Lambert-Eaton Myasthenic Syndrome (LEMS) Markets, 2017-2030 - ResearchAndMarkets.com

The "Lambert-Eaton Myasthenic Syndrome (LEMS) - Market Insights, Epidemiology, and Market Forecast-2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

The report delivers an in-depth understanding of the LEMS, historical and forecasted epidemiology as well as the LEMS market trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

The report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM LEMS market size from 2017 to 2030. The report also covers current LEMS treatment practice/algorithm, market drivers, market barriers, SWOT analysis, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Cases of LEMS, Total Diagnosed Prevalent Cases of LEMS, Gender-specific Diagnosed Prevalent Cases of LEMS, Type-specific Diagnosed Prevalent Cases of LEMS, and Diagnosed Prevalent Cases of LEMS by Malignancy scenario of LEMS in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom) and Japan from 2017 to 2030.

Key Findings

• Total prevalent cases of LEMS in the 7MM was found to be 5,977 in 2017.
• Based on the etiology, LEMS is divided into two types; paraneoplastic and idiopathic. There were a total 1,978 diagnosed prevalent cases of LEMS in the United States in 2017, out of which 1,154 accounted for paraneoplastic and 824 cases accounted for idiopathic.
• As per the assessments, in the 7MM, the majority of cases of LEMS are paraneoplastic form as compared to idiopathic form.
• Among the two forms of LEMS, the paraneoplastic form constitutes more than half of the cases and is mostly associated with intrathoracic neoplasms. Most of the cases were seen with small cell lung cancer (SCLC); other subtypes of lung cancer are extremely rare.
• Among the EU-5 countries, Germany witnessed the highest prevalent population accounting for 6.93% of the total prevalent cases in 7MM, followed by France and the United Kingdom. On the other hand, Spain witnessed the lowest number of prevalent cases in 2017.
• Japan was assessed with 21.32% of the total prevalent population, with 1,274 cases observed in 2017.

Drug Chapters

The drug chapter segment of the LEMS report encloses the detailed analysis of LEMS marketed drugs, mid-phase, and late-stage pipeline drugs. It also helps to understand the LEMS clinical trial details, expressive pharmacological action, agreements and collaborations, approval, and patent details of each included drug and the latest news and press releases.

Marketed Drugs

Firdapse: Catalyst Pharmaceutical

Firdapse (amifampridine) is a potassium channel blocker used to treat LEMS in adults. The active ingredient of Firdapse is amifampridine phosphate, which is a voltage-gated potassium channel blocker. Amifampridine phosphate is described chemically as 3, 4-diaminopyridine phosphate. Each Firdapse tablet contains 10 mg amifampridine (equivalent to 18.98 mg amifampridine phosphate). The drug was developed by BioMarin Pharmaceutical and is marketed by Catalyst Pharmaceutical, which secured the marketing rights to Firdapse in North America.

In June 2019, Catalyst Pharmaceutical filed a lawsuit against the US FDA over the regulatory agency's decision to approve a rival treatment for its newly-approved LEMS. Documents released in court, including partially redacted emails from the FDA, appear to show that issues regarding the list price for Firdapse (amifampridine). The lawsuit alleges that the approval of Ruzurgi, a rival drug from privately held Jacobus Pharmaceutical Co, in May 2019, violated provisions of FDA regulations and Catalyst's rights to exclusivity for its drug, Firdapse.

Catalyst recently announced plans to expand sales to Japan. They recently had talks with the Japanese Ministry of Health, Labor and Social Affairs (MHLW) to get Firdapse approved in Japan.

Ruzurgi: Jacobus Pharmaceutical Company Inc.

Ruzurgi (amifampridine) is a potassium channel blocker indicated for the treatment of LEMS in patients 6 years to less than 17 years of age. It is supplied as a tablet for oral administration. Ruzurgi is designed to prolong signals that are released from nerves to allow greater stimulation of the muscle. Ruzurgi works in a similar mechanism to Firdapse (amifampridine phosphate), which was approved in 2018 for the treatment of adults with LEMS. The drug was initially developed as a phosphate salt of amifampridine by Assistance Publique Hpitaux de Paris. It was later developed and commercialized by various companies, including BioMarin Pharmaceutical, Catalyst Pharmaceuticals, and Jacobus Pharmaceutical.

Market Outlook

The therapeutic market of LEMS in the seven major markets was assessed to be USD 59.43 million in 2017 (With Base Case Scenario* and Downside Scenario**) and is expected to grow during the study period (2017-2030).

