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DelveInsight’s “Generalized Myasthenia Gravis Market Insights, Epidemiology, and Market Forecast-2032″ report offers an in-depth understanding of the Generalized Myasthenia Gravis, historical and forecasted epidemiology as well as the Generalized Myasthenia Gravis market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

To Know in detail about the Generalized Myasthenia Gravis market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Generalized Myasthenia Gravis Market Forecast

Some of the key facts of the Generalized Myasthenia Gravis Market Report:

• The Generalized Myasthenia Gravis market size was valued approximately ~USD 2,700 million in 2021 and is anticipated to grow with a significant CAGR during the study period (2019-2032)

• In January 2025, The U.S. Food and Drug Administration (FDA) has granted Priority Review to Johnson & Johnson’s Biologics License Application (BLA) for nipocalimab, a treatment for generalized myasthenia gravis (gMG) in patients who are antibody-positive, including those with anti-AChR, anti-MuSK, and anti-LRP4 antibodies.

• In October 2024, Amgen (NASDAQ:AMGN) has announced the presentation of favorable top-line results from the Phase 3 MINT trial, which assessed the efficacy and safety of UPLIZNA® (inebilizumab-cdon) in treating adults with generalized myasthenia gravis (gMG), a rare autoimmune disease.

• In August 2024, Merck KGaA (Merck) administered the first dose in a Phase III trial of oral Cladribine for treating generalized myasthenia gravis (gMG), a rare neuromuscular condition. Named MyClad, this international, randomized, double-blind, placebo-controlled study will evaluate the efficacy and safety of Cladribine capsules in comparison to a placebo.

• In June 2024, Johnson & Johnson (NYSE: JNJ) announced positive findings from the Phase 3 Vivacity-MG3 study of nipocalimab in patients with generalized myasthenia gravis (gMG). Patients receiving nipocalimab in combination with standard of care (SOC) showed superior results compared to those treated with placebo and SOC, as indicated by the primary endpoint of improvement in the MG-ADL score from baseline over 24 weeks. These results will be featured in a presentation at the European Academy of Neurology (EAN) 2024 Congress and will be part of submissions to regulatory authorities later this year.

• In January 2024, RYSTIGGO® (rozanolixizumab-noli) is a targeted treatment approved for adults with generalized myasthenia gravis (gMG) who test positive for anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibodies.

• In February 2024, Johnson & Johnson (J&J) unveiled favorable top-line findings for nipocalimab across two rare disease categories: generalized myasthenia gravis (gMG) and Sjogren’s disease. These achievements follow positive outcomes observed in two other autoantibody-related conditions, namely, haemolytic disease of the foetus and newborn (HDFN) and rheumatoid arthritis (RA). The Phase III VIVACITY trial (NCT03772587) of nipocalimab in adult gMG patients achieved its primary objective, demonstrating a statistically significant decrease in daily activity scores (measured by the myasthenia gravis activities of daily living scale – MG-ADL) from baseline over a period of 22 to 24 weeks, relative to placebo.

• In 2021, the market size for Generalized Myasthenia Gravis (gMG) across the United States, EU5 countries (Germany, Spain, Italy, France, and the United Kingdom), and Japan was estimated to be around USD 2,700 million.

• According to Suresh et al. (2021), there are 20 cases of myasthenia gravis for every 100,000 people in the United States. It shows that those under 40 have a higher female predominance, while people over 50 have a higher male predominance

• The prevalence of MG varies from 1.5 to 17.9, or 2.19 to 36.71 cases/100.000 population, depending on the region. This suggests that there are roughly 60.000 sufferers in the US and 56,000–123,000 in Europe

• According to a Green et al. (2020) study, 5.6% of the North American sample had non-family myasthenia gravis. While the majority of instances of myasthenia gravis are still not familial, a prevalence of 5.6% is several hundred times higher than the overall prevalence of 1 in 5–10 000 cases of the condition

• Key Generalized Myasthenia Gravis Companies: Viela Bio, UCB Pharma, Ra Pharmaceuticals, Argenx, Alexion Pharmaceuticals, Takeda, Argenx-Halozyme Therapeutics, Horizon Therapeutics, Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Sanofi, Cartesian Therapeutics, Takeda, DAS Therapeutics, Inc., and others

• Key Generalized Myasthenia Gravis Therapies: Inebilizumab, Rozanolixizumab, Zilucoplan, Efgartigimod, Eculizumab, TAK-079, Subcutaneous Efgartigimod, Uplizna (Inebilizumab), Enspryng (Satralizumab), Nipocalimab, Batoclimab, Tolebrutinib, Descartes-08, Mezagitamab (TAK-079), DAS-001, and others

• The Generalized Myasthenia Gravis epidemiology based on gender analyzed that prevalence of Generalized Myasthenia Gravis is higher in females than males

• The Generalized Myasthenia Gravis market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Generalized Myasthenia Gravis pipeline products will significantly revolutionize the Generalized Myasthenia Gravis market dynamics.

