Acute Porphyria – Pipeline Review, 2024

Enzyme deficiencies in heme production pathways lead to porphyria. Inborn errors of metabolism or infectious agents and exposure to environmental toxins may lead to enzyme deficiency. Based on their predominant symptoms, porphyria cane be divided into cutaneous and acute categories. Symptoms like abdominal pain, autonomic instability, neuropathy, and psychosis are observed in patients suffering from acute porphyria whereas photosensitive lesions on the skin can be caused in patients suffering from cutaneous porphyria.f

Current treatment of porphyria includes decreasing heme synthesis and reducing the production of porphyrin precursors using glucose. As per the U.K. guidelines, 5% glucose in 0.9% sodium chloride solution is recommended to be infused intravenously in patients. Hematin is used for the treatment of patients experiencing severe attacks with acute neurologic symptoms.

Pharmaceutical companies have focussed on studying and developing new methods for treatment of porphyria. For instance; givosiran (ALN-AS1), which is being studied by Alnylam Pharmaceuticals, is currently in phase-2 clinical trials for the study on the long-term safety and clinical activity of subcutaneously administered drug.

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At present more than 50% of the pipeline candidates for Acute Porphyria are in the preclinical and phase 1 stage. Majority of the studies have been sponsored by the pharmaceutical companies.

Report Description

The report on ‘Acute Porphyria – Pipeline Review, 2024’ provides a comprehensive overview of the drugs that are in the R&D pipeline by indication or molecule for Acute Porphyria. The report provides a thorough analysis of the distribution of the pipeline products by clinical trial stage, indication, company, therapy area and details such as clinical trial stage, sponsor, description on every product in the pipeline. Products in the preclinical and clinical stage along with dormant & discontinued pipeline candidates are included in the report. The report also covers additional insights such as epidemiology overview and current market scenario for Acute Porphyria.

The report on ‘Acute Porphyria – Pipeline Review, 2024’, which is built by following a robust research methodology involving primary interviews and desk research, provides a complete overview of the R&D activity and pipeline products to assist companies in developing growth strategies and identifying emerging players.

Report Scope

• Thorough assessment of the pipeline products by areas such as development stage; route of administration; drug class; indication; sponsor; molecule type and drug target
• Comprehensive profiles of the pipeline products with details such as company overview; product description; R&D status; development activities; mechanism of action; molecule type; development stage; indications; funding and route of administration
• Overview of dormant and discontinued pipeline products
• Key insights in relation to the epidemiology of conditions being treated by the pipeline products and overview of the addressable or current market for the pipeline products
• Overview of the latest developments; news articles, press releases and relevant conferences

Report Methodology

• All pipeline reports are built through the analysis of data primarily collected through credible desk research sources. Secondary research is supplemented by interviews conducted with key opinion leaders.
• Desk research sources includes global and regional clinical trials databases; annual reports, websites, press releases & investor presentations of companies; white papers; news articles; reports published by industry associations; articles/reports published on databases such as NCBI, ResearchGate; internal databases

Reasons to Buy this Report

• Develop effective growth strategies based on comprehensive overview of the R&D activity and pipeline products for Acute Porphyria
• Identify emerging players or competition in the market based on pipeline products and develop strategies to counter the emergence of these players
• Identify the focus of leading players in relation to R&D for Acute Porphyria
• Identify potential companies from a partnership or acquisition point of view based on current synergy in R&D activities or strategies to diversify R&D focus to drive growth in business
• Analyse the reasons behind dormant and discontinued products to make changes in the R&D focus if necessary