Tuberous Sclerosis

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

Company to Feature First and Only Ready-to-Use Liquid Vigabatrin at Booth #846 MAPLE GROVE, Minn., April 4, 2025 /PRNewswire/ -- Upsher-Smith Laboratories, LLC (Upsher-Smith) today announced its participation at the 2025 American Academy of Neurology (AAN) Annual Meeting, from April 5-9, 2025 in San Diego, California. Representatives from Upsher-Smith will be available at Booth #846 to share information with clinicians about its expanding portfolio of products for rare diseases, including the first and only ready-to-use liquid vigabatrin, VIGAFYDE™ (vigabatrin) Oral Solution. VIGAFYDE™ is indicated for infantile spasms (IS) as monotherapy in infants 1 month to 2 years of age for whom the potential benefits outweigh the risk of vision loss. "Since the successful launch of VIGADRONE® (vigabatrin) for Oral Solution to the specialty pharmacy channel in 2018, we have broadened our offerings to include VIGADRONE® (vigabatrin) Tablets, TORPENZ™ (everolimus) Tablets, and VIGAFYDE™ ready-to-use solution," said Jim Maahs, Head of Commercial, Upsher-Smith. "As part of our commitment to supporting the rare disease community, Upsher-Smith is dedicated to not only maintaining, but enhancing the support and resources available to patients, caregivers, and healthcare practitioners. We encourage attendees to stop by our booth during this year's AAN meeting to learn more." About Upsher-Smith's Rare Disease Portfolio Upsher-Smith's rare disease portfolio continues to grow with the addition of high-quality medications that match or exceed the level of service provided by innovator brands. The Company offers three vigabatrin formulations including VIGAFYDE™ Oral Solution, VIGADRONE® Powder for Oral Solution and VIGADRONE® Tablets. VIGAFYDE™, the only ready-to-use vigabatrin oral solution, is the first new drug application (NDA) approved for infantile spasms in 15 years. As the only premixed, ready-to-use vigabatrin formulation, VIGAFYDE™ represents a welcome expansion of vigabatrin treatment options, offering patients and caregivers a simpler dose preparation process which may improve dosing accuracy. Upsher-Smith representatives will be available throughout the conference at exhibit booth #846 to engage with clinicians about their products including VIGAFYDE™, and VIGADRONE® and the Promise of Support™ Program, which has been providing comprehensive support to patients prescribed VIGADRONE® since 2018. Representatives will also be available to discuss Upsher-Smith's TORPENZ™ Tablets, an oral mTOR inhibitor used to treat certain manifestations of Tuberous Sclerosis Complex (TSC). TORPENZ™, is the only everolimus backed by the Upsher-Smith Promise of Support™ Program to deliver reliable and consistent support to patients, caregivers, and healthcare providers in the TSC community. TORPENZ™ is indicated for the treatment of adult patients with renal angiomyolipoma and TSC, not requiring immediate surgery and is also indicated in adult and pediatric patients aged 1 year and older with TSC for the treatment of subependymal giant cell astrocytoma (SEGA) that requires therapeutic intervention but cannot be curatively resected. About Upsher-Smith's Promise of Support™ Program Central to Upsher-Smith's commitment to rare disease communities is its Promise of Support™ Program, ensuring comprehensive assistance for patients, caregivers, and healthcare providers throughout the treatment journey. For nearly a decade, the program has offered resources for uninterrupted medication supply, prior authorization support, patient education materials, collaboration with a leading specialty pharmacy partner, and streamlined prescribing and enrollment procedures. It also includes copay assistance for eligible patients to help lessen the financial burden on patients and their families. To learn more about Upsher-Smith, its products, and the Promise of Support™ Program visit: . Vigabatrin REMS All vigabatrin products are governed by a Risk Evaluation and Mitigation Strategy (REMS) mandated by the FDA. The Vigabatrin REMS ensures that patients and healthcare providers make informed risk-benefit decisions about its use. Healthcare providers must be enrolled in the Vigabatrin REMS to prescribe these products, and patients must be enrolled to receive them. WHAT IS VIGAFYDE? VIGAFYDE™ (vigabatrin) Oral Solution is a prescription medicine used to treat: Infantile Spasms (IS) in babies 1 month to 2 years of age, if you and your baby's healthcare provider decide the possible benefits of taking VIGAFYDE are more important than the possible risk of vision loss. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT VIGAFYDE™ (vigabatrin)? Because VIGAFYDE might cause permanent vision loss, it is available to healthcare providers and patients only under a special program called the Vigabatrin Risk Evaluation and Mitigation Strategy (REMS) Program. Your baby's healthcare provider will explain the details of this Program to you. VIGAFYDE can damage the vision of anyone who takes it. Some people can have severe loss, particularly to their ability to see to the side when looking straight ahead (peripheral vision). With severe vision loss, your baby may only be able to see things straight in front of them (sometimes called "tunnel vision"). Your baby may also have blurry vision. If this happens, it will not get better. Tell your healthcare provider right away if your baby might not be seeing as well as before starting VIGAFYDE or is acting differently than normal. These changes can mean that vision damage has occurred. Regular vision testing is recommended. It is recommended that your healthcare provider test your baby's vision before or within 4 weeks after starting VIGAFYDE, and at least every 3 months during treatment until VIGAFYDE is stopped. It is also recommended that vision be tested about 3 to 6 months after VIGAFYDE is stopped. It is difficult to test vision in babies, but to the extent possible, all patients should have their vision tested. Your baby's healthcare provider will determine if testing can be done. Regular vision testing is important because damage can happen before any changes are noticed. Vision tests cannot prevent the vision damage that can happen with VIGAFYDE, but they do allow VIGAFYDE to be stopped if vision has gotten worse, which usually will lessen further damage. Even these regular vision tests may not show vision damage before it is serious and permanent. Parents, caregivers, and healthcare providers may not recognize the symptoms, or find vision loss in babies, until it is severe. If you do not have these vision tests regularly, your baby's healthcare provider may stop prescribing VIGAFYDE. Your baby may not be able to complete vision testing. If vision testing cannot be done, the healthcare provider may continue prescribing VIGAFYDE but will not be able to watch for any vision loss. Magnetic resonance imaging (MRI) changes in patients with infantile spasms (IS). Brain pictures taken by MRI show changes in some patients after they are given VIGAFYDE. It is not known if these changes are harmful. A type of swelling in the brain called intramyelinic edema (IME) has been seen in postmortem examination of infants treated with vigabatrin. Risk of suicidal thoughts or actions. VIGAFYDE is approved for use in patients 1 month to 2 years of age [see WHAT IS VIGAFYDE?]. VIGAFYDE is not approved for use in adolescents or adults. Like other antiepileptic drugs, vigabatrin may cause suicidal thoughts and actions in some adolescents and adults (about 1 in 500 people). A healthcare provider should be called right away if a patient taking vigabatrin experiences any symptoms, especially sudden changes in mood, behaviors, thoughts, or feelings, and especially if they are new, worse, or worrying. Do not stop VIGAFYDE without first talking to a healthcare provider. Stopping VIGAFYDE suddenly can cause seizures that will not stop. VIGAFYDE is more concentrated than vigabatrin solutions prepared from powder. The volume of VIGAFYDE solution prescribed by your baby's healthcare provider may be less than the volume prescribed for other vigabatrin solutions prepared from powder. Always double check the volume (mL) that you are to give your baby when you get a new prescription filled. VIGAFYDE can cause serious side effects such as low red blood cell counts (anemia), sleepiness and tiredness, and weight gain. VIGAFYDE may make certain types of seizures worse. Tell your healthcare provider right away if seizures get worse. Before starting VIGAFYDE, tell your baby's doctor about all of their medical conditions including any allergic reaction to vigabatrin, vision problems, kidney problems, or low red blood cell counts (anemia). Tell your doctor about all the medicines you baby is receiving. The most common side effects of VIGAFYDE in babies include: sleepiness (sleepy babies may have a harder time suckling and feeding or may be irritable), swelling in the bronchial tubes (bronchitis), ear infection and irritability. Tell your healthcare provider if your baby has any side effect that bothers them or that does not go away. This is the most important information to know about VIGAFYDE, but not all of the safety information. For more information, ask your healthcare provider or pharmacist, or please see the VIGAFYDE Medication Guide, full Prescribing Information including Boxed Warning for risk of permanent vision loss, and Instructions for Use. You can also visit VIGAFYDE.com, upsher-smith.com or call 1-888-650-3789. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-332-1088. WHAT IS VIGADRONE? VIGADRONE® (vigabatrin) is a prescription medicine used to treat: Infantile Spasms (IS) in patients 1 month to 2 years of age, if you and your healthcare provider decide the possible benefits of taking VIGADRONE are more important than the possible risk of vision loss. Refractory Complex Partial Seizures (CPS) in adults and children 2 years and older with refractory complex partial seizures (CPS) along with other treatments if: The CPS do not respond well enough to several other treatments, and You and your healthcare provider decide the possible benefit of taking VIGADRONE is more important than the risk of vision loss. VIGADRONE should not be the first medicine used to treat CPS. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT VIGADRONE® (vigabatrin)? Because VIGADRONE might cause permanent vision loss, it is available to healthcare providers and patients only under a special program called the Vigabatrin Risk Evaluation and Mitigation Strategy (REMS) Program. Your healthcare provider will explain the details of this Program to you. VIGADRONE can damage the vision of anyone who takes it. Some people can have severe loss, particularly to their ability to see to the side when looking straight ahead (peripheral vision). With severe vision loss, you may only be able to see things straight in front of you (sometimes called "tunnel vision"). You may also have blurry vision. If this happens, it will not get better. Tell your healthcare provider right away if you (or your child): might not be seeing as well as before starting VIGADRONE; start to trip, bump into things, or are more clumsy than usual; are surprised by people or things coming in front of you that seem to come out of nowhere; or if your baby is acting differently than normal. These changes can mean that vision damage has occurred. Regular vision testing is recommended. It is recommended that your healthcare provider test your (or your child's) vision before or within 4 weeks after starting VIGADRONE, and at least every 3 months during treatment until VIGADRONE is stopped. It is also recommended that vision be tested about 3 to 6 months after VIGADRONE is stopped. It is difficult to test vision in babies, but to the extent possible, all patients should have their vision tested. Your healthcare provider will determine if testing can be done. Regular vision testing is important because damage can happen before any changes are noticed. Vision tests cannot prevent the vision damage that can happen with VIGADRONE, but they do allow VIGADRONE to be stopped if vision has gotten worse, which usually will lessen further damage. Even these regular vision tests may not show vision damage before it is serious and permanent. Parents, caregivers, and healthcare providers may not recognize the symptoms, or find vision loss in patients, until it is severe. If you do not have these vision tests regularly, your healthcare provider may stop prescribing VIGADRONE for you (or your child). Some people are not able to complete vision testing. If vision testing cannot be done, your healthcare provider may continue prescribing VIGADRONE, but will not be able to watch for any vision loss. Magnetic resonance imaging (MRI) changes in patients with infantile spasms (IS). Brain pictures taken by MRI show changes in some patients after they are given VIGADRONE. It is not known if these changes are harmful. A type of swelling in the brain called intramyelinic edema (IME) has been seen in autopsy examination of patients treated with vigabatrin. Risk of suicidal thoughts or actions. Like other antiepileptic drugs, VIGADRONE may cause suicidal thoughts and actions in some people (about 1 in 500 people). Call a healthcare provider right away if you (or your child) have any symptoms, especially sudden changes in mood, behaviors, thoughts or feelings, and especially if they are new, worse, or worry you. Do not stop VIGADRONE without first talking to a healthcare provider. Stopping VIGADRONE suddenly can cause seizures that will not stop. VIGADRONE can cause serious side effects such as low red blood cell counts (anemia), sleepiness and tiredness, nerve problems, weight gain, and swelling. Because VIGADRONE causes sleepiness and tiredness, do not drive, operate machinery, or perform any hazardous task, unless it is decided that these things can be done safely. VIGADRONE may make certain types of seizures worse. Tell your healthcare provider right away if seizures get worse. Before starting VIGADRONE, tell your doctor about all of your (or your child's) medical conditions including depression, mood problems, suicidal thoughts or behavior, any allergic reaction to VIGADRONE, vision problems, kidney problems, low red blood cell counts (anemia), and any nervous or mental illnesses. Tell your doctor about all the medicines you (or your child) take. If you are breastfeeding or plan to breastfeed, VIGADRONE can pass into breast milk and may harm your baby. Breastfeeding is not recommended. If you are pregnant or plan to become pregnant, VIGADRONE can cause harm to your unborn baby. You and your healthcare provider will have to decide if