Generalized Myasthenia Gravis Overview

Generalized Myasthenia Gravis (gMG) is a chronic autoimmune neuromuscular disorder that causes muscle weakness. It occurs when the immune system produces antibodies that disrupt communication between nerves and muscles, targeting proteins like the acetylcholine receptor or related components at the neuromuscular junction.

Symptoms of gMG include weakness in the arms, legs, neck, eyes (causing drooping eyelids or double vision), and difficulties with speaking, swallowing, and breathing. The severity of symptoms can vary and may worsen with activity but improve with rest.

Get a Free sample for the Generalized Myasthenia Gravis Market Report:

https://www.delveinsight.com/report-store/generalized-myasthenia-gravis-gmg-market

Generalized Myasthenia Gravis Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Generalized Myasthenia Gravis Epidemiology Segmentation:

The Generalized Myasthenia Gravis market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:

• Total Prevalence of Generalized Myasthenia Gravis

• Prevalent Cases of Generalized Myasthenia Gravis by severity

• Gender-specific Prevalence of Generalized Myasthenia Gravis

• Diagnosed Cases of Episodic and Chronic Generalized Myasthenia Gravis

Download the report to understand which factors are driving Generalized Myasthenia Gravis epidemiology trends @ Generalized Myasthenia Gravis Epidemiology Forecast

Generalized Myasthenia Gravis Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Generalized Myasthenia Gravis market or expected to get launched during the study period. The analysis covers Generalized Myasthenia Gravis market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Generalized Myasthenia Gravis Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Generalized Myasthenia Gravis Therapies and Key Companies

• Inebilizumab: Viela Bio

• Rozanolixizumab: UCB Pharma

• Zilucoplan: Ra Pharmaceuticals

• Efgartigimod: Argenx

• Eculizumab: Alexion Pharmaceuticals

• TAK-079: Takeda

• Subcutaneous Efgartigimod: Argenx-Halozyme Therapeutics

• Uplizna (Inebilizumab): Horizon Therapeutics

• Enspryng (Satralizumab): Hoffmann-La Roche

• Nipocalimab: Janssen Research & Development, LLC

• Batoclimab: Immunovant Sciences GmbH

• Tolebrutinib: Sanofi

• Descartes-08: Cartesian Therapeutics

• Mezagitamab (TAK-079): Takeda

• DAS-001: DAS Therapeutics, Inc.

Discover more about therapies set to grab major Generalized Myasthenia Gravis market share @ Generalized Myasthenia Gravis Treatment Market

Generalized Myasthenia Gravis Market Strengths

• Growing research and development is increasing the demand for better diagnosis and treatment options for MG.

• Approval of targeted therapies such as Soliris, Vygart, and Ultomiris will fuel the market growth.

Generalized Myasthenia Gravis Market Opportunities

• Approval of new therapies will provide better treatment options

• Various big Pharma key players are currently active in the development of a novel drug for the treatment of MG.

Scope of the Generalized Myasthenia Gravis Market Report

• Study Period: 2019–2032

• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

• Key Generalized Myasthenia Gravis Companies: Viela Bio, UCB Pharma, Ra Pharmaceuticals, Argenx, Alexion Pharmaceuticals, Takeda, Argenx-Halozyme Therapeutics, Horizon Therapeutics, Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Sanofi, Cartesian Therapeutics, Takeda, DAS Therapeutics, Inc., and others

• Key Generalized Myasthenia Gravis Therapies: Inebilizumab, Rozanolixizumab, Zilucoplan, Efgartigimod, Eculizumab, TAK-079, Subcutaneous Efgartigimod, Uplizna (Inebilizumab), Enspryng (Satralizumab), Nipocalimab, Batoclimab, Tolebrutinib, Descartes-08, Mezagitamab (TAK-079), DAS-001, and others

• Generalized Myasthenia Gravis Therapeutic Assessment: Generalized Myasthenia Gravis current marketed and Generalized Myasthenia Gravis emerging therapies