Currently, there is no cure for LEMS and treatment is mainly symptomatic. This includes 3, 4-diaminopyridine phosphate (DAP), which is usually well-tolerated and effective. In some patients, the combination of pyridostigmine with 3, 4-DAP has been suggested to have an additional positive effect. If symptomatic treatment is insufficient, immunosuppressive therapy with prednisone, alone or in combination with azathioprine, can achieve long-term control of the disorder. Plasmapheresis and high dose administration of intravenous immunoglobulins (IVIGs) have a short effect. An effective treatment against any tumor present is mandatory, both to control the tumor and to improve the clinical symptoms of LEMS.

So far, there are only two FDA approved the medication for LEMS, i.e., Firdapse and Ruzurgi. The FDA's approval of Firdapse is a potentially transformative milestone in the lives of patients in the US suffering from LEMS, as it now gives adult LEMS patients access to a new first-in-class-therapy. Firdapse is approved in the US and the European Union for use by patients with LEMS.

After the approval of Firdapse, Ruzurgi is the second drug to be approved in the US for the treatment of LEMS and first approved the medication for the treatment of LEMS in patients 6 years to less than 17 years of age. Despite being a potential therapy in the treatment, a lawsuit has been filed by Catalyst Pharma against the US FDA and several related parties challenging the recent approval of an NDA and related drug labeling for Ruzurgi for the treatment of LEMS in pediatric patients. The lawsuit alleges that the approval of Ruzurgi, Catalyst's rival medicine from privately held Jacobus Pharmaceutical, in May violated provisions of US FDA's regulations and Catalyst's rights to exclusivity for its drug, Firdapse. Both drugs are approved to treat LEMS, but Firdapse was approved for adults, while Jacobus' drug was for children.

Presently there are no approved therapies of LEMS in Japan, Catalyst Pharma announced the plan to expand sales of Firdapse in Japan. They recently had talks with the Japanese Ministry of Health, Labor and Social Affairs (MHLW) to get Firdapse approved in Japan. About two years ago, the Japanese government approved Firdapse as a primary drug for the MHLW and actively asked companies to develop and submit an NDA for the drug. Once Catalyst and MHLW have reached agreement on what will be required to file this NDA in Japan, an update of the approval process for filing an NDA in Japan will be released.

*Base Case Scenario: With the Base Case Scenario, the research analysts estimate that Ruzurgi will not be launch in the market because Catalyst has filed a suit against Jacobus' drug (Ruzurgi). Therefore, it is estimated that Firdapse will cover a decent market in the upcoming years without any competition

**Downside Scenario: With Downside Scenario, the publisher is expecting the launch of Ruzurgi in the United States in the second half of 2020, which will compete and likely to take the market share of Firdapse.

Key Findings

• In 2017, the market size of LEMS in the 7MM was USD 59.43 million.
• Among the 7MM, the United States accounted for the majority of the market size with USD 32.85 million in 2017.
• The current market size of LEMS is entirely dependent on symptomatic treatment regimens, includes Firdapse, 3, 4-diaminopyridine (base), pyridostigmine, immunoglobulin, and others). Others include guanidine, acetylcholinesterase (AChE) inhibitors, prednisone, azathioprine, cyclosporine, and plasmapheresis.
• Firdapse (Catalyst Pharmaceuticals) is the only approved therapy for the treatment of LEMS in adults in the United States and EU5.
• Another Molecule Ruzurgi (Jacobus Pharmaceuticals), is approved by the US FDA in May 2019, for the treatment of patients with LEMS age six to less than 17 years. However, In June 2019, Catalyst filed its lawsuit in the US District Court for the Southern District of Florida and is seeking a reversal of FDA's decision.
• The publisher estimates that Japan has the second-highest market size after the United States in 2017, which accounted for USD 9.59 million.

Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the LEMS market or expected to get launched in the market during the study period 2017-2030. The analysis covers LEMS market uptake by drugs; patient uptake by therapies; and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows the comparison of the drugs based on market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Reimbursement Scenario

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In the report, we consider reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.

Competitive Intelligence Analysis

The publisher performs competitive and market Intelligence analysis of the LEMS market by using various competitive intelligence tools that include-SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

• The report covers the descriptive overview of LEMS, explaining its causes, signs and symptoms, risk factors, pathophysiology, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into the LEMS epidemiology and treatment.
• Additionally, an all-inclusive account of both the current and emerging therapies for LEMS is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
• A detailed review of the LEMS market; historical and forecasted is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MM LEMS market.

Report Highlights

• The increasing prevalence of the disease will positively drive the LEMS market.
• The companies and academics are working to assess challenges and seek opportunities that could influence LEMS R&D.
• Major players are involved in commercializing therapies for LEMS. Launch of the therapies will significantly impact the LEMS market.
• In-depth analysis of the different emerging trends and comparative analysis with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/xt23o0

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