Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company")
(Nasdaq: CPRX) today reported that Santhera Pharmaceuticals
("Santhera") has obtained U.S. Food and Drug Administration ("FDA")
approval for AGAMREE® (vamorolone) oral suspension 40 mg/mL for use
in treating Duchenne Muscular Dystrophy ("DMD") in patients aged
two years and older. AGAMREE offers a novel corticosteroid
treatment option for DMD, addressing a significant unmet medical
need. In July 2023, Catalyst secured the exclusive North American
license and commercial rights for AGAMREE from Santhera for DMD and
other potential indications, bolstering its neuroscience commercial
portfolio with a highly synergistic neuromuscular asset. As part of
that transaction, Santhera will promptly transfer the approved New
Drug Application for AGAMREE to Catalyst.
FDA’s approval of AGAMREE® was based on the data
from the pivotal Phase 2b VISION-DMD study as supplemented with
safety information collected from three open-label studies,
including extension studies. In these trials, AGAMREE was
administered at doses ranging from 2 to 6 mg/kg/day, extending for
a period of up to 48 months. Compared with current standard-of-care
corticosteroids, this novel corticosteroid treatment exhibited
comparable efficacy, with data suggesting a reduction in adverse
events, notably related to bone health, growth trajectory, and
behavior.
“We strongly believe that this novel steroid has
the transformational potential to make a significant difference for
patients living with Duchenne Muscular Dystrophy and potentially
other chronic inflammatory diseases. The approval of AGAMREE
underscores the potential of reshaping the DMD treatment paradigm
for this life-threatening rare disease. The addition of AGAMREE
expands our rare neuromuscular disease portfolio, and we look
forward to executing on our proven commercial capabilities to
bolster our long-term growth potential,” said Patrick J. McEnany,
Chairman and CEO of Catalyst Pharmaceuticals. “Our unwavering
commitment extends beyond this important milestone as we are
resolute in our mission to ensure that DMD patients in the U.S.
have access to this treatment option as we believe that AGAMREE may
offer the potential of increasing the duration of ambulation and
mobility in these patients, thereby significantly improving their
overall quality of life. We expect to launch the product in the
first quarter of 2024. At that time, we will introduce a
comprehensive financial assistance program aimed at helping ensure
accessibility and minimizing patient co-pays and deductibles,
thereby enhancing affordability for all DMD patients. We look
forward to successfully commercializing this product with a
continued commitment to serving our patient communities."
Upon the transfer of AGAMREE's NDA into its
neuromuscular franchise, Catalyst will harness its product
portfolio synergies by leveraging its well-established expertise
and proven commercial capabilities. The Company plans to launch the
product in Q1 2024, spearheaded by its seasoned and experienced
U.S. commercial and medical affairs neuromuscular teams.
AGAMREE was granted Orphan Drug and Rare
Pediatric Disease designations status for DMD in
the U.S. and will be eligible for seven years of orphan
drug exclusivity upon approval date and has issued pending patents
that could provide protection until 2040.
About Duchenne Muscular Dystrophy
(DMD)DMD, the most common form of muscular dystrophy, is a
rare and life-threatening neuromuscular disorder characterized by
progressive muscle dysfunction, ultimately leading to loss of
ambulation, respiratory failure, and fatality. Current standard
treatment for DMD involves corticosteroids, which often come with
significant side effects. It is estimated that between 11,000 and
13,000 patients in the U.S. are affected by DMD, with approximately
70% of patients currently receiving concomitant corticosteroid
treatment.
About AGAMREE® (vamorolone)
AGAMREE's unique mode of action is based on
differential effects on glucocorticoid and mineralocorticoid
receptors and modifying further downstream activity and, as such,
is considered a novel corticosteroid with dissociative properties
in maintaining efficacy, with a better-tolerated side effect
profile. This mechanism of action may allow vamorolone to emerge as
an effective alternative to the current standard of care
corticosteroids in children, adolescents, and adult patients with
DMD. In the pivotal VISION-DMD study, vamorolone met the primary
endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002)
at 24 weeks of treatment and showed a good safety and tolerability
profile. The most commonly reported adverse events versus placebo
from the VISION-DMD study were cushingoid features, vomiting, and
vitamin D deficiency. Adverse events were generally of mild to
moderate severity.