• Generalized Myasthenia Gravis Market Dynamics: Generalized Myasthenia Gravis market drivers and Generalized Myasthenia Gravis market barriers

• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies

• Generalized Myasthenia Gravis Unmet Needs, KOL’s views, Analyst’s views, Generalized Myasthenia Gravis Market Access and Reimbursement

To know more about Generalized Myasthenia Gravis companies working in the treatment market, visit @ Generalized Myasthenia Gravis Clinical Trials and Therapeutic Assessment

Table of Contents

1. Generalized Myasthenia Gravis Market Report Introduction

2. Executive Summary for Generalized Myasthenia Gravis

3. SWOT analysis of Generalized Myasthenia Gravis

4. Generalized Myasthenia Gravis Patient Share (%) Overview at a Glance

5. Generalized Myasthenia Gravis Market Overview at a Glance

6. Generalized Myasthenia Gravis Disease Background and Overview

7. Generalized Myasthenia Gravis Epidemiology and Patient Population

8. Country-Specific Patient Population of Generalized Myasthenia Gravis

9. Generalized Myasthenia Gravis Current Treatment and Medical Practices

10. Generalized Myasthenia Gravis Unmet Needs

11. Generalized Myasthenia Gravis Emerging Therapies

12. Generalized Myasthenia Gravis Market Outlook

13. Country-Wise Generalized Myasthenia Gravis Market Analysis (2019–2032)

14. Generalized Myasthenia Gravis Market Access and Reimbursement of Therapies

15. Generalized Myasthenia Gravis Market Drivers

16. Generalized Myasthenia Gravis Market Barriers

17. Generalized Myasthenia Gravis Appendix

18. Generalized Myasthenia Gravis Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare 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.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Gaurav Bora
Email: Send Email
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/

Press Release Distributed by ABNewswire.com

To view the original version on ABNewswire visit: Generalized Myasthenia Gravis Market on Track for Major Expansion by 2032, According to DelveInsight | Viela Bio, UCB Pharma, Ra Pharma, Argenx, Alexion Pharma, Takeda, Argenx-Halozyme Therapeutics

The Hemophilia B market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Hemophilia B pipeline products will significantly revolutionize the Hemophilia B market dynamics.

DelveInsight’s “Hemophilia B Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Hemophilia B, historical and forecasted epidemiology as well as the Hemophilia B market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Hemophilia B market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2020 to 2034. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

To Know in detail about the Hemophilia B market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Hemophilia B Market Insights

Some of the key facts of the Hemophilia B Market Report:

• The Hemophilia B market size was valued ~USD 3,400 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034)

• In December 2024, The first-in-human Phase 1/2 clinical trial for BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B, is now recruiting patients at two U.S. sites. BE-101 is a pioneering therapy designed to address the ongoing unmet needs of hemophilia B patients, particularly those who continue to experience frequent bleeding episodes. The two-part Phase 1/2 trial, named BeCoMe-9 (NCT06611436), aims to evaluate the safety and clinical effectiveness of a single intravenous infusion of BE-101 in adults with moderately severe to severe hemophilia B.

• In April 2024, Pfizer Inc. (NYSE: PFE) announced that the U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for treating adults with moderate to severe hemophilia B. This approval is for individuals who are on factor IX (FIX) prophylaxis therapy, have experienced life-threatening hemorrhages, or suffer from recurrent, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid, as identified by an FDA-approved test. BEQVEZ is a one-time treatment aimed at allowing people with hemophilia B to produce FIX on their own, eliminating the need for frequent intravenous FIX infusions, which are often required several times a week or month.

• In February 2024, Intellia Therapeutics and Regeneron are set to initiate the first human trial of a CRISPR/Cas9-based Factor 9 (F9) gene-editing therapy for hemophilia B by mid-year. This announcement, made in an Intellia financial report, comes after the recent approval by the U.S. Food and Drug Administration (FDA) of the investigational new drug (IND) application filed by the two companies.

• Replacement therapy is the most widely used treatment for hemophilia B. In the U.S., several recombinant factor IX products are available, including BeneFIX, RIXUBIS, IXINITY, ALPROLIX, IDELVION, and REBINYN. Additionally, bypassing agents like Feiba, Novoseven, and SEVENFACT, along with human plasma-derived coagulation factor IX products such as AlphaNine SD and Mononine, have also received FDA approval.

