results to differ from those
expressed in these forward-looking statements, as well as risks
relating to the business of the Company in general, see filings
Marinus has made with the Securities and Exchange Commission.
CONTACT:
Lisa M. Caperelli
Executive Director, Investor & Strategic Relations
Marinus Pharmaceuticals, Inc.
484-801-4674
lcaperelli@marinuspharma.com
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