An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed the MEX-DM-302 Study (ATLAS Study)

This is an open-label extension study intended to evaluate the long-term safety and efficacy of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2) who have completed the parent study MEX-DM-302.

Start Date27 Feb 2025

Sponsor / Collaborator

Lupin Ltd

[+1]

NCT06523400 / Not yet recruitingPhase 3

A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients With Myotonic Dystrophy Type 1 and Type 2 (Phase 3)

A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES study)

Start Date27 Sep 2024

Sponsor / Collaborator

Lupin Ltd

[+1]

CTRI/2024/07/071462 / RecruitingNot Applicable

An Open Label Balanced Randomized Single Dose One Treatment Fasting Vs Fed Two Sequence Fasting And Fed Two Period Two Way Crossover Oral Pharmacokinetics Study Comparing Mexiletine Hydrochloride Capsule 200 Mg Manufactured By Celogen Generics Pvt Ltd On Behalf Of Dawa Limited In Healthy Adult Human Male Subjects Under Fasting and Fed Conditions - Nil

Start Date03 Aug 2024

Sponsor / Collaborator

DAWA Ltd.

100 Clinical Results associated with Mexiletine Hydrochloride

100 Translational Medicine associated with Mexiletine Hydrochloride

100 Patents (Medical) associated with Mexiletine Hydrochloride

1,873

Literatures (Medical) associated with Mexiletine Hydrochloride

01 Nov 2024·PFLUGERS ARCHIV-EUROPEAN JOURNAL OF PHYSIOLOGY

BK channels promote action potential repolarization in skeletal muscle but contribute little to myotonia

Article

Author: Blake, Brianna ; Rich, Mark M ; Dupont, Chris ; Voss, Andrew A

Abstract:

Patients with myotonia congenita suffer from slowed relaxation of muscle (myotonia), due to hyperexcitability caused by loss-of-function mutations in the ClC-1 chloride channel. A recent study suggested that block of large-conductance voltage- and Ca2+- activated K+ channels (BK) may be effective as therapy. The mechanism underlying efficacy was suggested to be lessening of the depolarizing effect of build-up of K+ in t-tubules of muscle during repetitive firing. BK channels are widely expressed in the nervous system and have been shown to play a central role in regulation of excitability, but their contribution to muscle excitability has not been determined. We performed intracellular recordings as well as force measurements in both wild type and BK−/− mouse extensor digitorum longus muscles. Action potential width was increased in BK−/− muscle due to slowing of repolarization, consistent with the possibility K+ build-up in t-tubules is lessened by block of BK channels in myotonic muscle. However, there was no difference in the severity of myotonia triggered by block of muscle Cl− channels with 9-anthracenecarboxylic acid (9AC) in wild type and BK−/− muscle fibers. Further study revealed no difference in the interspike membrane potential during repetitive firing suggesting there was no reduction in K+ build-up in t-tubules of BK−/− muscle. Force recordings following block of muscle Cl− channels demonstrated little reduction in myotonia in BK−/− muscle. In contrast, the current standard of care, mexiletine, significantly reduced myotonia. Our data suggest BK channels regulate muscle excitability, but are not an attractive target for therapy of myotonia.

15 Oct 2024·Journal of the American Heart Association

Defining Cardiomyocyte Repolarization Response to Pharmacotherapy in Long‐QT Syndrome Type 3

Article

Author: O'Brien, Stephen T. ; Ge, Ning ; Ward, Deirdre ; Galvin, Joseph ; Shen, Sanbing ; O'Brien, Timothy ; Li, Rui ; Xia, Wenxin ; McGorrian, Catherine ; McInerney, Veronica ; Prendiville, Terence W. ; Liu, Min

Background:

Long‐QT syndrome is a primary cardiac ion channelopathy predisposing a patient to ventricular arrhythmia through delayed repolarization on the resting ECG. We aimed to establish a patient‐specific, human induced pluripotent stem cell (hiPSC)‐derived cardiomyocytes model of long‐QT syndrome type 3 (LQT3) using clustered regularly interspaced palindromic repeats (CRISPR/Cas9), for disease modeling and drug challenge.