• In 2023, the United States had the highest number of diagnosed prevalent cases of hemophilia B among the 7MM, with an estimated 4,000 cases. This number is anticipated to rise over the forecast period.

• In 2023, France reported the highest number of diagnosed prevalent cases of hemophilia B among the EU4 and the UK, whereas Germany had the lowest number of cases.

• Estimates show that in the US, hemophilia B was most commonly found in the 19-44 years age group, representing approximately 35% of the total cases.

• Key Hemophilia B Companies: Pfizer, CSL Behring/uniQure, HEMA Biologics/LFB, Spark Therapeutics, Sanofi, Alnylam Pharmaceuticals, CSL Behring, uniQure, Novo Nordisk, Freeline Therapeutics, ApcinteX, and others

• Key Hemophilia B Therapies: BEQVEZ (fidanacogene elaparvovec-dzkt), HEMGENIX (etranacogene dezaparvovec-drlb), SEVENFACT, Marstacimab (PF-06741086), SPK-9001, Fitusiran, AMT-061, Concizumab, FLT180a, Serpin, and others

• The lowest prevalent population of Hemophilia B were recorded in Spain

Hemophilia B Overview

Hemophilia B is a rare genetic bleeding disorder caused by a deficiency or dysfunction of clotting factor IX, a protein essential for blood clotting. It is an inherited condition, typically passed down through the X chromosome, affecting mostly males, while females are usually carriers. Individuals with Hemophilia B experience prolonged bleeding after injuries, surgeries, or spontaneously in severe cases. Common symptoms include frequent bruising, joint bleeds, and internal bleeding. Treatment involves replacing the missing factor IX through regular infusions or advanced gene therapies. Early diagnosis and proper management are crucial to improving quality of life and preventing complications.

Get a Free sample for the Hemophilia B Market Report:

https://www.delveinsight.com/report-store/hemophilia-b-market

Hemophilia B Market

The dynamics of the Hemophilia B market are anticipated to change in the coming years owing to the expected launch of emerging therapies and others during the forecasted period 2020-2034.

Hemophilia B Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Hemophilia B Epidemiology Segmentation:

The Hemophilia B market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total Prevalence of Hemophilia B

• Prevalent Cases of Hemophilia B by severity

• Gender-specific Prevalence of Hemophilia B

• Diagnosed Cases of Episodic and Chronic Hemophilia B

Download the report to understand which factors are driving Hemophilia B epidemiology trends @ Hemophilia B Epidemiological Insights

Hemophilia B Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Hemophilia B market or expected to get launched during the study period. The analysis covers Hemophilia B market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Hemophilia B Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Hemophilia B Therapies and Key Companies

• BEQVEZ (fidanacogene elaparvovec-dzkt): Pfizer

• HEMGENIX (etranacogene dezaparvovec-drlb): CSL Behring/uniQure

• SEVENFACT: HEMA Biologics/LFB

• Marstacimab (PF-06741086): Pfizer

• SPK-9001: Pfizer/Spark Therapeutics

• Fitusiran: Sanofi/ Alnylam Pharmaceuticals

• AMT-061: CSL Behring/ uniQure

• Concizumab: Novo Nordisk

• FLT180a: Freeline Therapeutics

• SerpinPC: ApcinteX

To know more about Hemophilia B treatment, visit @ Hemophilia B Medications

Hemophilia B Market Drivers

• Popularity of Gene Therapy

• Increased Focus on Prophylactic Treatment

• Development of Novel Treatments with extended half-life

Hemophilia B Market Barriers

• High Cost of Treatment

• Inconvenience and Scheduling Barriers

• Lack of Skilled Healthcare Professionals

Scope of the Hemophilia B Market Report

• Study Period: 2020–2034

• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

• Key Hemophilia B Companies: CSL Behring/uniQure, HEMA Biologics/LFB, Pfizer, Spark Therapeutics, Sanofi, Alnylam Pharmaceuticals, CSL Behring, uniQure, Novo Nordisk, Freeline Therapeutics, ApcinteX, and others

• Key Hemophilia B Therapies: BEQVEZ (fidanacogene elaparvovec-dzkt), HEMGENIX (etranacogene dezaparvovec-drlb), SEVENFACT, Marstacimab (PF-06741086), SPK-9001, Fitusiran, AMT-061, Concizumab, FLT180a, Serpin, and others

• Hemophilia B Therapeutic Assessment: Hemophilia B current marketed and Hemophilia B emerging therapies

