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Catalyst Pharmaceuticals to Present at MDA Clinical & Scientific Conference Details of Registry for Study of Long-Term Safety and Quality of Life in Duchenne Muscular Dystrophy Patients Treated with AGAMREE | ||
By: GlobeNewswire - 27 Feb 2024 | Back to overview list |
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CORAL GABLES, Fla., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult to treat diseases, today announced an upcoming poster about the establishment of a registry to study long-term safety and quality of life (QoL) in patients with Duchenne muscular dystrophy (DMD) who are treated with AGAMREE® (vamorolone) at the upcoming 2024 MDA Clinical & Scientific Conference taking place March 3-6 in Orlando. The poster will detail the establishment of a multicenter, observational, prospective, longitudinal registry at approximately 25 U.S. sites. Investigators will assess the progress of 250 male patients who are at least 2 years old at week 12 and then every 12 months for up to five years. The first interim analysis is expected in May 2025. Poster Information: Title: An Imminent Registry to Study Long-term Safety and Quality of Life in Patients with Duchenne Muscular Dystrophy treated with Vamorolone (AGAMREE®) "This registry will provide real-world data regarding long-term use of the novel corticosteroid, generating additional data to the current clinical results and underscoring the potential of AGAMREE® to reshape the DMD treatment paradigm with a focus on improving the quality of life for patients," said Gary Ingenito, MD, Ph.D., Chief Medical and Regulatory Officer of Catalyst. "We encourage physicians treating patients with DMD to consider enrolling eligible patients in this registry." Learn more about #MDAConference here: https://www.mdaconference.org/ About AGAMREE® (vamorolone) AGAMREE was granted U.S. FDA approval on October 26, 2023, and was granted Orphan Drug and Rare Pediatric Disease designation status for DMD in the U.S., 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. References: About Catalyst Pharmaceuticals With 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 Statements This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. These include statements regarding Catalyst's expectations, beliefs, plans or objectives regarding the intended use of net proceeds therefrom. 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 DMD patient will agree to enroll in the registry, and (ii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2022 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.
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