- First evidenced-based medicine approved to treat LEMS
- Catalyst Pathways™ designed to facilitate access to Firdapse® and provide dedicated patient
support
- Launch early in the first quarter of 2019
- Catalyst to hold conference call on December 13, 2018 at 8:30 AM ET to discuss launch details
CORAL GABLES, Fla., Nov. 29, 2018 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq: CPRX),
a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating,
chronic neuromuscular and neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved
Firdapse® (amifampridine) 10 mg tablets for the treatment of adults with Lambert-Eaton Myasthenic Syndrome (LEMS). Firdapse is
expected to be commercially available early in the first quarter of 2019. For full prescribing information, please click here.
“The FDA’s approval of Firdapse is a potentially transformative milestone in the lives of patients in the U.S.
suffering with LEMS, as it now gives adult LEMS patients access to a new first-in-class-therapy,” said Patrick J. McEnany, Chairman
and Chief Executive Officer of Catalyst Pharmaceuticals. “The approval of Firdapse is a crowning achievement for our company and an
important step forward in our transition into a premier neurological rare disease company. We continue to prepare for what we hope
will be a successful launch based on productive discussions with payers, our efforts in patient identification and our development
of a field-based commercial infrastructure. Our Catalyst Pathways™ patient services program is designed to assist patients
and physicians with education, clinical diagnostic tools and assistance in navigating the reimbursement landscape.”
“I am pleased that the FDA has recognized the comprehensive clinical data supporting the benefits of Firdapse
for patients with LEMS,” said Perry B. Shieh, M.D., Ph.D., Department of Neurology at UCLA Medical Center. “It’s
important that patients living with this debilitating disease now have access to an FDA-approved treatment that has shown
clinically meaningful benefits.”
LEMS is a rare autoimmune disease that affects approximately 1 in 100,000 people in the United States. The most
common symptoms of LEMS are proximal muscle weakness and fatigue. Symptoms can be life threatening when the weakness involves
respiratory muscles. Approximately 50% of LEMS patients have an underlying malignancy, typically small cell lung cancer.
“At the National Organization for Rare Diseases (NORD), it’s a great day for patients when we learn of FDA
approval of a much-needed treatment for a rare disease. We’d like to congratulate the entire team at Catalyst for its commitment to
providing LEMS patients and their doctors with access to an FDA-approved medicine,” said Peter Saltonstall, President and CEO of
NORD. “With 95% of rare diseases still without an FDA-approved treatment, continued research and new products remain of vital
importance to the rare disease community.”
The submission of the Firdapse NDA came following positive results from two Phase 3 studies where patients
treated with Firdapse experienced rapid, significant and sustained improvements in muscle function, and reduced weakness and
fatigability compared to patients receiving placebo. These benefits were observed by both patients and physicians. Please see below
for Important Safety Information. Firdapse had previously received Orphan Drug designation and Breakthrough Therapy designation, as
well as Priority Review from the FDA. Firdapse is the first and only approved drug in Europe for treatment of LEMS.
“This FDA approval marks the arrival of a first-in-class therapy for a rare and devastating condition with
limited treatment options,” said Gary Ingenito, M.D., Ph.D., Chief Medical Officer and Head of Regulatory Affairs at Catalyst. “We
extend our deepest gratitude to the patients who participated in the Firdapse clinical trials and their families and caregivers who
supported them. We are also grateful for the tireless efforts of the investigators and study staff, without whom this important
milestone would not have been possible. We also look forward to continuing to work with the FDA to evaluate other potential
indications for Firdapse.”
Catalyst Pathways™
Catalyst Pathways™ is an optional, free, personalized program that offers patients and their families one-on-one
support throughout their treatment journey with a dedicated team of specialists to help them manage their unique challenges.
The support team for each enrolled patient at Catalyst Pathways includes a Care Coordinator, with extensive experience in health
care; an Insurance Navigator, to help them understand their insurance coverage and the prescription drug benefit process; a Patient
Assistance Liaison, to assist them in understanding their disease and their prescription, and a specialty pharmacy experienced in
supporting rare disease patients to provide direct-to-patient delivery of prescriptions. The Catalyst Pathways support team
also helps enrolled patients understand and access financial assistance options for qualifying patients, including a copay support
program designed to minimize the patient’s burden of out-of-pocket costs. For more information on Catalyst Pathways click here.