Methods and Results:

HiPSCs were generated from a patient with LQT3 harboring an SCN5A pathogenic variant (c.1231G>A; p.Val411Met), and an unrelated healthy control. The same SCN5A pathogenic variant was engineered into the background healthy control hiPSCs via CRISPR/Cas9 gene editing to generate a second disease model of LQT3 for comparison with an isogenic control. All 3 hiPSC lines were differentiated into cardiomyocytes. Both the patient‐derived LQT3 ( SCN5A+/− ) and genetically engineered LQT3 ( SCN5A+/− ) hiPSC‐derived cardiomyocytes showed significantly prolonged cardiomyocyte repolarization compared with the healthy control. Mexiletine, a cardiac voltage‐gated sodium channel (Na V 1.5) blocker, shortened repolarization in both patient‐derived LQT3 and genetically engineered LQT3 hiPSC‐derived cardiomyocytes, but had no effect in the control. Notably, calcium channel blockers nifedipine and verapamil showed a dose‐dependent shortening of repolarization, rescuing the phenotype. Additionally, therapeutic drugs known to prolong the corrected QT in humans (ondansetron, clarithromycin, and sotalol) demonstrated this effect in vitro, but the LQT3 clones were not more disproportionately affected compared with the control.

Conclusions:

We demonstrated that patient‐derived and genetically engineered LQT3 hiPSC‐derived cardiomyocytes faithfully recapitulate pathologic characteristics of LQT3. The clinical significance of such an in vitro model is in the exploration of novel therapeutic strategies, stratifying drug adverse reaction risk and potentially facilitating a more targeted, patient‐specific approach in high‐risk patients with LQT3.

01 Oct 2024·LANCET NEUROLOGY

Mexiletine versus lamotrigine in non-dystrophic myotonias: a randomised, double-blind, head-to-head, crossover, non-inferiority, phase 3 trial

Article

Author: Skorupinska, Iwona ; McDermott, Michael P ; Vivekanandam, Vinojini ; Hanna, Michael G ; Matthews, Emma ; Jayaseelan, Dipa L ; Barohn, Richard J

BACKGROUND:

Non-dystrophic myotonias are skeletal muscle channelopathies caused by ion channel dysfunction. Symptom onset is frequently in the first decade of life, causing disability in a young cohort. Although there is no cure, symptomatic treatments exist. Previous trials provide evidence of the efficacy of mexiletine. More recently, lamotrigine has been shown to be effective. Both treatments have different profiles, including pharmacokinetics and adverse events. This trial aimed to investigate whether lamotrigine is non-inferior to mexiletine to directly inform clinical practice.

METHODS:

We did a randomised, double-blind, crossover, non-inferiority, phase 3 trial at the National Hospital for Neurology and Neurosurgery (London, UK). Participants (aged ≥18 years) who had genetically confirmed symptomatic non-dystrophic myotonia were randomly assigned (1:1), by means of a block randomisation schedule created by a computer program, to receive either mexiletine for 8 weeks followed by lamotrigine for 8 weeks, or lamotrigine followed by mexiletine, with a 7-day washout period in between. Investigators and participants were masked to treatment allocation. The primary outcome measure was the mean interactive voice response (IVR) diary stiffness score (0-9 scale) over the participant's final 2 weeks of diary reporting in each treatment period. Non-inferiority was assessed using a mixed-effects model with a predefined margin of 0·5 and included all randomly assigned participants who contributed at least 7 days of IVR-diary data in either treatment period. The trial is registered at ClinicalTrials.gov, NCT05017155, and EudraCT, 2020-003375-17.

FINDINGS:

Between Aug 1, 2021, and Dec 12, 2022, of 60 participants were screened (24 females and 36 males) and randomly assigned between Aug 1, 2021 and Dec 12, 2022, to either the mexiletine-lamotrigine sequence (n=30) or the lamotrigine-mexiletine sequence (n=30). 53 participants contributed data to the primary analysis. The mean IVR stiffness score after treatment with mexiletine was 2·54 (95% CI 1·98 to 3·10) versus 2·77 (2·21 to 3·32) with lamotrigine (mean mexiletine-lamotrigine difference -0·23 [95% CI -0·63 to 0·17]). The most common adverse event with both treatments was indigestion-reflux (eight participants, 208 participant-days receiving mexiletine; seven participants, 130 participant-days receiving lamotrigine). No serious adverse events were reported.

INTERPRETATION:

We were unable to conclude that lamotrigine is non-inferior to mexiletine; however, improvements in all outcome measures from baseline were similar between lamotrigine and mexiletine. Lamotrigine is an important treatment consideration in non-dystrophic myotonias alongside mexiletine; we propose a treatment algorithm to guide clinical practice.