• Hemophilia B Market Dynamics: Hemophilia B market drivers and Hemophilia B market barriers

• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies

• Hemophilia B Unmet Needs, KOL’s views, Analyst’s views, Hemophilia B Market Access and Reimbursement

Discover more about therapies set to grab major Hemophilia B market share @ Hemophilia B Treatment Landscape

Table of Contents

1. Hemophilia B Market Report Introduction

2. Executive Summary for Hemophilia B

3. SWOT analysis of Hemophilia B

4. Hemophilia B Patient Share (%) Overview at a Glance

5. Hemophilia B Market Overview at a Glance

6. Hemophilia B Disease Background and Overview

7. Hemophilia B Epidemiology and Patient Population

8. Country-Specific Patient Population of Hemophilia B

9. Hemophilia B Current Treatment and Medical Practices

10. Hemophilia B Unmet Needs

11. Hemophilia B Emerging Therapies

12. Hemophilia B Market Outlook

13. Country-Wise Hemophilia B Market Analysis (2020–2034)

14. Hemophilia B Market Access and Reimbursement of Therapies

15. Hemophilia B Market Drivers

16. Hemophilia B Market Barriers

17. Hemophilia B Appendix

18. Hemophilia B Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare 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.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Gaurav Bora
Email: Send Email
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/

Press Release Distributed by ABNewswire.com

To view the original version on ABNewswire visit: Hemophilia B Market to Show Remarkable Growth Trends from 2024 to 2034, DelveInsight Reports | Pfizer, Spark Therapeutics, Sanofi, Alnylam Pharma, CSL Behring, uniQure, Novo Nordisk

DelveInsight’s “Fragile X Syndrome Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Fragile X Syndrome, historical and forecasted epidemiology as well as the Fragile X Syndrome market trends in the United States, EU4 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

To Know in detail about the Fragile X Syndrome market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Fragile X Syndrome Market Forecast

Some of the key facts of the Fragile X Syndrome Market Report:

• The Fragile X Syndrome market size is anticipated to grow with a significant CAGR during the study period (2020-2034)

• In November 2024, Chris Peetz, the CEO of Mirum, highlighted the company’s strong performance in the third quarter, noting growth across all three commercial medicines, particularly the acceleration of LIVMARLI PFIC sales. He also emphasized significant progress in the clinical pipeline, including the granting of breakthrough designation for volixibat in PBC and the initiation of the EXPAND study for LIVMARLI in other cholestatic pruritus indications. Additionally, the rare genetic neurology franchise is advancing, with a December 28 PDUFA date for chenodiol for CTX and the addition of Phase 2-ready MRM-3379 for Fragile X syndrome.

• In October 2024, Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) will present an extensive update on its pipeline during its Investor Day event today, highlighting new data from its orexin-2 receptor agonist and next-generation pitolisant programs, along with long-term extension data from its pitolisant program for idiopathic hypersomnia. The company will also share updates on its strategic acquisitions in Fragile X syndrome (FXS) and rare epilepsies, which have bolstered its late-stage development pipeline.

• In May 2024, Spinogenix, Inc., a clinical-stage biopharmaceutical company focused on developing first-in-class therapies to restore synapses and enhance the lives of patients globally, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SPG601 for the treatment of Fragile X Syndrome (FXS).

• In April 2024, Spinogenix received approval from the U.S. Food and Drug Administration (FDA) to conduct a Phase II clinical trial of SPG601 for Fragile X Syndrome (FXS). This approval coincides with the European Commission granting orphan drug status to Shionogi’s zatolmilast (BPN14770).

• DelveInsight’s epidemiology model estimates that the total diagnosed prevalent cases of FXS in the 7MM were around 122,000 in 2023. This figure is expected to grow during the forecast period (2024-2034) due to heightened awareness, improved screening, and advancements in genetic testing.

• In 2023, the US had the highest number of diagnosed prevalent cases of FXS, totaling around 71,000 cases, whereas Spain reported the lowest, with nearly 5,900 cases.

• In 2023, among the EU4 and the UK, Germany had the highest number of diagnosed prevalent cases of FXS, with about 9,700 cases, followed by France with around 7,900 cases and the UK with nearly 7,800 cases.

• In 2023, around 42,000 males and 29,000 females in the US were diagnosed with FXS, indicating a greater prevalence in males than in females.

• In Japan, in 2023, there were about 5,700 cases in the 0–11 age group, 2,300 cases in the 12–17 age group, and 5,000 cases among individuals over 18, reflecting differences in prevalence across age groups.

