CDKL5 Deficiency Disorder

EPIDIOLEX has significant market potential due to the growing demand for alternative therapies and its effectiveness in treating rare seizure disorders like Lennox-Gastaut syndrome and Dravet syndrome. As awareness around the benefits of CBD in treating neurological conditions increases, EPIDIOLEX stands to benefit from expanding indications and market reach. LAS VEGAS, May 1, 2025 /PRNewswire/ -- DelveInsight's ' EPIDIOLEX Market Size, Forecast, and Market Insight Report' highlights the details around EPIDIOLEX, the first FDA-approved prescription cannabidiol (CBD) to treat seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, or tuberous sclerosis complex (TSC) in patients 1 year of age or older. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of EPIDIOLEX. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Jazz Pharmaceuticals' EPIDIOLEX (cannabidiol) Overview EPIDIOLEX (formerly known as GWP42003-P and marketed as EPIDYOLEX in Europe) is the first plant-based, prescription cannabis-derived oral medication developed by GW Pharmaceuticals. It represents a new category of antiepileptic drugs with a unique mechanism of action. The active compound, cannabidiol (CBD), is a naturally occurring cannabinoid found in the Cannabis sativa L. plant. While the exact way EPIDIOLEX works to reduce seizures in humans remains unclear, it is distinct from traditional anticonvulsants. CBD does not act through CB1 receptors or by blocking voltage-gated sodium channels, which are typical pathways for other antiepileptic drugs. Instead, CBD is thought to work by influencing multiple internal pathways, potentially producing a combined anticonvulsant effect. These include enhancing neuronal inhibition (through synaptic and extrasynaptic GABA channels), affecting intracellular calcium levels (via TRPV channels, VDAC, and GPR55), and reducing inflammation, possibly via adenosine modulation. Importantly, CBD does not directly bind to or activate the CB1 or CB2 receptors at levels relevant to its seizure-reducing effects. Ongoing research is examining other possible mechanisms such as interactions with adenosine, glycine, GABAergic systems, and serotonin receptors. EPIDIOLEX has received regulatory approval in numerous countries, including the U.S. (FDA), Europe (European Commission), Great Britain (MHRA), Australia (Therapeutic Goods Administration), Switzerland (Swissmedic), Israel (Ministry of Health), and New Zealand (Medicines and Medical Devices Safety Authority). It is formulated as an oral solution containing highly purified CBD. In the U.S., it is approved to treat seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC) in patients aged one year and older. In the European Union, under the name Epidyolex, it is approved as an add-on treatment with clobazam for LGS and DS in patients two years and older, and for TSC-associated seizures as well. Both the FDA and EMA have granted Orphan Drug Designation (ODD) for EPIDIOLEX/EPIDYOLEX for these conditions. EPIDIOLEX/EPIDYOLEX net product sales rose by 15% to USD 972.4 million in 2024 and by 14% to USD 275.0 million in the fourth quarter of 2024, compared to the corresponding periods in 2023. Learn more about EPIDIOLEX/EPIDYOLEX projected market size for DEE @ EPIDIOLEX Market Potential Developmental and epileptic encephalopathies (DEE) encompass a group of severe neurological conditions marked by developmental delays and persistent epilepsy. These disorders typically present early in life and are linked to significant cognitive deficits. According to DelveInsight's analysis, there were approximately 287K diagnosed prevalent cases of DEE across the 7MM in 2024. This number is projected to increase due to the rise in neurological disorders, including epilepsy, as well as environmental and maternal health factors contributing to a higher DEE prevalence throughout the forecast period (2025−2034). The US FDA has approved a range of medications for conditions like LGS, Dravet Syndrome, and CDKL5 Deficiency Disorder, including various drug classes such as sodium channel modulators, GABA receptor modulators, calcium channel blockers, receptor blockers, and others. Some of the available anti-epileptic drugs (AEDs) include FINTEPLA, EPIDIOLEX, TOPAMAX, BANZEL, LAMICTAL, FELBATOL, ONFI, KLONOPIN, DIACOMIT, SABRIL, and more. In 2024, the total market size for DEE in the 7MM was around USD 2.3 billion, and this figure is expected to grow by 2034, driven by wider adoption of approved therapies due to label expansions and the introduction of emerging treatments. Discover more about the DEE market in detail @ Developmental and Epileptic Encephalopathy Market Report Emerging Competitors of EPIDIOLEX Some of the drugs in the pipeline include EPX-100 (Harmony Biosciences), Bexicaserin (Lundbeck), Zorevunersen (Stoke Therapeutics/Biogen), Relutrigine (Praxis Precision Medicines), and others. In August 