Several novel and innovative therapeutic approaches, both small molecule and biologics, are being currently investigated at various phases of development in order to avoid the beginning of disease-induced attacks and other long-term effects of porphyria, noted the analysts.
Porphyria is a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme (which helps bind oxygen in the blood). Any anomaly caused by genetic or acquired abnormalities in heme biosynthesis, which is produced majorly in bone marrow and liver, can result in toxicity.
The publication indicates that the prevalence of porphyria still remains unknown. Several treatment options such as gene therapy, proteasome inhibition and pharmacologic chaperones are currently being investigated among various other targeted treatment options.
Clinical trials, approvals
More than 70% of those treatment candidates are currently under clinical evaluation, said the analysts. More than 30% of the overall trials are phase I studies.
However, three therapies: Panhematin, Givlaari, and Scenesse have already been approved for the treatment of different types of porphyria, they reported.
Around 50% of the therapeutics targeting this rare disorder are being developed as biologics, the majority of which are being designed for administration via the intravenous route, finds the Roots Analysis team.
Development hub
North America has emerged as a key hub for the development of porphyria therapies, featuring the presence of 65% developers, according to this report.