FUNDING:

Neuromuscular Study Group, Jon Moulton Charity Trust, UCLH BRC Fast Track Grant.

News (Medical) associated with Mexiletine Hydrochloride

06 Nov 2023

·www.prnewswire.com

Emerging Market Trends in Muscular Dystrophy: A DelveInsight Analysis of Becker Muscular Dystrophy, Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, and Limb-girdle Muscular Dystrophy

Muscular dystrophies are a group of genetic muscle disorders characterized by progressive muscle weakness. There are more than 40 different variations, and the most common ones in children are Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). LAS VEGAS, Nov. 6, 2023 /PRNewswire/ -- Muscular dystrophies encompass a range of muscle disorders resulting from genetic mutations. Over time, these conditions lead to muscle weakness, which hinders everyday activities. Muscular dystrophy takes various forms, each targeting specific muscle groups and manifesting at different ages with varying degrees of severity. It can either be hereditary or occur as a spontaneous genetic mutation. Additionally, there may be multiple genetic subtypes within each specific type of muscular dystrophy, and individuals with the same subtype may exhibit varying symptoms. DelveInsight has recently released a series of epidemiology-based market reports focusing on muscular dystrophy including Becker Muscular Dystrophy, Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, and Limb-girdle Muscular Dystrophy. These reports include a comprehensive understanding of current treatment practices, historical and forecasted patient pool, emerging drugs, market share of individual therapies, and historical and forecasted market size from 2019 to 2032 segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan]. Additionally, the reports feature exhaustive analysis of prominent companies working with their emerging candidates in different stages of clinical development. Let's deep dive into the assessment of these muscular dystrophy reports market individually. Becker Muscular Dystrophy Market Becker muscular dystrophy (BMD) is a genetic disorder that primarily impacts muscle structure. It leads to the gradual deterioration of limb, cardiac, and skeletal muscles. Interestingly, involuntary muscles remain unaffected. BMD is considered a milder form of Duchenne muscular dystrophy (DMD). Typically, BMD manifests in one's teenage years or early adulthood, and its progression is slower and less predictable compared to DMD. As per DelveInsight analysis, it can be observed that prevalence of BMD is high in the 7MM, and similarly, high rate of treatment is seen in patients. The approach to BMD treatment centers on symptom management since there is no definitive cure available. Instead, there are treatments designed to alleviate symptoms and enhance muscle functionality. It's essential for individuals living with BMD to maintain their physical conditioning and muscle usage. While there are no FDA-approved drugs specifically for BMD, researchers are actively investigating various therapeutic interventions. As per DelveInsight analysis, the dynamics of the becker muscular dystrophy market are anticipated to change in the coming years owing to the positive outcomes of the emerging pipeline candidates during the developmental stage by key players that are under phase III, II and I clinical development have the potential to create a significant positive shift in BMD market size. Becker Muscular Dystrophy Pipeline Therapies and Companies EDG-5506: Edgewise Therapeutics, Inc./Medpace, Inc. Vamorolone: ReveraGen BioPharma, Inc./Santhera Pharmaceuticals For a comprehensive view of the Becker muscular dystrophy market, check out the Becker Muscular Dystrophy Market Assessment Myotonic Dystrophy Market Myotonic dystrophy, an umbrella term for two rare genetic disorders with autosomal dominant inheritance patterns, impacts various bodily systems, featuring a clinical triad of progressive muscle weakness, myotonia, and early-onset cataracts. These disorders are classified into two primary types: DM type 1 (DM1) and DM type 2 (DM2). DelveInsight estimated that the total diagnosed prevalent patient population of myotonic dystrophy in the 7MM countries was close to 80K cases in 2021. As per the estimates, the US had the highest patient population of myotonic dystrophy in 2021. Although numerous pre-clinical advancements have been made, there is currently no targeted disease-modifying treatment available. The mainstay of management primarily involves monitoring for complications and adhering to standard care practices, such as utilizing assistive devices, administering hormone therapy, and providing pain relief. Several clinical studies have systematically assessed the efficacy of therapeutic agents in managing the condition. However, the scarcity of scientific evidence, coupled with the complex and diverse manifestations of the disease, poses significant challenges for healthcare providers when it comes to identifying and selecting appropriate medications. To tap the uncovered myotonic dystrophy market, few players have targeted the unmet need fortreatment. As per DelveInsight analysis, the myotonic dystrophy market is expected to grow from USD 80 million in 2021 at a significant CAGR by 2032. The current pipeline consists of both mid and late-stage candidates. The few promising therapies inthe mid and late-stage development are expected to experience significant reconstitution during the forecastperiod. Hence, more research and studies are needed to develop potential therapies that cater to the unmet need for curative and disease-modifying therapy in myotonic dystrophy patients. Myotonic Dystrophy Pipeline Therapies and Companies AMO-02 (tideglusib): AMO Pharma Limited Mexiletine: Lupin Ltd. Pitolisant: Harmony Biosciences, LLC AOC 1001: Avidity Biosciences, Inc. DYNE-101: Dyne Therapeutics To gain a deeper understanding of the myotonic dystrophy market, be sure to explore the Myotonic Dystrophya Market Outlook Facioscapulohumeral Muscular Dystrophy (FSHD) Market Facioscapulohumeral muscular dystrophy (FSHD), a genetically inherited disorder, progressively weakens muscles and significantly