{"id":666750,"date":"2023-09-07T19:56:01","date_gmt":"2023-09-07T19:56:01","guid":{"rendered":"https:\/\/www.abnewswire.com\/pressreleases\/?p=666750"},"modified":"2023-09-07T19:56:01","modified_gmt":"2023-09-07T19:56:01","slug":"niemannpick-disease-type-c-npc-pipeline-analysis-2023-covering-clinical-trials-emerging-therapies-fda-ema-and-pmda-approvals-competitive-landscape-mandos-orphazyme-intrabio-som-biotech","status":"publish","type":"post","link":"https:\/\/www.abnewswire.com\/pressreleases\/niemannpick-disease-type-c-npc-pipeline-analysis-2023-covering-clinical-trials-emerging-therapies-fda-ema-and-pmda-approvals-competitive-landscape-mandos-orphazyme-intrabio-som-biotech_666750.html","title":{"rendered":"Niemann-Pick Disease Type C (NPC) Pipeline Analysis (2023) Covering Clinical Trials, Emerging Therapies, FDA, EMA, and PMDA Approvals, Competitive Landscape | Mandos, Orphazyme, IntraBio, SOM Biotech"},"content":{"rendered":"<div style=\"float:right; width:250px; padding:8px 10px 10px 10px;\">\n<div><a href=\"https:\/\/www.abnewswire.com\/uploads\/1585677425.jpeg\" style=\"border:none !important;\" target=\"_blank\" rel=\"nofollow\" ><img loading=\"lazy\" decoding=\"async\" class=\"alignnone size-medium wp-image-29\" title=\"Niemann-Pick Disease Type C (NPC) Pipeline Analysis (2023) Covering Clinical Trials, Emerging Therapies, FDA, EMA, and PMDA Approvals, Competitive Landscape | Mandos, Orphazyme, IntraBio, SOM Biotech\" src=\"https:\/\/www.abnewswire.com\/uploads\/1585677425.jpeg\" alt=\"Niemann-Pick Disease Type C (NPC) Pipeline Analysis (2023) Covering Clinical Trials, Emerging Therapies, FDA, EMA, and PMDA Approvals, Competitive Landscape | Mandos, Orphazyme, IntraBio, SOM Biotech\" width=\"225\" height=\"225\" style=\"padding:0px 0px 10px 10px; border:0 solid !important;\" \/><\/a><\/div>\n<div class=\"quotes\">\n<div>Delveinsight Business Research LLP<\/div>\n<\/div>\n<\/div>\n<div style=\"font-style:italic; padding:8px 0px;\">As per DelveInsight\u2019s assessment, globally, about 13+ key pharma and biotech companies are working on 13+ pipeline drugs in the Niemann-Pick Disease Type C therapeutics landscape based on different Routes of Administration (ROA), Mechanism of Action (MOA), and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.<\/div>\n<p style=\"text-align: justify;\" dir=\"ltr\">&ldquo;<strong>Niemann-Pick Disease Type C (NPC) Pipeline Insight, 2023<\/strong>&rdquo; report by DelveInsight outlines a comprehensive assessment of the present clinical\/non-clinical development activities and growth prospects across the Niemann-Pick Disease Type C Market.&nbsp;<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">The Niemann-Pick Disease Type C Pipeline report embraces in-depth commercial, regulatory, and Niemann-Pick Disease Type C clinical trial assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the emerging Niemann-Pick Disease Type C drugs, including the mechanism of action, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers, acquisition, funding, designations, and other product-related details.<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/report-store\/niemann-pick-type-c-disease-pipeline-insight?utm_source=abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=rpr\" target=\"_blank\"><strong>Niemann-Pick Disease Type C Pipeline Analysis<\/strong><\/a><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>The report provides insights into:&nbsp;<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li dir=\"ltr\">\n<p dir=\"ltr\">The report provides detailed insights into the <strong>emerging therapies for Niemann-Pick Disease Type C treatment<\/strong> and the aggregate therapies developed by major pharma companies.<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">It accesses the different <strong>Niemann-Pick Disease Type C therapies<\/strong> segmented into early-stage, mid-stage, and late-stage of clinical development.<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">It outlines the major <strong>Niemann-Pick Disease Type C companies<\/strong> involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">The report evaluates the <strong>Niemann-Pick Disease Type C drugs<\/strong> that are under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.&nbsp;<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the <strong>Niemann-Pick Disease Type C therapeutic market.<\/strong><\/p>\n<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><img decoding=\"async\" style=\"display: block; margin-left: auto; margin-right: auto;\" src=\"https:\/\/www.abnewswire.com\/uploads\/8e9e1c43f2ecfdd33939b02cdb2b2ada.jpg\" alt=\"\" \/><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Niemann-Pick Disease Type C Therapeutics Landscape<\/strong><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">Treatment of NPC requires the coordinated efforts of a team of specialists. Pediatricians, neurologists, ophthalmologists, pulmonologists, gastroenterologists, and other healthcare professionals may need to systematically and comprehensively plan an affected child&rsquo;s treatment. Psychosocial support for the entire family is essential as well. Genetic counseling benefits affected individuals and their families. Current treatment is directed toward the specific symptoms apparent in each individual.&nbsp;<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">There is no cure for NPC, although patients benefit from palliative treatments. Occupational therapy can be used to help with posture, speech, and movement. Physical therapy is an important part of treatment to help maintain mobility as long as possible.<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">The only approved therapy in Europe and Japan is ZAVESCA\/BRAZAVES [miglustat], a substrate reduction therap developed by Actelion Pharmaceuticals. It has been denied approval for the treatment of NPC, by the US Food and Drug Administration (FDA) on the grounds of insufficient data. The US FDA requested for more data, which would take years to get, and almost impossible to satisfy the high standards of the FDA, mainly restrained by the limited or lack of patient population that could be recruited in the trials.