Catalyst Pharmaceuticals Teams Up with Patients and Advocacy Groups to Ring Nasdaq Opening Bell on Rare Disease Day 2022

Coral Gables, Florida, UNITED STATES


Ceremony Recognizes Importance of Biopharma Industry and Advocacy Group Collaboration to Address Needs for New Treatments in Orphan and Rare Disorders

Together, Will Ring the Nasdaq Opening Bell to Kick-Off Rare Disease Day on February 28, 2022

CORAL GABLES, Fla., Feb. 24, 2022 (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 diseases, today announced that the company is standing with the National Organization for Rare Disorders (NORD), Global Genes, Muscular Dystrophy Association, Myasthenia Gravis Foundation of America, Conquer MG, and Myasthenia Gravis Association, to ring the opening bell of the Nasdaq market on international Rare Disease Day, February 28, 2022. Organized by Catalyst, the ceremony is part of the ongoing efforts of the biopharma industry and patient advocacy groups to call attention to the need for collaboration to encourage research and development of orphan drugs and bring new treatments to market for rare diseases that affect small patient populations.

“Rare Disease Day and events such as this help individuals and families living with rare diseases know that they are not alone and that there is a community raising awareness on their behalf. NORD is grateful for this opportunity to champion health equity for the rare disease community and help shine a light on their unique experiences and challenges,” said Lisa Sarfaty, Vice President of Community Engagement for the National Organization for Rare Disorders.

“Rare Disease Day is an opportune moment to build awareness, consider our progress, and rally around what has yet to be done. Until the day rare disease becomes a global health priority, we must be relentlessly nimble, creative, resourceful, and collaborative in our efforts to improve the lives and care of the nearly one in 10 Americans and 400 million people worldwide living with rare conditions," added Craig Martin, CEO of Global Genes.

There are approximately 7,000 identified rare diseases affecting millions across the globe, including 30 million Americans. Only about 5% of the known rare diseases have approved treatments. The biopharmaceutical industry has made great progress, with more than 770 FDA-approved medicines since the enactment of the Orphan Drug Act in 1983. However, there is still much more work to be done. Partnering and collaborating with advocacy leaders and organizations will raise awareness and connect patients with appropriate providers.

“We are committed to making a meaningful impact on the lives of those living with rare diseases,” said Patrick J. McEnany, Chairman and CEO of Catalyst Pharmaceuticals. “Catalyst is working tirelessly with patients living with the rare condition Lambert-Eaton myasthenic syndrome (LEMS), helping them gain access to treatment. We thank our advocacy partners who are standing with us, echoing our relentless commitment to this cause. We are grateful to Nasdaq for the opening bell-ringing opportunity, allowing us to show our support for the rare disease community.”

The ceremony will begin at approximately 9:15 am ET and can be viewed on the Nasdaq live stream at: https://livestream.com/accounts/27896496/events/10011861. A replay of the ceremony will be available at a later date on the Company website at www.catalystpharma.com.

About Catalyst Pharmaceuticals

Catalyst Pharmaceuticals is a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare diseases. With exceptional patient focus, Catalyst is committed to developing a robust pipeline of cutting-edge, first- or best-in-class medicines for other rare diseases. Catalyst's New Drug Application for FIRDAPSE® (amifampridine) Tablets 10 mg for the treatment of adults with Lambert-Eaton myasthenic syndrome ("LEMS") was approved in 2018 by the U.S. Food & Drug Administration ("FDA"), and FIRDAPSE is commercially available in the United States as a treatment for adults with LEMS. 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.

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 those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2020 and Catalyst's 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.

About the National Organization for Rare Disorders (NORD)

The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 300 disease-specific member organizations, more than 17,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit https://rarediseases.org/.

About Global Genes

Global Genes is a 501(c)(3) non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf ⁠— helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease or are searching for a diagnosis, contact Global Genes (www.globalgenes.org) at 949-248-RARE or visit our Resource Hub (https://resource-hub.globalgenes.org/home/)

About the Muscular Dystrophy Association

For 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA's MOVR is the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports the largest network of multidisciplinary clinics providing best-in-class care at more than 150 of the nation's top medical institutions. Our Resource Center serves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. MDA Advocacy supports equal access for our community, and each year thousands of children and young adults learn vital life skills and gain independence at summer camp and through recreational programs, at no cost to families. For more information, visit https://www.mda.org/.

About the Myasthenia Gravis Foundation of America

The Myasthenia Gravis Foundation of America (MGFA) is the largest, leading patient advocacy organization solely dedicated to finding a cure and improved treatments for the rare neuromuscular disease myasthenia gravis (MG) by funding critical research discoveries and providing patient-centric programs, guidance, events, and webinars to connect, educate, and assist members of the global MG Community. You can visit MGFA at www.myasthenia.org.

About Myasthenia Gravis Association

The Myasthenia Gravis Association (MGA) is dedicated to supporting patients, families, friends, and communities impacted by myasthenia gravis. MGA works in those communities to increase public awareness through education and awareness events, 1-1 consultations, new patient packets, clinics, and support groups. For more information, visit www.mgakc.org

About Conquer MG

Conquer MG offers help and hope to myasthenia gravis patients through their caring staff, and support groups in Illinois and beyond. For more information, visit www.myastheniagravis.org.

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