Hypophosphatasia– Pipeline Review, 2019

Hypophosphatasia is a rare genetic disorder that affects the development of bones and teeth. Hypophosphatasia weakens the bones in the body and causes skeletal abnormalities. Hypophosphatasia is also called deficiency of alkaline phosphatase or phosphoethanolaminuria. The symptoms of hypophosphatasia vary widely. Affected infants are born with short limbs, respiratory problems, abnormally shaped chest, and soft skull bones. Symptoms that are identified in children and adult patients are less severe than those that appear in infants.

Current treatment of hypophosphatasia includes use of Asfotase alpha which has been approved by the Food and Drug Administration in 2015. Asfotase alpha is the first approved medication for the treatment of perinatal, infantile, and juvenile-onset hypophosphatasia. Asfotase alpha is a recombinant human alkaline phosphatase available for long term enzyme replacement to treat the bone manifestation of the disease. Another treatment is used to improve the symptoms of hypophosphatasia includes drugs like NSAIDs and Vitamin B6.

Research Institutes and pharmaceutical companies have focussed on studying and developing new methods for the treatment of hypophosphatasia. For instance; Asfotase alpha is being studied by Alexion Pharmaceuticals is in phase 2 of clinical trials for the study to evaluate its pharmacokinetic and pharmacodynamics in adult participants with pediatric-onset hypophosphatasia.

Currently, more than 50% of the pipeline candidates for hypophosphatasia are in phase 2 of the clinical trials. Majority of the studies is being sponsored by the pharmaceutical companies.

REPORT DESCRIPTION

The report on ‘Hypophosphatasia– Pipeline Review, 2019’ provides a comprehensive overview of the drugs that are in the R&D pipeline by indication or molecule for hypophosphatasia. 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 hypophosphatasia.

The report on ‘Hypophosphatasia – Pipeline Review, 2019’, 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 hypophosphatasia


• 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 hypophosphatasia


• 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