Exploring Growth Opportunities in the Hypophosphatasia Treatment Market: Navigating the Ultra-rare Disease Landscape
DelveInsight estimates that the total market size for hypophosphatasia in the 7MM was nearly $1 billion in 2023, and it is expected to grow at a CAGR of 7.2% by 2034. This growth is anticipated to be driven by the introduction of new therapies, such as AstraZeneca’s ALXN1850 (efzimfotase alfa), AM Pharma’s Ilofotase alfa, and others, and increased disease awareness.
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Newsletter/Whitepaper
Currently, there are no FDA-approved treatments for acute concussion. The current standard of care is rest, followed by a gradual return to normal activity. Off-label therapies are used to manage TBI. In July 2024, the first cell therapy, AKUUGO, was approved in Japan for improving chronic motor paralysis resulting from TBI. It obtained conditional and time-limited marketing approval, which was further granted full approval in October 2025. The company is planning to launch AKUUGO in the Japanese market in 2026.
A mid pharma client based in Germany, involved in late-stage development of a therapy for PID, with effective marketing and commercialisation presence in Europe, wanted to assess the launch readiness of competitors involved in the same arena for strategic positioning of their product. They wished to identify and evaluate the launch timelines, geographies of launch, First Launch company, commercial readiness, and salesforce of competitors.
A large pharma client was working to develop their cyclophosphamide API and FD and was interested to assess competitive products in the same domain and area to provide benchmarking of their asset in comparison to their competitors The client requested periodic reports (Quarterly) on the developmental milestones of competitor companies, thorough assessment of the competitor products through Secondary and Primary CI inputs.
A mid-sized pharma client was working to develop their asset portfolio and was interested in assessing best possible asset for atrial fibrillation for future development and licensing needs. The client requested evidence-based priorities in assets and products for Phase II development in atrial fibrillation, through potential licensing collaborations. Also, the client wanted to prioritise research efforts to determine the best possible asset to leverage their research portfolio.
A small pharmaceutical client based at Sweden, Europe with capabilities around a neonatal mesenchymal cells involved DelveInsight to identify a licensing partner for their stem cells product, from across the globe. They wished to identify a potential list of companies, to become a fit for their neonatal high quality MSCs and can prove to be a good business partner as a licensee for their proprietary “API” of the stem cell therapy domain.
A large client based in the USA, involved in drug delivery devices, with effective marketing and commercialisation presence across the globe, wanted to in-license potential dMTS based drug delivery asset from companies involved in microneedle technology research for delivering therapeutic products. They wished to identify and assess the technological competencies, clinical capabilities and commercial readiness of potential companies with a novel and non-invasive way of delivering drugs, which can include a variety of drugs/vaccines in their patch and successfully develop the product for commercial purposes, were of client’s interest.
A medium pharmaceutical client based in the United States with biosimilar commercial capabilities involved DelveInsight to identify commercial processes and strategies to launch their biosimilar in Europe through Competitive Assessment of their EU based competitors in the same area of interest. They wished to identify manufacturing sites, logistic operations, distribution network, salesforce assessment and their sales targets.