reduces the functional capacity of those affected. Recent years have witnessed substantial progress in our understanding of how epigenetic factors play a role in this condition. FSHD can be categorized into two subtypes based on the mechanisms of reduced methylation and failed epigenetic repression. Type 1 accounts for approximately 95% of FSHD cases and is characterized by the loss of D4Z4 repeats. The remaining 5% fall into Type 2, where methylation and epigenetic changes occur independently of deletions. Regardless of the subtype, FSHD leads to the same outcome. As per DelveInsight analysis, in the year 2021, the total prevalent cases of FSHD were 78K in the 7MM which are expected to grow during the study period, i.e., 2019–2032. As of now, there are no officially approved therapies for managing FSHD. However, individuals dealing with FSHD can find relief by utilizing orthotic devices designed to support their feet. These devices are particularly effective in addressing common FSHD symptoms like foot drop and shoulder weakness. Depending on the degree of foot drop, a custom-made ankle-foot orthosis (AFO) can provide significant benefits. In cases where knee extensor weakness may worsen gait with an AFO, alternatives like floor reaction ankle-foot orthosis (FRAFO) or advanced knee-ankle-foot orthosis (KAFO) may be considered. Regular exercise, especially hydrotherapy, plays a crucial role in preserving mobility and pain management. Additionally, weight management, potentially through dietary adjustments, is essential to reduce strain on already weakened muscles. It's important to note that there is currently no cure for FSHD, but there are treatments and devices available to alleviate many of its symptoms. Non-steroidal anti-inflammatory drugs (NSAIDs) are commonly prescribed to enhance comfort and mobility. According to DelveInsight, the FSHD market in 7MM is expected to witness a major change in the study period (2019–2032). As per estimates, in 2021, the total FSHD market size was USD 25.55 million which is expected to rise by 2032. This increase in market size is mainly due to extensive research and development activities of pharmaceutical companies, along with the expected launch of approved therapies. Facioscapulohumeral Muscular Dystrophy Pipeline Therapies and Companies Losmapimod: Fulcrum Therapeutics/GSK GYM329 (RO7204239/RG 6237): Roche Explore in-depth for a comprehensive understanding of the FSHD Clinical Trials Limb-girdle Muscular Dystrophy Market Limb-girdle muscular dystrophies (LGMD), also known as pelvofemoral muscular dystrophy or proximal muscular dystrophy, encompass a group of infrequent genetic disorders characterized by the progressive wasting and weakening of voluntary muscles located in the hip and shoulder regions. This muscle weakness and atrophy gradually advance and can eventually impact other muscle groups in the body. As per data from the National Organization for Rare Disorders, LGMD affects both males and females in roughly equal proportions. The exact prevalence of LGMD remains uncertain, but estimates suggest a range between one in 14,500 to one in 123,000 individuals. It's important to note that the age at which symptoms manifest can vary significantly, even within the same family. Additionally, the relative frequencies of the different LGMD types differ across populations, though it is worth mentioning that LGMD2G, 2H, and 2J are exceedingly rare on a global scale. Currently, there is no known cure for LGMD. However, there are supportive therapies designed to manage and alleviate its symptoms. The approach to LGMD management is individualized, focusing on addressing specific symptoms that each patient experiences. Treatment options may involve physical and occupational therapy to enhance muscle strength and prevent contractures, the utilization of assistive devices like canes, braces, walkers, or wheelchairs to aid mobility, corrective surgery for skeletal issues like scoliosis, and regular monitoring of cardiac and respiratory health for potential complications associated with certain LGMD subtypes. According to DelveInsight's' estimates, the limb girdle muscular dystrophy market in 7MM is expected to show a positive growth, during the forecast period (2023–2032), mainly attributed to anticipated launch of emerging therapy and the increasing cases of LGMD. Limb-girdle Muscular Dystrophy Pipeline Therapies and Companies SRP-6004: Sarepta Therapeutics, Inc. LION-101: Asklepios Biopharmaceutical, Inc. BBP-418 (ribitol): ML Bio Solutions, Inc. To access a complete analysis of the limb-girdle muscular dystrophy market, visit Limb-girdle Muscular Dystrophy Market Assessment Trending Genetic Disorders Reports Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2032 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 DMD companies, including Pfizer, Santhera Pharmaceuticals, ReveraGen BioPharma, Taiho Pharmaceutical, FibroGen, Sarepta Therapeutics, Capricor Therapeutics, Daiichi Sankyo, Italfarmaco, Antisense Therapeutics, Solid Biosciences, among others. Alpha-1 Antitrypsin Deficiency Market Alpha-1 Antitrypsin Deficiency Market Insights, Epidemiology, and Market Forecast – 2032 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 A1ATD companies, including Arrow Head Pharmaceuticals, Mereo Biopharma, Kamada Ltd, Grifols, CSL Behring, Shire, LFB Biotechnologies, among others. Wilson Disease Market Wilson Disease Market Insights, Epidemiology, and Market Forecast – 2032 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 Wilson disease companies, including Orphalan, AstraZeneca, Vivet Therapeutics, Pfizer, Ultragenyx Pharmaceutical, among others. Cystic Fibrosis Market Cystic Fibrosis Market Insights, Epidemiology, and Market Forecast – 2032 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 cystic fibrosis companies, including Verona Pharmaceuticals, Eloxx Pharmaceuticals, Inc., Santhera Pharmaceuticals, Laurent Pharmaceuticals Inc., Vertex Pharmaceuticals, among others. About DelveInsight DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. 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] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