Conference Call
Catalyst will host a conference call and webcast to discuss its commercialization plan for Firdapse, including
the cost of therapy, on December 13, 2018 at 8:30 AM ET. Investors who wish to participate in the conference call may do so by
dialing (877) 407-8912 for domestic and Canadian callers or (201) 689-8059 for international callers. Those interested in listening
to the conference call live via the internet may do so by visiting the Investors page of the company's website at
www.catalystpharma.com and clicking on the webcast link on the Investors home page. A webcast replay will be available on the
Catalyst website following the call by visiting the Investor page of the company's website at https://www.catalystpharma.com.
About Lambert-Eaton Myasthenic Syndrome (LEMS)
LEMS, is a rare autoimmune disorder, most often characterized by fatigable limb muscle weakness. The disease is caused by
autoantibodies against voltage-gated calcium channels located in the nerve-muscle junction, resulting in improper nerve-muscle
communication, leading to progressive muscle weakness, when left untreated. In approximately 50% of cases, LEMS is associated
with an underlying malignancy, most commonly small cell lung cancer, and in some individuals, LEMS is the first symptom of such
malignancy. LEMS generally affects the extremities, especially the legs. In early course of the disease, muscles
closest to the trunk are affected, which are manifested as difficulties with climbing stairs or rising from a sitting position.
Physical exercise and high temperatures tend to worsen the symptoms. Other symptoms occasionally seen include weakness
of the muscles of the mouth, throat, and eyes. Individuals affected with LEMS also may have a disruption of the autonomic
nervous system, with symptoms of dry mouth, dry skin, constipation, blurred vision, impaired sweating, and/or inappropriate blood
pressure variations.
About Firdapse® (amifampridine)
Firdapse® (amifampridine) 10 mg tablets is an oral, nonspecific, voltage-dependent, potassium (K+) channel
blocker that causes depolarization of the presynaptic membrane and slows or inhibits repolarization. This action results in the
opening of slow voltage-dependent calcium (Ca2+) channels, allowing for a subsequent influx of Ca2+. In turn, it induces the
exocytosis of synaptic vesicles containing Acetylcholine (ACh) to release more ACh into the synaptic cleft, enhancing neuromuscular
transmission, and providing for improved muscle function. Firdapse is approved in the U.S. and the European Union for use by
patients with LEMS.
Important Safety Information
CONTRAINDICATIONS
FIRDAPSE is contraindicated in patients with:
- A history of seizures
- Hypersensitivity to amifampridine phosphate or another aminopyridine
WARNINGS AND PRECAUTIONS
Seizures: FIRDAPSE can cause seizures. Consider discontinuation or dose
reduction of FIRDAPSE in patients who have a seizure while on treatment. FIRDAPSE is contraindicated in patients with a history of
seizures.
Hypersensitivity: If a hypersensitivity reaction such as anaphylaxis occurs,
FIRDAPSE should be discontinued and appropriate therapy initiated.
ADVERSE REACTIONS
The most common (> 10%) adverse reactions are: paresthesia, upper respiratory tract infection, abdominal
pain, nausea, diarrhea, headache, elevated liver enzymes, back pain, hypertension, and muscle spasms.
To report SUSPECTED ADVERSE REACTIONS, contact Catalyst Pharmaceuticals at 1-844-347-3277
(1?844-FIRDAPSE) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
About Catalyst Pharmaceuticals
Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative
therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including LEMS, congenital myasthenic
syndromes (CMS), MuSK antibody positive myasthenia gravis (MuSK-MG), and spinal muscular atrophy (SMA) type 3. Amifampridine
phosphate has received Orphan Drug Designation from the United States FDA for CMS and myasthenia gravis. Firdapse (amifampridine)
10 mg tablets is the first and only approved drug in Europe for symptomatic treatment in adults with LEMS.
Catalyst is also developing a generic version of vigabatrin.
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 Catalyst will be successful in commercializing Firdapse (ii) whether, even if Catalyst
is successful in commercializing Firdapse, Catalyst will become profitable, (iii) whether Firdapse will ever be approved for the
treatment of CMS, MuSK-MG, SMA type 3, or any other disease, and (iv) those other factors described in Catalyst's Annual Report on
Form 10-K for the fiscal year 2017 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.
Investor Contact
Brian Korb
Solebury Trout
(646) 378-2923
bkorb@troutgroup.com
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Company Contact
Patrick J. McEnany
Catalyst Pharmaceuticals
Chief Executive Officer
(305) 420-3200
pmcenany@catalystpharma.com |
Media Contact
David Schull
Russo Partners
(212) 845-4271
david.schull@russopartnersllc.com |
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