2024, the FDA removed a partial clinical hold as the company advances toward a Phase III registrational study of zorevunersen (STK-001) in children and adolescents with Dravet syndrome. In July 2024, Longboard Pharmaceuticals announced that the FDA had granted Breakthrough Therapy designation for its investigational drug bexicaserin for the treatment of seizures associated with DEEs for patients 2 years of age or older. To know more about the number of competing drugs in development, visit @ EPIDIOLEX Alternatives Key Milestones of EPIDIOLEX In August 2024, Jazz Pharmaceuticals reported top-line results from a Phase 3 open-label, single-arm study conducted in Japan, assessing the safety and effectiveness of its cannabidiol oral solution (marketed globally as EPIDIOLEX/EPIDYOLEX) as an add-on therapy for seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC). While the trial did not achieve its primary efficacy goal—defined as a pre-specified percentage reduction in seizure frequency during the up-to-16-week treatment period compared to baseline in Japanese pediatric patients—numerical improvements were noted in both the primary and several secondary measures. The study did not identify any new safety concerns. In May 2021, Jazz Pharmaceuticals acquired EPIDIOLEX as part of the acquisition of GW Pharmaceuticals plc, or GW, which expanded its growing neuroscience business with a global, high-growth childhood-onset epilepsy franchise. In April 2021, EPIDYOLEX was approved for adjunctive therapy of seizures associated with TSC for patients 2 years of age and older in the EU. In July 2020, EPIDYOLEX was approved for the treatment of seizures associated with TSC in patients one year of age and older. FDA also approved the expansion of all existing indications, LGS and DS, to patients one year of age and older. In September 2019, the European Commission approved EPIDYOLEX for use as adjunctive therapy of seizures associated with LGS or DS, in conjunction with clobazam, for patients two years of age and older. In June 2018, EPIDIOLEX was approved in the US for the treatment of seizures associated with two rare and severe forms of epilepsy, LGS and DS, in patients two years of age and older. EPIDIOLEX Launch Status EPIDIOLEX was launched on November 1, 2018, in the US market after FDA approval for the treatment of seizures associated with LGS or Dravet syndrome in patients two years of age and older. EPIDYOLEX was launched in 2019 in Germany and the UK market for use as adjunctive therapy for seizures associated with LGS or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older (GW Pharmaceuticals, 2019b). In 2021, EPIDYOLEX was launched in Spain. In the first quarter of 2022, the company launched EPIDYOLEX for LGS, Dravet syndrome, and TSC in Ireland, for TSC in Scotland and Wales. In the third quarter of 2022, the company launched EPIDYOLEX for TSC in Italy and Switzerland. In the fourth quarter of 2022, the company successfully completed the pricing and reimbursement process and commercial launch of EPIDYOLEX in France. Discover how EPIDIOLEX/EPIDYOLEX is shaping the DEE treatment landscape @ EPIDIOLEX CBD EPIDIOLEX Market Dynamics EPIDIOLEX, the first FDA-approved cannabidiol (CBD)-based medication for the treatment of rare forms of epilepsy, has had a significant impact on the pharmaceutical market since its approval in 2018. The drug is primarily used to treat seizures associated with Dravet syndrome and Lennox-Gastaut syndrome, two rare and severe forms of epilepsy. The market dynamic for EPIDIOLEX is shaped by several factors, including increasing awareness of its therapeutic potential, the expanding medical cannabis industry, and the growing demand for alternative treatments to traditional anti-epileptic drugs. One of the main drivers of the EPIDIOLEX market is the rising acceptance of CBD-based therapies. As medical cannabis becomes more mainstream and research into its medicinal properties continues to grow, patients and healthcare providers are increasingly turning to EPIDIOLEX as a treatment option. The growing body of clinical evidence supporting its effectiveness and safety has reinforced this trend. Additionally, as the stigma around cannabis-derived medications continues to fade, it opens up more opportunities for EPIDIOLEX in both pediatric and adult patient populations. However, the market for EPIDIOLEX is not without its challenges. High treatment costs and insurance coverage issues can limit accessibility for some patients, especially in markets outside the United States. Despite its clinical efficacy, the price point for EPIDIOLEX remains a point of contention, as it may not be affordable for all families, especially when compared to other conventional anti-epileptic drugs. Furthermore, competition from other cannabinoid-based therapies, as well as new developments in the broader epilepsy drug market, could impact the drug's market