<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">There are approx. <strong>13+ key companies<\/strong> are developing therapies for Niemann-Pick Disease Type C. Currently, <strong>Orphazyme<\/strong> is leading the therapeutics market with its Niemann-Pick Disease Type C drug candidates in the most advanced stage of clinical development.<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Niemann-Pick Disease Type C Companies Actively Working in the Therapeutic Market Include:<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li dir=\"ltr\">\n<p dir=\"ltr\">Mandos LLC<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Cyclo Therapeutics<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Orphazyme<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">IntraBio Inc<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Synaptogenix<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Evox Therapeutics<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">SOM Biotech<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Sarepta Therapeutics<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">StrideBio<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">ENDECE<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Amicus Therapeutics<\/p>\n<\/li>\n<\/ul>\n<p style=\"text-align: justify;\" dir=\"ltr\">And Many Others<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Emerging and Marketed Niemann-Pick Disease Type C Drugs Covered in the Report Include:<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li dir=\"ltr\">\n<p dir=\"ltr\">Arimoclomol: Orphazyme<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Adrabetadex: Mandos, LLC<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">VTS-270: Mallinckrodt<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Arimoclomol: Orphazyme<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Trappsol Cyclo: CTD Holdings, Inc.<\/p>\n<\/li>\n<\/ul>\n<p style=\"text-align: justify;\" dir=\"ltr\">And Many Others<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Niemann-Pick Disease Type C Companies Working in the Market:<\/strong><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/sample-request\/niemann-pick-type-c-disease-pipeline-insight?utm_source=abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=rpr\" target=\"_blank\"><strong>https:\/\/www.delveinsight.com\/sample-request\/niemann-pick-type-c-disease-pipeline-insight<\/strong><\/a><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>The report covers the emerging products under different phases of clinical development like &#8211;<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li dir=\"ltr\">\n<p dir=\"ltr\">Late-stage products (Phase III)<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Mid-stage products (Phase II)<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Early-stage product (Phase I)&nbsp;<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Pre-clinical and Discovery stage candidates<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Discontinued and inactive candidates<\/p>\n<\/li>\n<\/ul>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Route of Administration<\/strong><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Niemann-Pick Disease Type C pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Niemann-Pick Disease Type C Products have been categorized under various ROAs such as<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li dir=\"ltr\">\n<p dir=\"ltr\">Oral<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Parenteral<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Intravenous<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Subcutaneous<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Topical<\/p>\n<\/li>\n<\/ul>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Molecule Type<\/strong><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Niemann-Pick Disease Type C Products have been categorized under various Molecule types such as<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li dir=\"ltr\">\n<p dir=\"ltr\">Monoclonal Antibody<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Peptides<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Polymer<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Small molecule<\/p>\n<\/li>\n<li dir=\"ltr\">\n<p dir=\"ltr\">Gene therapy<\/p>\n<\/li>\n<\/ul>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Learn How the Niemann-Pick Disease Type C Treatment Outlook will Evolve with the Ongoing Clinical &amp; Commercial Activities in the Therapeutic Market @&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/sample-request\/niemann-pick-type-c-disease-pipeline-insight?utm_source=abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=rpr\" target=\"_blank\"><strong>https:\/\/www.delveinsight.com\/sample-request\/niemann-pick-type-c-disease-pipeline-insight<\/strong><\/a><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Table of Content (TOC)<\/strong><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">1. Report Introduction<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">2. Executive Summary<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">3. Niemann-Pick Disease Type C Treatment Patterns<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">4. Niemann-Pick Disease Type C &#8211; DelveInsight&#8217;s Analytical Perspective<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">5. Therapeutic Assessment<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">6. Niemann-Pick Disease Type C Late Stage Products (Phase-III)<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">7. Niemann-Pick Disease Type C Mid-Stage Products (Phase-II)<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">8. Niemann-Pick Disease Type C Early