you should take VIGADRONE while you are pregnant. The most common side effects of VIGADRONE in adults include: blurred vision, sleepiness, dizziness, problems walking or feeling uncoordinated, shaking (tremor) and tiredness. The most common side effect of VIGADRONE in children 3 to 16 years of age is weight gain. Also expect side effects like those seen in adults. The most common side effects of VIGADRONE in babies include: sleepiness (sleepy babies may have a harder time suckling and feeding or may be irritable), swelling in the bronchial tubes (bronchitis), ear infection and irritability. Tell your healthcare provider if you or your child have any side effect that bothers you or that does not go away. This is the most important information to know about VIGADRONE, but it is not all the safety information. For more information, ask your healthcare provider or pharmacist, or please see the VIGADRONE Medication Guide, full Prescribing Information including Boxed Warning for risk of permanent vision loss, and Instructions for Use. You can also visit VIGADRONE.com, upsher-smith.com or call 1-888-650-3789. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-332-1088. WHAT ARE TORPENZ (everolimus) TABLETS? TORPENZ is a prescription medicine used to treat the following types of benign (non-cancerous) tumors that are seen with a genetic condition called tuberous sclerosis complex (TSC): Brain tumors called subependymal giant cell astrocytoma (SEGA), when the tumor cannot be removed completely by surgery, in adults and children 1 year and older. Kidney tumors called angiomyolipoma, when the tumor does not require surgery right away, in adults. For additional information, including safety information, for non-TSC indications, see the full Prescribing Information, including Patient Information. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT TORPENZ™ ( everolimus) TABLETS in Tuberous Sclerosis Complex? Do not take TORPENZ if you have had an allergic reaction to everolimus. Talk to your healthcare provider before taking TORPENZ if you are allergic to sirolimus or temsirolimus. Ask your provider if you do not know. TORPENZ can cause serious side effects, including: Lung or breathing problems. In some people, these may be severe and can be life threatening. Tell your healthcare provider right away if you have new or worsening cough, shortness of breath, chest pain, difficulty breathing or wheezing. Higher likelihood of infection such as pneumonia, or a bacterial, fungal, or viral infection. Viral infections may include active hepatitis B in people who have had hepatitis B in the past (reactivation). In some adults and children, these infections may be severe and can be life threatening. You may need to be treated as soon as possible. Tell your healthcare provider right away if you have a temperature of 100.5˚F or above, chills, or do not feel well. Symptoms of infection may include: Severe allergic reactions. Get medical help right away if you have signs of an allergic reaction, including rash, itching, hives, flushing, trouble breathing or swallowing, chest pain or dizziness. Possible increased risk for a type of allergic reaction called angioedema in people who take an Angiotensin-Converting Enzyme (ACE) inhibitor with TORPENZ. Talk with your healthcare provider before taking TORPENZ if you are not sure if you take an ACE inhibitor. Get medical help right away if you have trouble breathing or develop swelling of your tongue, mouth, or throat during treatment with TORPENZ. Mouth ulcers and sores. Mouth ulcers and sores are common during treatment with TORPENZ but can also be severe. When you start TORPENZ, your healthcare provider may tell you to also start a prescription mouthwash to reduce the likelihood of getting mouth ulcers or sores and to reduce their severity. Follow your provider's instructions on how to use this mouthwash. If you develop pain, discomfort, or open sores in your mouth, tell your provider. Kidney failure. In some people, this may be severe and can be life threatening. Your healthcare provider should check your kidney function before and during treatment. Wound healing problems. Wounds may not heal properly during TORPENZ treatment. Tell your healthcare provider if you plan to have any surgery before starting or during treatment. You should stop TORPENZ at least 1 week before planned surgery. Your provider should tell you when to start taking TORPENZ again after surgery. Increased blood sugar and fat (cholesterol and triglyceride) levels in the blood. Your healthcare provider should check your fasting blood sugar, cholesterol, and triglyceride levels in the blood before you start and during treatment. Decreased blood cell counts. TORPENZ can cause you to have decreased red blood cells, white blood cells and platelets. Your healthcare provider should check your blood cell counts before you start and during treatment. Worsening side effects