share over time. Looking ahead, the market dynamics for EPIDIOLEX will likely continue to evolve as the global regulatory landscape changes and new clinical trials further establish its benefits in other neurological conditions. With increasing research into CBD's potential applications, there may be opportunities for EPIDIOLEX to expand its indications, providing a solid foundation for its continued growth. However, market growth will also depend on the ability to address pricing concerns, improve insurance coverage, and navigate potential competitive pressures from both traditional and emerging therapies. Dive deeper to get more insight into EPIDIOLEX/EPIDYOLEX's strengths & weaknesses relative to competitors @ EPIDIOLEX Market Drug Report Table of Contents Related Reports Developmental and Epileptic Encephalopathies Market Developmental and Epileptic Encephalopathies Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DEE companies, including vid Therapeutics, Stoke Therapeutics, Biogen, Harmony Biosciences, Lundbeck (Longboard Pharmaceuticals), Praxis Precision Medicines, SK Life Science, Cerecin Neurosciences, Encoded Therapeutics, Praxis Precision Medicines, among others. Developmental and Epileptic Encephalopathies Pipeline Developmental and Epileptic Encephalopathies Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key DEE companies, including Denovo Biopharma, Cantex Pharmaceuticals, CNS Pharmaceuticals, CANbridge Pharmaceuticals, Vaximm, Inovio Pharmaceuticals, Mustang Bio, Bullfrog AI Holdings, Cantex, Chimeric Therapeutics, Philogen, Boehringer Ingelheim, Photonamic GmbH, Berg Pharma, Beyond Bio, Genenta Science, Polaris Pharmaceuticals, Telix Pharmaceuticals, Shanghai Simnova Biotechnology, NEONC Technologies, among others. Dravet Syndrome Market Dravet Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Dravet syndrome companies including Biocodex, GW Pharmaceuticals, Zogenix, Ovid Therapeutics, PTC Therapeutics, among others. Lennox-Gastaut Syndrome Market Lennox-Gastaut Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Lennox-Gastaut syndrome companies, including GlaxoSmithKline, Meda Pharmaceuticals, Roche, Lundbeck, Greenwich Biosciences, Janssen Pharmaceuticals, Eisai, Zogenix, Takeda, Ovid Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Mr. Jafar is a distinguished international business leader and co-founder of the Loulou Foundation OXFORD, UK and CLEVELAND, Ohio, US, 29 April 2025 – The Oxford-Harrington Rare Disease Centre (‘OHC’), a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio, aimed at driving cutting-edge rare disease breakthroughs, announces the appointment of Mr. Majid Jafar to its Advisory Council. The Advisory Council, led by former UK Prime Minister David Cameron, supports the OHC’s mission to deliver new treatments for the nearly half a billion people affected by rare diseases worldwide. Council members are leaders of relevant industries and sectors, from different regions of the world who bring expertise, resources and networks towards the initial goal of developing 40 new drugs for rare diseases in the next 10 years. Mr. Jafar is a distinguished business leader serving as Vice-Chairman of the Crescent Group and CEO of Crescent Petroleum, based in the United Arab Emirates. He is a prominent, global rare disease advocate and philanthropist with a strong commitment to driving positive social change in healthcare and education, with extensive networks and interests in business, investment and philanthropy in the US, Europe and the Middle East. Among his roles, Mr. Jafar is the co-founder, with his wife Lynn, of the Loulou Foundation, a private non-profit foundation dedicated to advancing science and treatments for CDKL5 Deficiency Disorder (CDD), a rare neurogenetic disorder that affects their eldest daughter, Alia. The Loulou Foundation has funded important research projects at leading universities and institutions in the US, Europe and Asia, with a total of over 60 projects at 45 different institutions. Mr. Jafar also sits on various non-profit boards including the Board of Fellows of Harvard Medical School, where he co-chairs the Discovery Council, and is a member of the Academy of the University of Pennsylvania and the Global Precision Medicine Council of the World Economic Forum, as well as co-chairing the campaign for Cambridge Children’s Hospital in the UK. David Cameron, Chair of the OHC’s Advisory Council, said: “We are delighted to welcome Majid to the OHC Advisory Council. His extensive experience within the business and investment communities and in policy engagement - alongside his philanthropic efforts in education and healthcare - make him an invaluable addition to our team. Furthermore, as a parent of a child with a rare disease, Majid has a deep empathy with families in similar situations as they seek a diagnosis and treatment. We look forward to his contribution to the Council and