Phase 3

08 Jul 2021

·www.biospace.com

PANTHERx® Rare Selected by Mitsubishi Tanabe Pharma America, Inc. (MTPA) as Exclusive Provider of U.S. Specialty Pharmacy Services for EXSERVAN™ (riluzole) oral film

PITTSBURGH, July 8, 2021 /PRNewswire/ -- PANTHERx Rare announced today that it was selected by Mitsubishi Tanabe Pharma America, Inc. (MTPA) as the exclusive U.S. pharmacy distribution partner for EXSERVAN™ (riluzole). The drug, an oral film formulation of riluzole, was approved by the U.S. Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS). "PANTHERx is excited to be supporting ALS patients who have been prescribed EXSERVAN through medication dispensing and patient education as part of our proprietary RxARECARE® model," said Rob Snyder, President of PANTHERx. PANTHERx utilizes technology and human capital to create RxARECARE programs tailored to the unique needs of the medications we dispense and the preferences of each patient we support. About PANTHERx Rare PANTHERx, part of Envolve Pharmacy Solutions, is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions. Although the incidence is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an approved therapy. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community. PANTHERx was recently awarded Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy (NASP) and earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC). PANTHERx is a three-time winner of the prestigious MMIT Patient Choice Award, including the 2020 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC. About EXSERVAN (riluzole) oral film EXSERVAN, an oral film formulation of riluzole, was approved by the U.S. Food and Drug Administration (FDA) in November 2019. EXSERVAN was developed by Aquestive Therapeutics using its PharmFilm® innovative drug delivery technology. The oral film is placed on top of the patient's tongue and dissolves. Under the terms of a licensing and supply deal agreement, MTPA is commercializing EXSERVAN in the U.S. and Aquestive is serving as the exclusive sole manufacturer and supplier for the product. For more information, visit . INDICATION EXSERVAN™ (riluzole) is indicated for the treatment of amyotrophic lateral sclerosis (ALS). IMPORTANT SAFETY INFORMATION Do not use if you are allergic to riluzole or to any of its ingredients. Before using EXSERVAN, tell your doctor about all the medicines you take and all your health conditions, including if you: Have liver problems. Are taking certain drugs known as strong or moderate CYP1A2 inhibitors such as ciprofloxacin, enoxacin, fluvoxamine, methoxsalen, mexiletine, oral contraceptives, thiabendazole, vemurafenib, or zileuton. Are pregnant or intend to become pregnant during EXSERVAN therapy, or if you are breastfeeding or intend to breastfeed during EXSERVAN therapy. EXSERVAN can cause serious side effects, including: Liver Problems: Cases of liver injury, some fatal, have occurred in patients taking riluzole. Consult your doctor promptly if you experience unexplained nausea, vomiting, stomach (abdominal) pain, fatigue, weight loss (anorexia), jaundice (yellowing of the skin or whites of the eyes), and/or dark urine. Low White Blood Cell Counts (Neutropenia): Tell your doctor if you develop a fever while taking EXSERVAN. Serious Lung Problems (Interstitial Lung Disease): Tell your doctor if you have problems with your lungs or breathing such as dry cough and difficult or labored breathing. Discontinue EXSERVAN immediately if you develop interstitial lung disease. The most common side effects include numbness in the mouth or tongue, muscle weakness, nausea, lung problems, high blood pressure, and stomach (abdominal) pain. These are not all the possible side effects of EXSERVAN. Consult your doctor for medical advice about side effects. To report side effects, contact Mitsubishi Tanabe Pharma America, Inc. at 1-888-292-0058 or the FDA at 1-800-FDA-1088 or . Use EXSERVAN as prescribed. The recommended dosage for EXSERVAN is 50 mg taken orally twice daily at least 1 hour before or 2 hours after a meal. Place EXSERVAN oral film strip on the top of the tongue where it will adhere and dissolve. Swallow in a normal manner. Do not cut or split the film or take liquids with EXSERVAN. Do not chew, spit, or talk while EXSERVAN is dissolving. Read the Instructions for Use for EXSERVAN. Please click here to see the full Prescribing Information for EXSERVAN. Contact: Allyson Ayoob, 855-726-8479 x 1142, aayoob@pantherxrare.com

