The UK Hypophosphatasia (HPP) Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
Hypophosphatasia (HPP) is also known as Rathbun disease is an ultra-rare metabolic genetic disorder characterized by the abnormal development of bones and teeth due to defective mineralization, a process by which bones and teeth use minerals such as calcium and phosphorus. It caused by mutations in the tissue nonspecific alkaline phosphatase (TNSALP) gene, also called the ALPL gene. Such mutations lead to low activity of this enzyme that should be breaking down a chemical called inorganic pyrophosphate that blocks mineralization, profound muscle weakness, seizures, respiratory failure, and premature death.
According to a report, the disease is more common in older children and adults, affecting 1 per 6,370 of the population. HPP affects males and females in equal numbers. Juvenile and adult-onset forms of HPP have much lower mortality rates than those appearing in infancy, where 100% of those diagnosed in the first six months of their lives die before they reach one year. The total cost per person per year of treatment is a whopping £366,912 (assuming an average weight of 19.3 kg and 100% adherence). It seems to be rare in individuals with black ancestry.
Market Growth Drivers
The HPP community is helping to bring programs that will bring new and treatment options to people with rare diseases. Currently, clinical trials are conducted, wherein a large number of organizations are actively participating in several research programs to develop novel therapies for infantile and juvenile-onset hypophosphatasia treatment.
Introduction of several novel therapies, breakthrough therapy designation, orphan drug designation for rare diseases, financial incentives and grants by the government, clinical
studies on the safety and efficacy of products are expected to be the biggest hit in the UK infantile and juvenile-onset hypophosphatasia market over the forecast period.
Gaps in making hypophosphatasia (HPP) health priority, patients’ non-adherence to therapy regime, high therapy cost, a smaller number of approved therapies, failure in a clinical trial at the late phase are some of the factors restraining the infantile and juvenile-onset hypophosphatasia treatment market.