• Key Fragile X Syndrome Companies: Zynerba Pharmaceuticals, Confluence Pharmaceuticals, Tetra Therapeutics, Neuren Pharmaceuticals, Healx, Craig Erickson, Nova Mentis Life Science, and others

• Key Fragile X Syndrome Therapies: Zygel, Acamprosate, BPN14770, Trofinetide, Sulindac (HLX-0201), Gaboxadol, ZYN002, Psilocybin, and others

• The Fragile X Syndrome epidemiology based on gender analyzed that males are affected more as compared to females, in the case of Fragile X Syndrome (FXS)

• The Fragile X Syndrome market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Fragile X Syndrome pipeline products will significantly revolutionize the Fragile X Syndrome market dynamics.

Fragile X Syndrome Overview

Fragile X Syndrome (FXS) is a genetic disorder caused by a mutation in the FMR1 gene on the X chromosome. It is the most common inherited cause of intellectual disability and a leading genetic cause of autism. FXS primarily affects males, but females can also be impacted, though often less severely. Symptoms include developmental delays, learning difficulties, behavioral challenges, anxiety, and physical features like a long face, prominent ears, and flexible joints. There is no cure, but early interventions such as therapy, educational support, and medications can help manage symptoms and improve quality of life.

Get a Free sample for the Fragile X Syndrome Market Report:

https://www.delveinsight.com/report-store/fragile-x-syndrome-fxs-market

Fragile X Syndrome Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Fragile X Syndrome Epidemiology Segmentation:

The Fragile X Syndrome market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total Prevalence of Fragile X Syndrome

• Prevalent Cases of Fragile X Syndrome by severity

• Gender-specific Prevalence of Fragile X Syndrome

• Diagnosed Cases of Episodic and Chronic Fragile X Syndrome

Download the report to understand which factors are driving Fragile X Syndrome epidemiology trends @ Fragile X Syndrome Epidemiology Forecast

Fragile X Syndrome Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Fragile X Syndrome market or expected to get launched during the study period. The analysis covers Fragile X Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Fragile X Syndrome Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Fragile X Syndrome Therapies and Key Companies

• Zygel: Zynerba Pharmaceuticals

• Acamprosate: Confluence Pharmaceuticals

• BPN14770: Tetra Therapeutics

• Trofinetide: Neuren Pharmaceuticals

• Sulindac (HLX-0201): Healx

• Gaboxadol: Craig Erickson

• ZYN002: Zynerba Pharmaceuticals

• Psilocybin: Nova Mentis Life Science

Discover more about therapies set to grab major Fragile X Syndrome market share @ Fragile X Syndrome Treatment Market

Fragile X Syndrome Market Strengths

• Growing Research and development are increasing the demand for FXS treatment.

• Advances in DNA technology, including CRISPRCas9, have been used to correct the expanded CGG repeat in induced pluripotent stem cells. Eventually, it might be possible to add these corrected stem cells into the central nervous systems of those with FXS

Fragile X Syndrome Market Opportunities

• The rise in the prevalence of FXS stimulates the research and development of the drug, as it is likely to provide an appropriate environment for newer products to be profitable.

• Increasing population gives a lucrative opportunity to increase the awareness and innovation of therapeutics to drive FXS market.

Scope of the Fragile X Syndrome Market Report

• Study Period: 2020–2034

• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

• Key Fragile X Syndrome Companies: Zynerba Pharmaceuticals, Confluence Pharmaceuticals, Tetra Therapeutics, Neuren Pharmaceuticals, Healx, Craig Erickson, Nova Mentis Life Science, and others

• Key Fragile X Syndrome Therapies: Zygel, Acamprosate, BPN14770, Trofinetide, Sulindac (HLX-0201), Gaboxadol, ZYN002, Psilocybin, and others

• Fragile X Syndrome Therapeutic Assessment: Fragile X Syndrome current marketed and Fragile X Syndrome emerging therapies

• Fragile X Syndrome Market Dynamics: Fragile X Syndrome market drivers and Fragile X Syndrome market barriers

• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies

• Fragile X Syndrome Unmet Needs, KOL’s views, Analyst’s views, Fragile X Syndrome Market Access and Reimbursement

To know more about Fragile X Syndrome companies working in the treatment market, visit @ Fragile X Syndrome Clinical Trials and Therapeutic Assessment

