Catalyst Pharmaceuticals, Inc. (Catalyst) operates as a commercial-stage, patient-centric biopharmaceutical company.
The company focuses on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult to treat diseases. The company markets three drug products, FIRDAPSE (amifampridine), FYCOMPA (perampanel), and AGAMREE (vamorolone). The company is seeking to further expand its drug portfolio, with a focus on acquiring the rights to late-...
Catalyst Pharmaceuticals, Inc. (Catalyst) operates as a commercial-stage, patient-centric biopharmaceutical company.
The company focuses on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult to treat diseases. The company markets three drug products, FIRDAPSE (amifampridine), FYCOMPA (perampanel), and AGAMREE (vamorolone). The company is seeking to further expand its drug portfolio, with a focus on acquiring the rights to late-stage products to treat orphan, rare diseases across therapeutic areas. With an unwavering patient focus embedded in everything the company do, it is committed to providing innovative, best-in-class medications with the hope of making a meaningful impact on those affected by these conditions.
FIRDAPSE Product Overview
FIRDAPSE is Catalyst’s registered trade name in the U.S. for amifampridine phosphate tablets. FIRDAPSE contains the phosphate salt of amifampridine, hence the name ‘amifampridine phosphate.’ The company refers to its drug by its trade name in the U.S. (FIRDAPSE), by the INN and USAN (amifampridine), or by the specific salt in the company’s product (amifampridine phosphate), throughout this report.
Based on available information, the company estimates that there are at least 3,600 LEMS patients in the U.S., approximately half of which it diagnosed and identified and the remainder of which the company is undiagnosed or misdiagnosed. However, until awareness of the disease is increased, it is unlikely that the total number of LEMS patients in the U.S. can be determined with better certainty (as is typical of rare diseases), and the actual number of patients in the U.S. with LEMS may be higher or lower than its estimate.
License Agreements for FIRDAPSE
License Agreement with BioMarin
On October 26, 2012, the company licensed the exclusive North American rights to FIRDAPSE pursuant to a License Agreement (the License Agreement) between it and BioMarin Pharmaceutical Inc. (BioMarin).
On May 29, 2019, the company entered into an amendment to the License Agreement. Under the amendment, the company expanded its commercial territory for FIRDAPSE, which originally was consists of North America, to include Japan. Additionally, in December 2023 the company’s territory automatically expanded to include most of Asia, as well as Central and South America, upon the acceptance by the MHLW in Japan of an application to market its product in Japan. Under the amendment, the company will pay royalties on net sales in Japan of a similar percentage to the royalties that it is paying under the company’s original License Agreement for North America.
In January 2020, the company was advised that BioMarin had sold certain rights under the License Agreement to SERB SA.
License Agreement with Jacobus
In May 2019, the FDA approved an NDA for RUZUR, Jacobus Pharmaceuticals’ version of amifampridine (3,4-DAP), for the treatment of pediatric LEMS patients (ages 6 to under 17). In June 2019 the company filed suit against the FDA and several related parties challenging this approval and related drug labeling. Jacobus later intervened in the case. The company ultimately prevailed in its litigation in September 2021, when U.S. Court of Appeals for the 11th Circuit determined that the FDA’s approval of RUZURGI violated the company’s rights to Orphan Drug Exclusivity.
On July 11, 2022, the company settled its disputes with Jacobus. In connection with the settlement, the company licensed the rights to develop and commercialize RUZURGI in the U.S. and Mexico. Simultaneously, the company purchased, among other intellectual property rights, Jacobus’ U.S. patents related to RUZURGI, its NDAs in the U.S. for RUZURGI, and certain RUZURGI.
At the same time, the company received a license from Jacobus for use of its know-how related to the manufacture of RUZURGI. Further, the company settled its pending patent lawsuit against Jacobus, which has been dismissed without prejudice.
Clinical Trials Supporting the company’s NDA for FIRDAPSE for LEMS and Approval of its NDA
The company conducted two successful Phase 3 double-blind, placebo-controlled clinical trials evaluating FIRDAPSE for the treatment of LEMS. The results of the first trial published in 2016 in Muscle & Nerve (Muscle Nerve, 2016, 53(5):717-725). The results of the second trial were published in March 2019 in the Journal of Clinical Neuromuscular Disease (J. Clin Neuromusc Dis 2019; 20:111-119). In March 2018, the company submitted an NDA seeking approval of FIRDAPSE for the treatment of LEMS. The company’s NDA was accepted for filing in May 2018 and, on November 28, 2018, the FDA granted approval of FIRDAPSE for the treatment of LEMS in adult patients.