Stage Products (Phase-I)<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">9. Pre-clinical Products and Discovery Stage Products<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">10. Inactive Products<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">11. Dormant Products<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">12. Niemann-Pick Disease Type C Discontinued Products<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">13. Niemann-Pick Disease Type C Product Profiles<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">14. Major Niemann-Pick Disease Type C Companies in the Market<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">15. Key Products in the Niemann-Pick Disease Type C Therapeutics Segment<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">16. Dormant and Discontinued Products<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">17. Niemann-Pick Disease Type C Unmet Needs<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">18. Niemann-Pick Disease Type C Future Perspectives<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">19. Niemann-Pick Disease Type C Analyst Review&nbsp;&nbsp;<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">20. Appendix<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">21. Report Methodology<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><em>*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.<\/em><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Download Sample PDF to Explore the Key Offerings of the Report @<\/strong><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/sample-request\/niemann-pick-type-c-disease-pipeline-insight?utm_source=abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=rpr\" target=\"_blank\"><strong>https:\/\/www.delveinsight.com\/sample-request\/niemann-pick-type-c-disease-pipeline-insight<\/strong><\/a><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">&nbsp;<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">&nbsp;<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>About DelveInsight<\/strong><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.<\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\"><strong>Other Trending Healthcare Reports By DelveInsight<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong><a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/report-store\/reactive-airway-disease-market\">Reactive Airway Disease Market<\/a><\/strong><\/p>\n<p style=\"text-align: justify;\" dir=\"ltr\">&#8220;Reactive Airway Disease Market Insights, Epidemiology, and Market Forecast-2032&#8221; report deliver an in-depth understanding of the historical and forecasted epidemiology as well as the Reactive Airway Disease market size, share, trends, and growth analysis in the seven major markets (i.e. the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom) and Japan. Moreover, it also covers the current treatment practices, emerging drugs, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the Reactive Airway Disease market.<\/p>\n<p><span style='font-size:18px !important;'>Media Contact<\/span><br \/><strong>Company Name:<\/strong> <a href=\"https:\/\/www.abnewswire.com\/companyname\/delveinsight.com_.html\" rel=\"nofollow\">DelveInsight Business Research LLP<\/a><br \/><strong>Contact Person:<\/strong> Shruti Thakur<br \/><strong>Email:<\/strong> <a href=\"https:\/\/www.abnewswire.com\/email_contact_us.php?pr=niemannpick-disease-type-c-npc-pipeline-analysis-2023-covering-clinical-trials-emerging-therapies-fda-ema-and-pmda-approvals-competitive-landscape-mandos-orphazyme-intrabio-som-biotech\" rel=\"nofollow\">Send Email<\/a><br \/><strong>Phone:<\/strong> 09650213330<br \/><strong>Address:<\/strong>304 S. Jones Blvd #2432  <br \/><strong>City:<\/strong> Las Vegas<br \/><strong>State:<\/strong> NV<br \/><strong>Country:<\/strong> United States<br \/><strong>Website:<\/strong> <a href=\"https:\/\/www.delveinsight.com\/market-research\" target=\"_blank\" rel=\"nofollow\">https:\/\/www.delveinsight.com\/market-research<\/a><\/p>\n<p><object type=\"text\/html\" data=\"https:\/\/www.delveinsight.com\/market-research\" style=\"width:900px; height:400px;\"><\/object><img decoding=\"async\" src=\"https:\/\/www.abnewswire.com\/press_stat.php?pr=niemannpick-disease-type-c-npc-pipeline-analysis-2023-covering-clinical-trials-emerging-therapies-fda-ema-and-pmda-approvals-competitive-landscape-mandos-orphazyme-intrabio-som-biotech\" alt=\"\" width=\"1px\" height=\"1px\" \/><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Delveinsight Business Research LLP As per DelveInsight\u2019s assessment, globally, about 13+ key pharma and biotech companies are working on 13+ pipeline drugs in the Niemann-Pick Disease Type C therapeutics landscape based on different Routes of Administration (ROA), Mechanism of Action &hellip; <a href=\"https:\/\/www.abnewswire.com\/pressreleases\/niemannpick-disease-type-c-npc-pipeline-analysis-2023-covering-clinical-trials-emerging-therapies-fda-ema-and-pmda-approvals-competitive-landscape-mandos-orphazyme-intrabio-som-biotech_666750.html\">Continue reading <span class=\"meta-nav\">&rarr;<\/span><\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[425,426,417,406,404],"tags":[],"class_list":["post-666750","post","type-post","status-publish","format-standard","hentry","category-Finance","category-Financial-Market","category-Marketing-Sales","category-Pharmaceuticals-Biotech","category-US"],"_links":{"self":[{"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/posts\/666750","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/comments?post=666750"}],"version-history":[{"count":0,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/posts\/666750\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/media?parent=666750"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/categories?post=666750"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/tags?post=666750"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}