Table of Contents

1. Fragile X Syndrome Market Report Introduction

2. Executive Summary for Fragile X Syndrome

3. SWOT analysis of Fragile X Syndrome

4. Fragile X Syndrome Patient Share (%) Overview at a Glance

5. Fragile X Syndrome Market Overview at a Glance

6. Fragile X Syndrome Disease Background and Overview

7. Fragile X Syndrome Epidemiology and Patient Population

8. Country-Specific Patient Population of Fragile X Syndrome

9. Fragile X Syndrome Current Treatment and Medical Practices

10. Fragile X Syndrome Unmet Needs

11. Fragile X Syndrome Emerging Therapies

12. Fragile X Syndrome Market Outlook

13. Country-Wise Fragile X Syndrome Market Analysis (2020–2034)

14. Fragile X Syndrome Market Access and Reimbursement of Therapies

15. Fragile X Syndrome Market Drivers

16. Fragile X Syndrome Market Barriers

17. Fragile X Syndrome Appendix

18. Fragile X Syndrome Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare 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.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Gaurav Bora
Email: Send Email
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/

Press Release Distributed by ABNewswire.com

To view the original version on ABNewswire visit: Fragile X Syndrome Market Predicted to See Upsurge Through 2034, Highlights DelveInsight | Novartis, Neuren Pharma, Roche, Seaside Therapeutics, Inc., Marinus Pharma, Lysogene

DelveInsight’s “Guillain-Barré Syndrome Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Guillain-Barré Syndrome, historical and forecasted epidemiology as well as the Guillain-Barré Syndrome market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

To Know in detail about the Guillain-Barré Syndrome market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Guillain-Barré Syndrome Market Forecast

Some of the key facts of the Guillain-Barré Syndrome Market Report:

• The Guillain-Barré Syndrome market size is anticipated to grow with a significant CAGR during the study period (2020-2034)

• In December 2024, Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company focused on developing innovative therapies for patients with severe complement-mediated neuroinflammatory diseases affecting the body, brain, and eyes, has announced positive topline results from a real-world evidence (RWE) study supporting ANX005 as a potential treatment for Guillain-Barré Syndrome (GBS). GBS is an acute neuromuscular condition with no FDA-approved treatments. ANX005, the most advanced targeted immunotherapy in development for GBS, is designed to rapidly block C1q and complement activity with a single dose to halt disease progression during the critical progressive phase.

• In June 2024, Annexon Biosciences announced encouraging topline results from its Phase III clinical trial evaluating ANX005, a C1q-blocking antibody, for the treatment of Guillain-Barré syndrome (GBS). The randomized, placebo-controlled study included 241 participants from Bangladesh and the Philippines, testing two doses of ANX005 (30mg/kg and 75mg/kg). The trial achieved its primary endpoint at week eight with the 30mg/kg dose, showing a 2.4-fold improvement on the GBS-disability scale.

• As per the National Organization of Rare Disorders (NORD) (n.d.), Guillain-Barré Syndrome (GBS) impacts approximately one to two individuals annually per 100,000 population. The precise cause of this condition remains uncertain. It’s noted that around half of those affected experience a gastrointestinal or respiratory infection in the days leading up to the onset of symptoms.

• The outlook for Guillain-Barré Syndrome (GBS) varies depending on its specific form. While some patients achieve full recovery, others may still face challenges with walking six months after the onset of the disease. In severe cases, GBS can unfortunately lead to a fatal outcome.

• Key Guillain-Barré Syndrome Companies: Chafic Karam, Hansa Biopharma AB, Annexon, and others

• Key Guillain-Barré Syndrome Therapies: Efgartigimod Alfa-Fcab, Imlifidase, ANX005, and others

• The Guillain-Barré Syndrome epidemiology based on gender analyzed that Guillain-Barré Syndrome is more common in men than in women

• The Guillain-Barré Syndrome market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Guillain-Barré Syndrome pipeline products will significantly revolutionize the Guillain-Barré Syndrome market dynamics.

Guillain-Barré Syndrome Overview

Guillain-Barré Syndrome (GBS) is a rare neurological disorder in which the body’s immune system attacks the peripheral nerves. It often begins with weakness, tingling, or numbness in the extremities, which can progress to muscle weakness and, in severe cases, paralysis. The exact cause is unknown, but GBS is often triggered by infections, surgeries, or vaccinations. While most patients recover fully with proper treatment, including immunotherapy and supportive care, some may experience long-term nerve damage. Early diagnosis and intervention are crucial to managing symptoms and preventing complications.

