Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a
commercial-stage biopharmaceutical company, today announced the
U.S. commercial launch of AGAMREE® (vamorolone) oral suspension 40
mg/mL for the treatment of Duchenne Muscular Dystrophy ("DMD") in
patients aged two years and older. Following the U.S. Food and Drug
Administration ("FDA") approval on October 26, 2023, AGAMREE is now
available by prescription and dispensed throughout the United
States through a specialty pharmacy network.
"Today, we proudly announced the U.S. commercial
availability of AGAMREE, an innovative alternative steroid
treatment for Duchenne Muscular Dystrophy. This significant
milestone offers hope for improved quality of life for individuals
living with this devastating rare disease, as current steroid
treatment options often involve a significant side effect burden,"
stated Richard J. Daly, CEO of Catalyst. "With the potential to
extend ambulation and mobility for patients, AGAMREE marks an
important therapeutic advancement with the prospect of reshaping
the treatment paradigm for this life-threatening condition. We are
firmly positioned to leverage our well-established commercial
capabilities for a successful U.S. launch and remain resolute in
our mission to help ensure that all patients have access to this
novel therapy. Our Catalyst Pathways, a personalized program, is
readily available to assist Duchenne Muscular Dystrophy patients
and their families with the one‐on‐one education and financial
support they need, thereby enhancing the access and affordability
of AGAMREE for every patient. We eagerly look forward to
collaborating with healthcare providers to facilitate patient
access, reinforcing our unwavering dedication to serving our
patient communities."
Catalyst Pathways® Patient Assistance
Program for AGAMREE® is a comprehensive patient support program
that includes a dedicated, personalized support team that assists
families throughout the AGAMREE treatment journey for eligible
patients. For more information, caregivers and healthcare
professionals can call 1-833-422-8259 or visit the Catalyst
Pathways® website at www.yourcatalystpathways.com.
Duchenne Muscular Dystrophy, 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 a corticosteroid treatment. Steroids are expected to
remain the backbone of therapy for DMD patients and dosed
concomitantly with other therapies.
The 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
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
improved behavior.
About AGAMREE® (vamorolone)
AGAMREE's unique mode of action is based on differential effects on
glucocorticoid and mineralocorticoid receptors and modifying
further downstream activity. As such, it is considered a novel
corticosteroid with dissociative properties in maintaining efficacy
that we hope has the potential to demonstrate comparable efficacy
to steroids, with the potential for a better-tolerated side effect
profile. This mechanism of action may allow AGAMREE 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, AGAMREE 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, psychiatric
disorders, vomiting, weight increases, 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 has been granted Orphan Drug
Exclusivity (ODE) for DMD and New Chemical Entity Exclusivity (NCE)
in the U.S., conferring seven years and 5 years of
exclusivity, respectively, from the date of approval. AGAMREE also
has granted pending patents that could provide protection until
2040. In Europe, it has received Promising Innovative Medicine
(PIM) status from the UK MHRA for DMD.
References:[1] Dang UJ et al. (2024) Neurology
024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.[2]
Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014.
doi:10.1001/jamaneurol.2022.2480. Link.[3] Liu X et al.
(2020). Proc Natl Acad Sci USA 117:24285-24293[4] Heier
CR et al (2019). Life Science Alliance DOI: 10.26508[5] Ward et
al., WMS 2022, FP.27 - Poster 71. Link.[6] Hasham et al., MDA 2022
Poster presentation. Link.[7] Applicable drug labeling: Summary of
Product Characteristics (SmPC). Link
About Catalyst
PharmaceuticalsWith exceptional patient focus, Catalyst is
committed to developing and commercializing innovative
first-in-class medicines that address rare and difficult-to-treat
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 by the FDA for
commercialization in the U.S. on October 26,
2023.
For more information about Catalyst
Pharmaceuticals, Inc., please visit the Company's website
at www.catalystpharma.com. For Full Prescribing and Safety
Information for FIRDAPSE®, please visit www.firdapse.com. For
Full Prescribing Information, including Boxed WARNING for FYCOMPA®,
please visit www.fycompa.com. For Full Prescribing Information
for AGAMREE®, please visit www.agamree.com.
Forward-Looking StatementsThis
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
its Licensor Santhera Pharmaceuticals, AG 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, as AGAMREE is
commercialized by Catalyst, the drug will be successfully
integrated into Catalyst's business activities, and (iv) those
factors described in Catalyst's those factors described in
Catalyst's Annual Report on Form 10-K for the fiscal year 2023 and
its other filings with the U.S. Securities and Exchange
Commission ("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.
Investor Relations Contact:
Mary Coleman, Catalyst Pharmaceuticals
(305) 420-3200
mcoleman@catalystpharma.com
Media Contact:
David Schull, Russo Partners
(858) 717-2310
david.schull@russopartnersllc.com
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