AGAMREE was granted U.S FDA approval on October
26, 2023, and was granted Orphan Drug and Rare Pediatric Disease
designations status for DMD in the U.S. and making it
eligible for seven years of orphan drug exclusivity upon approval.
AGAMREE also has issued and pending patents that could provide
protection until 2040. In Europe, it has received Promising
Innovative Medicine (PIM) status from the UK MHRA for
DMD.
About Catalyst Pathways AGAMREE®
Patient Assistance Program
AGAMREE will be available in the U.S. and
marketed by Catalyst, supported by its Catalyst Pathway Program®.
This comprehensive patient support program includes a dedicated,
personalized support team that assists families throughout the
AGAMREE treatment journey, from answering questions to coordinating
financial assistance programs for eligible patients. For more
information, caregivers and healthcare professionals can visit
www.yourcatalystpathways.com or call 1-833-422-8259.
About Catalyst Pharmaceuticals
With exceptional patient focus, Catalyst is
committed to developing and commercializing innovative
first-in-class medicines that address rare neurological and
epileptic diseases. Catalyst's flagship U.S. commercial
product is FIRDAPSE® (amifampridine) Tablets 10 mg, approved
for the treatment of Lambert-Eaton myasthenic syndrome ("LEMS") for
adults and for children ages six to seventeen. In January
2023, Catalyst acquired the U.S. commercial rights to
FYCOMPA® (perampanel) CIII, a prescription medicine approved
in people with epilepsy aged four and older alone or with other
medicines to treat partial-onset seizures with or without
secondarily generalized seizures and with other medicines to treat
primary generalized tonic-clonic seizures for people with epilepsy
aged 12 and older. Further, Canada's national healthcare
regulatory agency, Health Canada, has approved the use of
FIRDAPSE for the treatment of adult patients
in Canada with LEMS. Finally, on July 18, 2023,
Catalyst acquired an exclusive license for North
America for AGAMREE (vamorolone) oral suspension 40 mg/mL, a
novel corticosteroid treatment for Duchenne Muscular Dystrophy.
AGAMREE previously received FDA Orphan Drug and Fast Track
designations and was approved for commercialization in the U.S. FDA
on October 26, 2023.
For more information about Catalyst
Pharmaceuticals, Inc., visit the Company's website
at www.catalystpharma.com. For Full Prescribing and Safety
Information for FIRDAPSE®, visit www.firdapse.com. For Full
Prescribing Information, including Boxed WARNING for FYCOMPA®,
please visit www.fycompa.com.
Forward-Looking Statements
This press release contains forward-looking
statements. Forward-looking statements involve known and unknown
risks and uncertainties, which may cause Catalyst's actual results
in future periods to differ materially from forecasted results. A
number of factors, including (i) whether AGAMREE's
commercialization by Catalyst in the U.S. will prove to be
accretive to Catalyst, (ii) whether Catalyst and Santhera will
successfully develop additional indications for AGAMREE and obtain
the approvals required to commercialize the product in the licensed
territory for those additional indications, (iii) whether, if
AGAMREE is commercialized by Catalyst, the drug will be
successfully integrated into Catalyst's business activities, and
(iv) those factors described in Catalyst's Annual Report on Form
10-K for the 2022 fiscal year, Catalyst's Quarterly Report on Form
10-Q for the first quarter of 2023, and Catalyst's other filings
with the SEC, could adversely affect Catalyst. Copies of
Catalyst's filings with the SEC are available from
the SEC, may be found on Catalyst's website, or may be
obtained upon request from Catalyst. Catalyst does not undertake
any obligation to update the information contained herein, which
speaks only as of this date.
Source: Catalyst Pharmaceuticals, Inc.
Contact Information
Media ContactDavid Schull, Russo Partners(858)
717-2310david.schull@russopartnersllc.com
Investor ContactMary Coleman, Catalyst
Pharmaceuticals, Inc.(305) 420-3200mcoleman@catalystpharma.com
Catalyst Pharmaceuticals (NASDAQ:CPRX)
Gráfico Histórico do Ativo
De Mai 2024 até Jun 2024
Catalyst Pharmaceuticals (NASDAQ:CPRX)
Gráfico Histórico do Ativo
De Jun 2023 até Jun 2024