Get a Free sample for the Guillain-Barré Syndrome Market Forecast, Size & Share Analysis Report:

https://www.delveinsight.com/report-store/guillain-barre-syndrome-gbs-market

Guillain-Barré Syndrome Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Guillain-Barré Syndrome Epidemiology Segmentation:

The Guillain-Barré Syndrome market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total Prevalence of Guillain-Barré Syndrome

• Prevalent Cases of Guillain-Barré Syndrome by severity

• Gender-specific Prevalence of Guillain-Barré Syndrome

• Diagnosed Cases of Episodic and Chronic Guillain-Barré Syndrome

Download the report to understand which factors are driving Guillain-Barré Syndrome epidemiology trends @ Guillain-Barré Syndrome Epidemiology Forecast

Guillain-Barré Syndrome Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Guillain-Barré Syndrome market or expected to get launched during the study period. The analysis covers Guillain-Barré Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Guillain-Barré Syndrome Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Guillain-Barré Syndrome Therapies and Key Companies

• Efgartigimod Alfa-Fcab: Chafic Karam

• Imlifidase: Hansa Biopharma AB

• ANX005: Annexon, Inc.

Discover more about therapies set to grab major Guillain-Barré Syndrome market share @ Guillain-Barré Syndrome Treatment Landscape

Guillain-Barré Syndrome Market Drivers

• Increased Awareness and Early Diagnosis

• Advancements in Treatment Options

• Government Support and Research Initiatives

• Growing Healthcare Infrastructure

• Rising Incidence of Autoimmune Diseases

• Increased Focus on Orphan Diseases

• Pipeline Drugs

Guillain-Barré Syndrome Market Barriers

• High Treatment Costs

• Lack of Standardized Treatment Protocols

• Limited Awareness in Developing Regions

• Side Effects of Current Treatments

• Regulatory Hurdles

• Low Market Penetration of Novel Therapies

• Limited Patient Pool

Scope of the Guillain-Barré Syndrome Market Report

• Study Period: 2020–2034

• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

• Key Guillain-Barré Syndrome Companies: Chafic Karam, Hansa Biopharma AB, Annexon, Inc., and others

• Key Guillain-Barré Syndrome Therapies: Efgartigimod Alfa-Fcab, Imlifidase, ANX005, and others

• Guillain-Barré Syndrome Therapeutic Assessment: Guillain-Barré Syndrome current marketed and Guillain-Barré Syndrome emerging therapies

• Guillain-Barré Syndrome Market Dynamics: Guillain-Barré Syndrome market drivers and Guillain-Barré Syndrome market barriers

• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies

• Guillain-Barré Syndrome Unmet Needs, KOL’s views, Analyst’s views, Guillain-Barré Syndrome Market Access and Reimbursement

To know more about Guillain-Barré Syndrome companies working in the treatment market, visit @ Guillain-Barré Syndrome Clinical Trials and Therapeutic Assessment

Table of Contents

1. Guillain-Barré Syndrome Market Report Introduction

2. Executive Summary for Guillain-Barré Syndrome

3. SWOT analysis of Guillain-Barré Syndrome

4. Guillain-Barré Syndrome Patient Share (%) Overview at a Glance

5. Guillain-Barré Syndrome Market Overview at a Glance

6. Guillain-Barré Syndrome Disease Background and Overview

7. Guillain-Barré Syndrome Epidemiology and Patient Population

8. Country-Specific Patient Population of Guillain-Barré Syndrome

9. Guillain-Barré Syndrome Current Treatment and Medical Practices

10. Guillain-Barré Syndrome Unmet Needs

11. Guillain-Barré Syndrome Emerging Therapies

12. Guillain-Barré Syndrome Market Outlook

13. Country-Wise Guillain-Barré Syndrome Market Analysis (2020–2034)

14. Guillain-Barré Syndrome Market Access and Reimbursement of Therapies

15. Guillain-Barré Syndrome Market Drivers

16. Guillain-Barré Syndrome Market Barriers

17. Guillain-Barré Syndrome Appendix

18. Guillain-Barré Syndrome Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare 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.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Gaurav Bora
Email: Send Email
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/

Press Release Distributed by ABNewswire.com

To view the original version on ABNewswire visit: Guillain-Barré Syndrome Market to Reach New Heights in Growth by 2034, DelveInsight Predicts | Chafic Karam, Hansa Biopharma AB, Annexon