On September 29, 2022, the FDA approved the company’s NDA to expand the indicated age range for FIRDAPSE for the treatment of LEMS to include pediatric patients, six years of age and older. Further, on May 30, 2024, the FDA approved the company’s NDA increasing the indicated maximum daily dose of FIRDAPSE for adults and pediatric patients weighing more than 45 kg from 80 mg to 100 mg for the treatment of LEMS.
Required Post-Approval Studies
As part of the approval of the company’s NDA for FIRDAPSE for LEMS, the FDA required it to conduct two studies. The first was a clinical trial to evaluate the effect of hepatic impairment on the exposure of amifampridine after oral administration of FIRDAPSE relative to that in subjects with normal hepatic function. The second was to perform a second carcinogenicity study of amifampridine phosphate in mice. Both have been completed and submitted to the FDA which considers these commitments complete. For a third commitment, the company has also established a pregnancy surveillance program to collect and analyze information for a minimum of 10 years on pregnancy complications and birth outcomes related to FIRDAPSE. Finally, in connection with the approval of the company’s NDA for FIRDAPSE for the treatment of children ages six through seventeen with LEMS, it is conducting a pediatric safety study of juvenile toxicity in a rodent.
Compassionate Use Programs
The company continues to make FIRDAPSE available to a limited number of patients diagnosed with congenital myasthenic syndromes or Downbeat Nystagmus through investigator-sponsored compassionate use programs. Further, when the company acquired the U.S. rights to RUZURGI in July 2022, it agreed to continue to supply RUZURGI to these patients with neuromuscular conditions other than LEMS who are without access to an approved drug and were being treated with RUZURGI under investigator-sponsored INDs at the time of the company’s settlement with Jacobus. However, this program has recently been discontinued because the API for RUZURGI is no longer available and the company is therefore unable to manufacture additional product.
Sales, Marketing and Distribution
Marketing of FIRDAPSE
In January 2019, the company launched FIRDAPSE in the U.S. the company sells FIRDAPSE in the U.S. through a field-based force experienced in neurologic, central nervous system or rare disease products consisting at this time of approximately 40 field personnel, including sales (Regional Account Managers), thought leader liaisons and patient assistance and insurance navigation support (Patient Access Liaisons). The company also have a field-based force of nine medical science liaisons who are helping educate the medical community about scientific literature concerning LEMS and FIRDAPSE. Additionally, the company uses non-personal promotion to reach the 20,000 neurologists who are potential LEMS treaters and the 16,000 oncologists who might be treating a LEMS patient who also has small cell lung cancer. Further, the company continues to make available at no-cost a LEMS voltage gated calcium channel antibody diagnostic testing program for use by physicians who suspect that one of their patients may have LEMS and wish to reach a definitive diagnosis.
When the company launched AGAMREE, in March 2024, it utilized the FIRDAPSE commercial field-based force for AGAMREE as well. In early 2025, the company made a strategic decision to split its commercial field-based force into two units, one expressly focused on the marketing of FIRDAPSE, and one expressly focused on the marketing of AGAMREE.
The company is supporting the distribution of FIRDAPSE through Catalyst Pathways, its personalized treatment support program. Catalyst Pathways is a single source for personalized treatment support, education and guidance through the challenging dosing and titration regimen to an effective therapeutic dose. It also includes distributing the drug through a very small group of exclusive specialty pharmacies (primarily AnovoRx), which is consistent with the way that most pharmaceutical products for ultra-orphan diseases are distributed and dispensed to patients. By using specialty pharmacies in this way, the difficult task of navigating the health care system is far better for the patient needing treatment for their rare disease and the health care community in general.
In addition, Catalyst Pathways is the gateway for the company’s free bridge medication for patients transitioning from investigational product while they are waiting for a coverage determination or, later, for patients whose access is threatened by the bureaucratic complications arising from a change of insurer. The Catalyst Pathways program is also the access point for the company’s Patient Assistance Program, which provides longer-term free medication for those who are uninsured or functionally uninsured with respect to FIRDAPSE because they may be unable to obtain coverage from their payer despite having health insurance.
The company is continuing efforts on the challenging process to identify patients and their physicians who have diagnosed LEMS, but have not had access, awareness or understanding of this treatment for their rare disease. These patients often do not see their physician frequently, have many questions about changing treatment(s), and may not perceive the need to change to a new therapy. Further, the company have begun to focus its commercial efforts to locate misdiagnosed and undiagnosed LEMS patients and provide educational and sales activities to help improve the diagnosis, understanding of the treatment, and information on the prescribing process. The company plans to continue to support LEMS and rare disease patient organizational groups’ efforts to generate awareness and educate patients and physicians on the diagnosis of LEMS, the impact of the disease, and the support services and treatments available.