CollaborateOrphan DrugInnovative Drug

25 Jun 2021

·www.biospace.com

MTPA Announces PANTHERx® Selected as Exclusive Provider of U.S. Specialty Pharmacy Services for EXSERVAN™ (riluzole) Oral Film

JERSEY CITY, N.J., June 25, 2021 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced PANTHERx Rare Pharmacy has been selected as the exclusive provider of specialty pharmacy services for EXSERVAN™ (riluzole), an oral film formulation of riluzole for the treatment of amyotrophic lateral sclerosis (ALS). "As we explored options for specialty pharmacy partners for the distribution of EXSERVAN, it was important to find an organization with specific knowledge and experience that could best support the ALS community," said Kevin O'Brien, Vice President of Market Access, MTPA. "PANTHERx is able to deliver on both fronts, in addition to also having an unwavering dedication to patients. This is closely aligned with our commitment to helping patients." Once a patient is prescribed EXSERVAN, they may be enrolled in access information services. Upon enrollment, PANTHERx will provide patients with a review and summary of insurance benefit options, including information about eligibility for the Out-of-Pocket Assistance Program for EXSERVAN. MTPA plans to make EXSERVAN available to patients by the end of June 2021. "PANTHERx prides itself on being focused on people living with the implications of rare disease," said Rob Snyder, President of PANTHERx. "We hope our extensive experience will positively impact the members of the ALS community that we aim to serve through our dispensing of EXSERVAN. We look forward to working with MTPA to help address the needs of people impacted by this devastating disease." About EXSERVAN™ (riluzole) oral film EXSERVAN, an oral film formulation of riluzole, was approved by the U.S. Food and Drug Administration (FDA) in November 2019. EXSERVAN was developed by Aquestive Therapeutics, Inc. using its PharmFilm® innovative drug delivery technology. The oral film is placed on top of the patient's tongue and dissolves. Under the terms of a licensing and supply deal agreement, MTPA is commercializing EXSERVAN in the U.S. and Aquestive is serving as the exclusive sole manufacturer and supplier for the product. For more information, visit . INDICATION EXSERVAN™ (riluzole) is indicated for the treatment of amyotrophic lateral sclerosis (ALS). IMPORTANT SAFETY INFORMATION Do not use if you are allergic to riluzole or to any of its ingredients. Before using EXSERVAN, tell your doctor about all the medicines you take and all your health conditions, including if you: Have liver problems. Are taking certain drugs known as strong or moderate CYP1A2 inhibitors such as ciprofloxacin, enoxacin, fluvoxamine, methoxsalen, mexiletine, oral contraceptives, thiabendazole, vemurafenib, or zileuton. Are pregnant or intend to become pregnant during EXSERVAN therapy, or if you are breastfeeding or intend to breastfeed during EXSERVAN therapy. EXSERVAN can cause serious side effects, including: Liver Problems: Cases of liver injury, some fatal, have occurred in patients taking riluzole. Consult your doctor promptly if you experience unexplained nausea, vomiting, stomach (abdominal) pain, fatigue, weight loss (anorexia), jaundice (yellowing of the skin or whites of the eyes), and/or dark urine. Low White Blood Cell Counts (Neutropenia): Tell your doctor if you develop a fever while taking EXSERVAN. Serious