to his support of the OHC as it continues its groundbreaking work in rare disease research and development of new treatments for patients worldwide.” Majid Jafar commented: “I am honoured to join the Oxford-Harrington Rare Disease Centre Advisory Council. Supporting the development of innovative treatments for rare diseases is a cause close to my heart with my family’s own experience deeply shaping my commitment to delivering urgently needed treatments to rare disease patients. I look forward to helping advance the OHC’s mission by fostering key partnerships in new regions and sourcing philanthropic investment that can accelerate progress for the many patients in need.” Matthew Wood, Director and Chief Scientific Officer of the OHC, added: “Majid brings a unique perspective to our Advisory Council, with his strong leadership background and dedication to advancing healthcare. His commitment to social impact, coupled with his ability to expand the OHC’s reach into new regions, will be instrumental as we work towards delivering rare disease drugs in the coming years. We are thrilled to have him on board and look forward to his contributions to our mission.” Majid Jafar joins Lord Cameron (Chair of the OHC’s Advisory Council and Former UK Prime Minister), Professor Sir John Bell (President, Ellison Institute of Technology Oxford, and former Regius Professor of Medicine at Oxford University), Baroness Nicola Blackwood (Chair of Genomics England and of Oxford University Innovation), John F. Crowley (President and CEO of the Biotechnology Innovation Association (BIO)), Ronald G. Harrington (a renowned entrepreneur and philanthropist, and co-founder of Harrington Discovery Institute), and Jonathan S. Stamler, MD (President and co-founder of Harrington Discovery Institute and Distinguished University Professor of Medicine), on the OHC Advisory Council. *** Notes to Editors About Majid JafarMajid Jafar serves as the Vice-Chairman of the Crescent Group and CEO of Crescent Petroleum, the core oil & gas company that founded the group. He is also the Board Managing Director of Dana Gas (PJSC), a leading publicly listed natural gas company in the Middle East. Prior to this, he gained international oil & gas experience at Shell Exploration & Production and Shell Gas & Power. Beyond his professional roles, Mr. Jafar serves on several influential non-profit boards, including the Queen Rania Foundation, Kalimat Foundation, the Arab Forum for Environment and Development (AFED), and the Iraq Energy Institute. He is also a member of the Board of Fellows at Harvard Medical School, the Panel of Senior Advisers at Chatham House, and the International Advisory Boards of The Prince's Trust International and The Atlantic Council. He was named a Young Global Leader by the World Economic Forum.Mr. Jafar holds a degree in Engineering (Fluid Mechanics and Thermodynamics) from Cambridge University, an MA in International Studies and Diplomacy from SOAS, and an MBA from Harvard Business School. About Rare DiseasesMore than 400 million people worldwide are living with a rare disease, and approximately 50 percent are children. There are about 7,000 known rare diseases, with new diseases being discovered every day. A rare disease affects one in 10 Americans, or 10 percent of the US population. In the Middle East, the prevalence of rare diseases is rising, and many countries in the region are now prioritising research and treatment options. The majority of all rare diseases are genetic in origin, which means they are present throughout a person’s life. Only five percent of rare diseases have a treatment approved by the US Food and Drug Administration (FDA) and similar estimates have been made for treatments approved by the European Medicine Agency (EMA). Therefore, someone with a rare disease today faces a lifelong, often life-threatening, condition with little hope for a cure, or even an effective treatment option. About Oxford-Harrington Rare Disease CentreThe Oxford-Harrington Rare Disease Centre (OHC) is a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, in Cleveland, Ohio, US. The OHC combines world-leading strengths in research and therapeutics development from across these organizations to set the science and innovation agenda and drive cutting-edge rare disease breakthroughs to address the unmet need in rare diseases across the globe to deliver major clinical impact for patients. For more information, visit: OxfordHarrington.org | LinkedIn | X About the University of OxfordThe University of Oxford is rated the best in the world for medicine and life sciences, and it is home to the UK’s top-ranked medical school. It has one of the largest clinical trial portfolios in the UK and great expertise in taking discoveries from the lab into the clinic. Partnerships with the local NHS Trusts enable patients to benefit from close links between medical research and healthcare delivery. For