Canadian Market
The company’s market FIRDAPSE for the treatment of LEMS in Canada through a sublicensee, KYE. The company’s product was approved for commercialization in Canada in 2020.
Japanese Market
In May 2019, the company entered into an amendment to its license agreement for FIRDAPSE with BioMarin. Under the amendment, the company expanded its commercial territory for FIRDAPSE, which originally was consists of North America, to include Japan. Further, the company were granted orphan drug designation in Japan for FIRDAPSE for the symptomatic treatment of LEMS.
On June 28, 2021, the company entered into a sub-license agreement with DyDo Pharma, Inc., or DyDo, pursuant to which its sub-licensed to DyDo the Japanese rights for FIRDAPSE for the treatment of LEMS. In December 2021, the company announced that DyDo had initiated a Phase 3 registrational study in Japan to evaluate the efficacy and safety of FIRDAPSE for the treatment of LEMS. In December 2023, DyDo submitted a Japan NDA for FIRDAPSE to the PMDA. On September 24, 2024, DyDo informed the company’s that the MHLW of Japan has approved their New Drug Application for FIRDAPSE for the treatment of LEMS and DyDo began commercialization of FIRDAPSE in Japan on January 21, 2025.
Future Markets for FIRDAPSE
Under the amendment to the company’s license agreement that added Japan to its territory, the company’s territory in which it has the right to seek to commercialize FIRDAPSE has automatically expanded to include several countries in Asia-Pacific and South and Central America, and the company is working to expand its FIRDAPSE activities into some of these.
Intellectual property and regulatory exclusivity protections for FIRDAPSE
The bulk of the company’s patent rights related to FIRDAPSE are derived from its license agreement with BioMarin, which was transferred to SERB in 2020. In August 2020, the U.S. Patent and Trademark Office (USPTO) allowed Patent No. 10,793,893 (the ’893 patent) to the company’s licensor and thereby to it, and the patent issued on October 6, 2020. The patent is directed to the use of suitable doses of amifampridine to treat patients, regardless of the therapeutic indication, that are slow metabolizers of amifampridine. Any drug product containing amifampridine with a label that states the patented dosing regimens and doses in the Dosing and Administration section prior to May 26, 2034, the expiration date of the patent, could possibly infringe this patent. Generic drug product labels would necessarily have to do this, and the company intends to take all appropriate actions to protect its intellectual property.
In April 2021, the USPTO also allowed a further application in the family of licensed intellectual property, and this second patent, Patent No. 11,060,128 (the ’128 patent), issued on July 13, 2021. The patent is directed to the use of suitable doses of amifampridine to treat patients suffering with LEMS that are slow metabolizers of amifampridine. Any drug product containing amifampridine with a label for the treatment of LEMS, that states the patented dosing regimens and doses in the Dosing and Administration section of a product label, including generic drug product labels, could possibly infringe this patent prior to this patent’s expiration date.
Additional patents claiming priority from the patents noted above issued in March 2022 as Patent Nos. 11,268,128, 11,274,332, and 11,274,331, and extended the company’s patent coverage to include fast metabolizers of amifampridine. These patents are listed in the Orange Book for FIRDAPSE.
As part of the company’s transaction with Jacobus Pharmaceuticals, it also acquired two patents. One of these patents, 10,626,088 issued by the USPTO on April 21, 2020, was suitable for listing in the Orange Book and has been listed in further support of FIRDAPSE.
On December 19, 2023, and January 16, 2024, the USPTO issued Patent Nos. 11,845,977 and 11,873,525, respectively. These new patents cover methods of treating LEMS with FIRDAPSE under fasting and fed conditions of dosing. The company is also pursuing additional patent applications for FIRDAPSE to further protect its drug product.
Until FIRDAPSE was approved in November 2018, no drug product containing amifampridine for any indication had been approved by the FDA such that the company received five-year ‘new chemical entity’ exclusivity from the FDA. Further, when FIRDAPSE was approved for the treatment of LEMS patients, the company’s received seven-year orphan drug exclusivity (ODE) for its product for the treatment of LEMS, precluding a generic filer from receiving final FDA approval until the ODE exclusivity period has expired. Because the company has Orange Book listed patents for FIRDAPSE, potential generic filers were permitted to submit ANDA filings to the FDA starting on the ‘NCE-1’ date (November 28, 2022).