Lung Problems (Interstitial Lung Disease): Tell your doctor if you have problems with your lungs or breathing such as dry cough and difficult or labored breathing. Discontinue EXSERVAN immediately if you develop interstitial lung disease. The most common side effects include numbness in the mouth or tongue, muscle weakness, nausea, lung problems, high blood pressure, and stomach (abdominal) pain. These are not all the possible side effects of EXSERVAN. Consult your doctor for medical advice about side effects. To report side effects, contact Mitsubishi Tanabe Pharma America, Inc. at 1-888-292-0058 or the FDA at 1-800-FDA-1088 or . Use EXSERVAN as prescribed. The recommended dosage for EXSERVAN is 50 mg taken orally twice daily at least 1 hour before or 2 hours after a meal. Place EXSERVAN oral film strip on the top of the tongue where it will adhere and dissolve. Swallow in a normal manner. Do not cut or split the film or take liquids with EXSERVAN. Do not chew, spit, or talk while EXSERVAN is dissolving. Read the Instructions for Use for EXSERVAN. Please click here to see the full Prescribing Information for EXSERVAN. About Mitsubishi Tanabe Pharma America, Inc. Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation's (MTPC) 100 percent owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. It was established by MTPC to commercialize approved pharmaceutical products in North America. For more information, please visit or follow us on Twitter, Facebook and LinkedIn. Overview of Mitsubishi Tanabe Pharma Corporation Mitsubishi Tanabe Pharma, which was founded in 1678, has its headquarters in Doshomachi, Osaka, which is the birthplace of Japan's pharmaceutical industry. With business centered on ethical pharmaceuticals, Mitsubishi Tanabe Pharma is a well-established company with one of the longest histories of pharmaceutical companies in Japan.1 In accordance with the corporate philosophy of "contributing to the healthier lives of people around the world through the creation of pharmaceuticals," the Company formulated the key concept of Open Up the Future under the Medium-Term Management Plan 2016-2020. Through the discovery of drugs that address unmet medical needs, centered on its priority disease areas — immune-inflammation diseases, diabetes and kidney, central nervous system, and vaccines — Mitsubishi Tanabe Pharma will strive to contribute to the health of patients around the world. MTPC is the parent company of MTPA. For more information, go to . PharmFilm® and the Aquestive logo are registered trademarks of Aquestive Therapeutics, Inc. All other registered trademarks referenced herein are the property of their respective owners. 1 Research by TOKYO SHOKO RESEARCH, LTD. Media Inquiries: Debbie Etchison 908-340-8578 Media_MTPA@mt-pharma-us.com

Innovative DrugVaccine

100 Deals associated with Mexiletine Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D00639	Mexiletine Hydrochloride	C01BB02	DB00379

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Non dystrophic myotonia	EU	Lupin Ltd	18 Dec 2018
Non dystrophic myotonia	IS	Lupin Ltd	18 Dec 2018
Non dystrophic myotonia	LI	Lupin Ltd	18 Dec 2018
Non dystrophic myotonia	NO	Lupin Ltd	18 Dec 2018
Lung Abscess	JP	Chemix, Inc.	08 Jul 2005
Peritonitis	JP	Chemix, Inc.	08 Jul 2005
Pneumonia	JP	Chemix, Inc.	08 Jul 2005
Arrhythmias, Cardiac	JP	Taiyo Pharma Co., Ltd.	31 Jan 1985
Diabetic Neuropathies	JP	Taiyo Pharma Co., Ltd.	31 Jan 1985