from radiation treatment that can sometimes be severe. Tell your healthcare provider if you have had or are planning to receive radiation therapy. Before taking TORPENZ, tell your healthcare provider about all of your medical conditions, including if you: Have or have had kidney or liver problems, have diabetes or high blood sugar, have high blood cholesterol, have any infections, or previously had hepatitis B. Are scheduled to receive any vaccinations. You should not receive a "live vaccine" or be around people who have recently received a "live vaccine" during your treatment with TORPENZ. If you are not sure about the type of immunization or vaccine, ask your provider. For children, work with your provider to complete the recommended childhood series of vaccines before your child starts TORPENZ. Are pregnant, can become pregnant, or have a partner who can become pregnant. TORPENZ can cause harm to your unborn baby. For females who are able to become pregnant, your provider will give you a pregnancy test before you start TORPENZ. Use effective birth control during treatment and for 8 weeks after your last dose of TORPENZ. Males with a female partner should use effective birth control during treatment and for 4 weeks after your last dose of TORPENZ. Are breastfeeding or plan to breastfeed. Do not breastfeed during treatment and for 2 weeks after your last dose of TORPENZ. Are planning to have or have had a recent surgery. You should stop taking TORPENZ at least 1 week before planned surgery. Have received or are planning to receive radiation therapy. Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking TORPENZ with some medicines can cause serious side effects. Keep a list of medications you take, and show it to your healthcare provider and pharmacist when you get a new medicine. Especially tell your provider if you take: St. John's Wort (Hypericum perforatum) Medicine for fungal or bacterial infections, tuberculosis, seizures, HIV-AIDS, heart conditions or high blood pressure, including ACE inhibitors Medicines that weaken your immune system (your body's ability to fight infections and other problems) Ask your provider or pharmacist if you are not sure if your medicine is one of those taken for the conditions listed above. If you are taking any medicines for the conditions listed above, your provider might need to prescribe a different medicine or your dose of TORPENZ may need to be changed. Tell your provider before you start any new medicine. You should not drink grapefruit juice or eat grapefruit during your treatment with TORPENZ. It may make the amount of TORPENZ in your blood increase to a harmful level. The most common side effect of TORPENZ in people who have SEGA or renal angiomyolipoma include respiratory tract infection. Other side effects that may occur with TORPENZ: Absence of menstrual periods (menstruation). Tell your healthcare provider if this happens. TORPENZ may affect fertility and may affect your ability to become pregnant if you are female or your ability to father a child if you are male. Talk to your provider if this is a concern. Tell your healthcare provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of TORPENZ. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You can also visit TORPENZ.com, upsher-smith.com or call 1-888-650-3789. You are encouraged to report negative side effects of prescription drugs to FDA. Visit or call 1-800-332-1088. About Upsher-Smith Upsher-Smith Laboratories, LLC, now a member of Bora Group, is a trusted U.S. pharmaceutical company that strives to improve the health and lives of patients through an unwavering commitment to high-quality products and sustainable growth. We bring generics and brands to a wide array of customers, always backed by our attentive level of service, strong industry relationships, and dedication to uninterrupted supply. For more information, visit . About Bora Founded in 2007, Bora Pharmaceutical Co., Ltd. ("Bora" or "the Company", 6472.TW) now is the largest pharmaceutical manufacturer in Taiwan with well-connected global distribution to supply more than 100 countries around the world. Bora is dedicated to becoming a global leader in pharmaceutical manufacturing by offering its clients the best quality, efficiency and reliability. For more information, visit . VIGADRONE, TORPENZ and Promise of Support are trademarks of Upsher-Smith Laboratories, LLC. VIGAFYDE is a trademark of Pyros Pharmaceuticals, Inc. SOURCE Upsher-Smith Laboratories, LLC WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Simufilam did not show a significant reduction in co-primary endpoints of cognitive or functional decline versus placebo in patients with mild-to-moderate Alzheimer’s disease Simufilam continued to demonstrate an overall favorable safety profile Cassava’s Alzheimer’s disease development program with simufilam will be completely discontinued by the end of Q2 2025 March 25, 2025 -- Cassava Sciences, Inc. (NASDAQ: SAVA, “Cassava”, the “Company”), a clinical-stage biotechnology company focused on developing a novel, investigational treatment for central nervous system disorders, including Alzheimer’s disease (AD) dementia and tuberous sclerosis complex (TSC)-related epilepsy, today shared topline results from the Phase 3 REFOCUS-ALZ study of simufilam in mild-to-moderate AD. Topline data indicate that REFOCUS-ALZ did not meet each of the prespecified co-primary, secondary and exploratory biomarker endpoints. The co-primary endpoints were the change in cognition and function from baseline to the end of the double-blind treatment period at week 76, assessed by the ADAS-COG12 and ADCS-ADL scales, comparing simufilam to placebo. REFOCUS-ALZ enrolled 1,125 patients and was discontinued on November 25, 2024, following the report that a prior 52-week Phase 3 study, RETHINK-ALZ, did not meet its co-primary endpoints. A large portion of subjects enrolled in REFOCUS-ALZ completed their final study visit prior to the termination of the trial. Simufilam continued to demonstrate an overall favorable safety profile. “We are disappointed that the results of REFOCUS-ALZ and RETHINK-ALZ showed no treatment benefit for patients with mild-to-moderate Alzheimer’s disease. These results were unambiguous. Working with patients, their families and their caregivers has brought a special dignity to our Phase 3 Alzheimer’s disease clinical trial program and to each of us at Cassava. We are deeply grateful for the dedication and committed efforts of study investigators and site teams, who enabled us to conduct these trials with integrity and scientific rigor and whose efforts provided a clear data read out,” said Rick Barry, President and Chief Executive Officer of Cassava. “Cassava will discontinue all efforts to develop simufilam for Alzheimer’s disease and we expect to phase out the program by the end of Q2 2025,” continued Mr. Barry. “We remain dedicated to our mission of developing novel medicines for central nervous system disorders. While we have initiated preclinical studies to evaluate simufilam’s potential as a treatment for TSC-related epilepsy, we maintain ongoing strategic expense management efforts.” Eric Schoen, Chief Financial Officer of Cassava commented, “We remain focused on the interests of Cassava shareholders and are committed to enhancing shareholder value. Cassava is well-capitalized with approximately $128.6 million in cash and cash equivalents as of December 31, 2024.” REFOCUS-ALZ (NCT05026177) is a Phase 3 trial designed as a multi-center, double-blinded, placebo-controlled, randomized parallel group study to evaluate the safety and efficacy of two doses of simufilam compared to a placebo in a study involving over 75 clinical trial sites in the U.S., Canada, Puerto Rico and South Korea. The clinical trial sites that conducted REFOCUS-ALZ were completely distinct from the clinical trial sites that conducted RETHINK-ALZ. REFOCUS-ALZ randomized approximately 1,125 people utilizing the same eligibility criteria as RETHINK-ALZ. Subjects were randomized 1:1:1 to receive simufilam, dosed in 50 mg or 100 mg tablets, or a matched placebo, dosed orally twice daily (BID) for 76 weeks. On November 25, 2024, the Company announced plans to discontinue the REFOCUS-ALZ study and its intention to report topline data from that trial, including the complete 52-week dataset and a large portion of 76-week data. The prespecified co-primary endpoints for this study included the change in cognition and function from baseline to the end of the double-blind treatment period at week 76, assessed by the ADAS-COG12 and ADCS-ADL scales, comparing each dose of simufilam to placebo. Secondary endpoints included several well validated measures of neuropsychiatric symptoms and caregiver burden. Safety was evaluated by adverse event monitoring, as well as standard laboratory and ECG assessments. The study also included an evaluation of changes in plasma and cerebrospinal fluid biomarkers from baseline to week 76, including P-tau217 (phosphorylated tau at threonine 217), GFAP (glial fibrillary acidic protein) and NFL (neurofilament light chain), as well as an evaluation of various brain volumes using MRI (magnetic resonance imaging) and amyloid and tau deposition using PET (positron emission tomography) scans from baseline to week 76. Simufilam is a proprietary, investigational oral small molecule that targets the filamin A protein. Cassava Sciences, Inc. (NASDAQ: SAVA), a clinical-stage biotechnology company focused on developing novel, investigational treatments for central nervous system disorders, including Alzheimer’s disease and tuberous sclerosis complex (TSC)-related epilepsy. Simufilam is a proprietary, investigational oral small molecule that targets the filamin A protein. The Company is based in Austin, Texas. 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