FYCOMPA Product Overview
The FDA approved FYCOMPA in October 2012 as an adjunctive agent for the treatment of focal onset seizures with or without secondary generalization in patients with epilepsy at least 4 years of age. In June 2015, the agency approved a second indication for primary generalized tonic-clonic seizures in patients with epilepsy who are at least 12 years of age.
FYCOMPA is a novel non-competitive selective antagonist at the postsynaptic ionotropic alpha-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) glutamate receptor. In the nervous system, glutamate is known to be a major excitatory neurotransmitter, but the exact antiepileptic mechanism of perampanel in humans is unknown. Studies suggest that AMPA receptor antagonism can lead to reduced overstimulation and anticonvulsant effects, as well as inhibiting seizure generation and spread. In addition, AMPA receptor antagonists may prevent neuronal death.
At the time of its approval, the FDA included specific significant warnings for FYCOMPA in the prescribing information that it required to be included prominently in all communications about the product. Such warnings are known as ‘black box’ warnings because they are traditionally surrounded by a black box to emphasize their significance. For FYCOMPA, the warning addresses rare but serious behavioral changes that occur in some patients using FYCOMPA, including aggression (up to and including homicidal behavior), hostility, anger, distrust and other extreme behavioral changes; visual and auditory hallucinations; and difficulty with memory. In addition, FYCOMPA was classified as a Class III controlled substance prior to its approval due to evidence of prolonged use creating a physical dependence in some patients and the possibility of abuse.
Epilepsy
Epilepsy is a long-term (chronic) disease that causes repeated seizures due to abnormal electrical signals produced by damaged brain cells. A burst of uncontrolled electrical activity within brain cells causes a seizure. Epilepsy is generally diagnosed after an individual suffers two seizures within a 24-hour period. Generally, cells in the brain send messages to and receive messages from all areas of the body. These messages are transmitted via a continuous electrical impulse that travels from cell to cell. Epilepsy disrupts this rhythmic electrical impulse pattern. Instead, there are bursts of electrical energy — like an unpredictable lightning storm — between cells in one or more areas of brain. This electrical disruption causes changes in awareness (including loss of consciousness), sensations, emotions and muscle movements. In the U.S., about 3.4 million people have epilepsy. Of this number, approximately 3 million are adults and 470,000 are children. There are 150,000 new cases of epilepsy in the U.S. each year. Worldwide, about 65 million people have epilepsy.
Access to FYCOMPA
The company is supporting patients using FYCOMPA through an Instant Savings Card Program. Through the program, eligible commercially insured patients could pay as little as $5 for their FYCOMPA co-pay (with a maximum savings of $2,500 per year). The FYCOMPA instant savings card program is not available to patients enrolled in state or federal healthcare programs, including Medicare, Medigap, VA, DoD, or TRICARE.
Clinical Trials Supporting FYCOMPA
Primary Generalized Tonic-Clonic Seizures
The efficacy of FYCOMPA as adjunctive therapy in patients 12 years of age and older with idiopathic generalized epilepsy experiencing primary generalized tonic-clonic seizures was established in one multicenter, randomized, double-blind, placebo-controlled study (Study 4), conducted at 78 sites in 16 countries.
Intellectual Property Protections for FYCOMPA
Patent protection for FYCOMPA is primarily from two patents listed in the Orange Book. The first, U.S. patent no. 6,949,571 (the '571 patent) will expire no earlier than May 23, 2025, inclusive of patent term extension.
Expiration of Patent Protection for FYCOMPA
The company’s executing its strategy in preparation for the patent expiry of the solid dose of FYCOMPA in the face of generic competition.
AGAMREE
AGAMREE is a structurally unique steroidal anti-inflammatory drug to treat children and adults living with DMD. In clinical studies, AGAMREE showed evidence of inhibition of pro-inflammatory NF-kB (nuclear factor kappa light chain enhancer of activated B cells) pathways, which are, a family of highly conserved transcription factors that regulate many important cellular behaviors, in particular, inflammatory responses and cellular growth. This inhibition is achieved through high-affinity binding to the glucocorticoid receptor, high-affinity antagonism for the mineralocorticoid receptor, and membrane stabilization properties. It is hoped that AGAMREE will demonstrate similar efficacy to traditional corticosteroids with reduced negative downstream impacts and side effects.
The FDA approved AGAMREE for the treatment of DMD in patients aged 2 and older on October 26, 2023.
