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
Porphyria is a rare disorder that is characterized by excessive accumulation of porphyrin, a compound that aids in the formation of heme (an essential part of hemoglobin that helps carry oxygen in blood). Any anomaly caused by genetic or acquired abnormalities in heme biosynthesis (produced majorly in bone marrow and liver) can result in toxicity. It is worth highlighting that, till date, more than 1,000 mutations that can cause porphyria have been identified. However, 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.
More than 70% of the aforementioned candidates are currently under clinical evaluation. Further, three therapies, namely Panhematin™, GIVLAARI® and SCENESSE®, have already been approved for the treatment of different types of porphyria.
Around 50% of the therapeutics are being developed as biologics
Majority (over 65%) of the abovementioned biologic drugs have been / are being designed for administration via the intravenous route. Furthermore, majority of the drugs (37%) have been / are being targeting acute intermittent porphyria.
Over 30% of the therapies have been / are being developed for erythropoietic protoporphyria
More than 65% the abovementioned therapies are currently being evaluated in clinical phases. Further, around 60% of the aforementioned therapy candidates are being developed as small molecules.
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