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Myotonic Dystrophy	Phase 3	FR	Lupin Ltd	03 Sep 2021
Myotonic Dystrophy	Phase 3	-	Lupin Ltd	03 Sep 2021
HIV Infections	Phase 2	US	Boehringer Ingelheim GmbH	31 Aug 2001
HIV Infections	Phase 2	TZ	Boehringer Ingelheim GmbH	31 Aug 2001
Neuropathy, Painful	Phase 2	US	Boehringer Ingelheim GmbH	31 Aug 2001
Neuropathy, Painful	Phase 2	TZ	Boehringer Ingelheim GmbH	31 Aug 2001

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


MDS2023	Not Applicable	Myotonia	183	Mexiletine	mxcgydobjn(uxzaroybmy) = mexiletine significantly causes some GIT symptoms compared to placebo (RR 3.7, 95% CI [1.79 to 7.64]) exabewxtiw (fclijgauqn )	Positive	27 Aug 2023
ESC2020	Not Applicable	Long Qt Syndrome 2	20	mexiletine	rutprkdjxl(ijemecnsaz) = osdzlmyhhp kcbhskzxbh (dgllojsqoz )	-	28 Aug 2020
2531 (AAN2020)	Phase 2	Amyotrophic Lateral Sclerosis	20	Placebo	dlefsehmpr(gzxkhucmno) = spepsclsma ecntagpuxs (opdtgqnato, 2.2) View more	Positive	14 Apr 2020
				Mexiletine 300 mg	vkuayxdgvr(ndyzdkosfs) = evutsehack yujpdtnmvc (tpdvdkbrqm, 0.315 - 1.102)
NCT02781454 (Mexiletine-2, CTgov)	Phase 2	Amyotrophic Lateral Sclerosis	20	Mexiletine (Mexiletine, 300 Milligrams)	tiggzoszmj(zrfxuzhrfr) = ffgbcispgl igxgswdpdt (razffrstlb, kpsszhbxdi - ufwrbahkxt) View more	-	05 Dec 2019
				Mexiletine (Mexiletine, 600 Milligrams)	tiggzoszmj(zrfxuzhrfr) = rrrkidqpot igxgswdpdt (razffrstlb, pahfdlfovr - rljlyfcgdx) View more
WCN2019	Not Applicable	Non dystrophic myotonia	-	Safinamide	mltnyiqszt(ijpfeaskdt) = ndlxsvrttu wwifnwrbsy (efztbuevrq ) View more	-	15 Oct 2019
				Mexiletine	mltnyiqszt(ijpfeaskdt) = vcekqpsqlf wwifnwrbsy (efztbuevrq ) View more
NCT02260388 (PAIN-CONTRoLS, CTgov)	Phase 4	Pain \| Sensory polyneuropathy \| Neuropathy	402	Nortriptyline (Nortriptyline)	xugnsocqnh(roefxgefpf) = jtgkjdclpl mtvwpykuef (jcvxphbtfu, vbariqyaro - kaktmowttn) View more	-	06 Jul 2018
				Duloxetine (Duloxetine)	xugnsocqnh(roefxgefpf) = jgvkcwvbtn mtvwpykuef (jcvxphbtfu, dqvpibyxqb - ectbfeaxjk) View more
AAN2018	Not Applicable	Non dystrophic myotonia	30	Mexiletine	ravpecqdjj(vcumtcfqxa) = ajngswiwcm frkxiwcpka (aeidfcssax ) View more	Positive	10 Apr 2018
NCT01406873 (CTgov)	Phase 2	Myotonic Dystrophy	42	Mexiletine	nnetjleslf(rrxriogskj) = dzrrhbohca orbynuxzjm (guxrcedafn, anhfrsimjl - duoqzhiyrk) View more	-	29 Mar 2018
NCT01811355 (CTgov)	Phase 4	Muscle Cramp \| Amyotrophic Lateral Sclerosis	23	Mexiletine (Mexiletine)	yutjwjndtu(doxrbozubv) = sriymxqxke zjbqgrfana (ynyxwylycx, xbrgfishqu - qldkesndpt) View more	-	27 Jun 2017
				Placebo (Placebo)	yutjwjndtu(doxrbozubv) = vkoorvcmjf zjbqgrfana (ynyxwylycx, dssaqwhifv - samxmgyyma) View more
S38.004 (AAN2017)	Not Applicable	Muscle Cramp	20	Mexiletine 150mg BID	ppcjevqjfp(zqvvkkrvuv) = zrdnabwvqc ggdrjzgarm (apiyrkeukq ) View more	Positive	18 Apr 2017

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