Clinical Trials Supporting AGAMREE
A randomized, double-blind, placebo and prednisone-controlled trial of vamorolone was carried out in patients with DMD. The trial met the primary (time to stand velocity after 24 weeks for vamorolone, 6 mg/kg per day vs placebo) and first 4 sequential secondary motor function endpoints. Study participants receiving vamorolone, 2 mg/kg per day, and vamorolone, 6 mg/kg per day, showed improvements in multiple functional endpoints over the 24-week treatment period as compared to placebo. The statistical thresholds for the primary outcome and first 4 secondary outcomes for vamorolone treatment were met, and vamorolone demonstrated efficacy across both dose ranges. The differences in time to stand from supine velocity (TTSTAND) were clinically meaningful. The differences in 6-minute walk test (6MWT) were also clinically meaningful.
The trial validated previous open-label findings where a more normal growth trajectory was observed over 18-month and 30-month periods in vamorolone treated boys with DMD. Furthermore, bone turnover markers supported the improved profile of vamorolone on bone health.
Access to AGAMREE
The company’s supporting AGAMREE through its Catalyst Pathways Program, which includes a dedicated, personalized support team that assists families through the AGAMREE treatment journey, from answering questions to coordinating financial assistance programs for eligible patients.
Acquisition of AGAMREE
On June 19, 2023, the company entered into a License and Collaboration Agreement (License Agreement) and an Investment Agreement (Investment Agreement) with Santhera. Under the License Agreement, the company contracted to obtain an exclusive North America license, manufacturing and supply agreement for Santhera’s investigational product candidate, AGAMREE (vamorolone), a novel corticosteroid for the treatment of DMD. Under the Investment Agreement, the company agreed to make a strategic investment into Santhera.
FIRDAPSE
The company has entered into agreements with a supplier of the active pharmaceutical ingredient (API) contained in FIRDAPSE for future requirements and it has contracted with third party contract manufacturers who are manufacturing FIRDAPSE tablets for the company.
FYCOMPA
Under the company’s Supply Agreement with Eisai, Eisai has agreed to manufacture and supply to it finished bulk FYCOMPA tablets for the company for a seven-year period that will run through at least the end of 2029. In addition, Eisai has assigned to the company’s third-party manufacturing contracts related to final packaging of bulk FYCOMPA tablets and the manufacture of the oral solution formulation.
FIRDAPSE
Before the approval of FIRDAPSE, LEMS was generally treated with unapproved drugs and therapies, including steroids, azathioprine, other immunosuppressants and intravenous immunoglobulin, which work by suppressing the immune system, and pyridostigmine.
Finally, the company is aware that amifampridine has been available in the past from compounding pharmacies and may remain available, even though FIRDAPSE has been approved and is commercially marketed in the U.S.
FYCOMPA
FYCOMPA is the first and only AED that targets a specific receptor in the brain called ‘AMPA’. The receptor plays a role in allowing seizures to occur. Seizures have historically been treated with benzodiazepines, such as clonazepam (Klonopin) and lorazepam (Ativan), GABA inhibitors, such as gabapentin (Neurontin), phenobarbital (Luminal), and pregabalin (Lyrica), and sodium channel blockers, such as carbamazepine (Tegretol) and lacosamide (Vimpat). Additionally, surgical options, such as deep brain stimulation have been used in patients who have failed polypharmacy. Finally, there are multiple compounds that have been recently approved or are in late-stage development for focal epilepsy.
After the expiration of patent protection for FYCOMPA, the company expects that it will also face competition from generic manufacturers.
AGAMREE
On February 9, 2017, the FDA approved Emflaza (deflazacort), a corticosteroid marketed by PTC Therapeutics for the treatment of DMD in patients 5 years old and older.
In addition, there are many companies which have announced plans for pre-clinical candidates and clinical development for the treatment of DMD, including gene transfer and/or gene editing therapies. One of those gene therapy treatments, Sarepta’s Elevidys has been approved for the treatment of DMD and, in June 2024, received an expanded label for the treatment of DMD patients over age five, even though the drug failed the primary endpoint in a Phase 3 clinical trial in that population.
Regulatory Matters
Schedule III drugs are subject to certain DEA import volume limits and state regulations relating to manufacturing, storage, distribution and physician prescription procedures, including limitations on prescription refills. In addition, the third parties who perform the company’s clinical and commercial manufacturing, distribution, and dispensing for FYCOMPA are required to maintain necessary DEA registrations and state licenses.
In addition to regulation by the FDA and certain state regulatory agencies, the company is also subject to a variety of foreign regulations governing clinical trials and the marketing of other products.
Research and Development
The company’s research and development expenses were approximately $12.6 million for the year ended December 31, 2024.
History
The company was founded in 2002. It was incorporated in Delaware in 2006. The company was formerly known as Catalyst Pharmaceutical Partners, Inc. and changed its name to Catalyst Pharmaceuticals, Inc. in 2015.