more information, visit: www.ox.ac.uk About Harrington Discovery InstituteHarrington Discovery Institute at University Hospitals in Cleveland, Ohio aims to advance academic discoveries into medicines to address unmet need. It was created in 2012 with a $50 million founding gift from the Harrington family and has supported more than 200 scientists throughout the US, UK. and Canada. Harrington Discovery Institute uses an innovation model that surrounds scientists with drug development and business expertise and employs both philanthropic funding and catalytic investment capital to advance projects into the clinic and through commercialization. For more information, visit: HarringtonDiscovery.org| LinkedIn | X Media contacts: UK/ EU – MEDiSTRAVA Sylvie Berrebi / Mark SwallowOHC@medistrava.com US – For University HospitalsAnsley Kelmansley.kelm@UHhospitals.org Attachment Majid Jafar _ Headshot

Stockholm, Sweden – February 11, 2025 – Immedica Pharma AB (“Immedica”), a leading global rare disease company, announced today the completion of the acquisition of Marinus Pharmaceuticals, Inc. (“Marinus”) (Nasdaq: MRNS) through its indirect wholly owned subsidiary Matador Subsidiary, Inc., a Delaware corporation (“Purchaser”). Following the completion of Immedica’s successful tender offer to purchase all outstanding shares of common stock of Marinus for USD 0.55 per share, net to the seller thereof in cash (the “Offer Price”), without interest and subject to any applicable withholding taxes, Immedica acquired all remaining shares of common stock of Marinus through a merger pursuant to Section 251(h) of the General Corporation Law of the State of Delaware. As a result of the transaction, Marinus has become an indirect wholly owned subsidiary of Immedica, and the common stock of Marinus will cease to be traded on the Nasdaq Global Market. At the effective time of the merger, and subject to any perfected appraisal rights, all of the remaining shares of common stock of Marinus not purchased in the tender offer were converted into the right to receive the Offer Price without interest and subject to any applicable withholding taxes. This press release contains forward-looking statements by Immedica and the acquisition of Marinus by Immedica (the “Transaction”) that involve risks and uncertainties and reflects Immedica’s judgment as of the date of this press release. These forward-looking statements generally are identified by words such as “believe,” “can,” “could,” “seek,” “project,” “expect,” “anticipate,” “estimate,” “intend,” “strategy,” “future,” “opportunity,” “plan,” “may,” “might,” “should,” “will,” “would,” “will be,” “will continue,” “will likely result” and similar expressions. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties, many of which are outside of Immedica’s control. Forward-looking statements are based on Immedica’s current expectations and beliefs, and Immedica cannot give assurances that its expectations or beliefs will be achieved. These forward-looking statements are not a guarantee of future performance and are subject to a number of known and unknown risks, uncertainties and other factors that could cause actual results or events to differ, possibly materially, from the expectations or estimates reflected in such forward-looking statements, including, among others: litigation relating to the Transaction; the ability of Immedica to retain key personnel from Marinus; competitive responses to the Transaction; the financial condition, results of operations and business of Immedica; the risk that Immedica may be unable to achieve the anticipated benefits of the Transaction; other business effects, including the effects of industry, economic or political conditions outside of the companies’ control; Immedica’s ability to achieve the growth prospects and synergies expected from the transaction, as well as delays, challenges and expenses associated with integrating Marinus with its existing businesses; and other risks described in Immedica’s prior press releases. Forward-looking statements speak only as of the date they are made. Immedica assumes no obligation and does not intend to update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise, except as required by law. Immedica does not give any assurance that it will achieve its expectations. About Marinus Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. Marinus’ product, ZTALMY® (ganaxolone) oral suspension CV, is an FDA-approved prescription medication introduced in the United States in 2022. For more information, please visit www.marinuspharma.com and follow us on Facebook, LinkedIn and X. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within genetic & metabolic diseases, hematology & oncology and specialty care. Immedica was founded in 2018 and employs today around 130 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo. For more information visit www.immedica.com.