Potential Porphyria Treatment Include: Enzyme Replacement Therapy, Gene Replacement Therapy, mRNA Therapeutics, And Hepatocyte Transplantation

 

Porphyria is a group of disorders caused by an over-accumulation of porphyrin which helps haemoglobin, the protein that carries oxygen in the blood. The specific names of the eight types of porphyrias are: acute intermittent porphyria, congenital erythropoietic porphyria, delta-aminolevulinate-dehydratase deficiency porphyria, erythropoietic protoporphyria, hereditary coproporphyria, hepatoerythropoitic porphyria, porphyria cutanea tarda, and variegate porphyria. In Germany, analyses of clinical and laboratory features of patients with acute porphyrias are only available for hereditary coproporphyria.

The condition can't be cured. However, certain lifestyle changes may aid in avoiding symptoms of porphyria. Porphyria Treatment involves intravenous (IV) heme or glucose infusions (adding heme or glucose directly into a vein). The most common inborn errors of metabolism are evident in acute intermittent porphyria (AIP), a defect of heme synthesis enzymes. The condition leads to excessive accumulation and excretion of porphyrins and their precursors, including neurotoxic δ-aminolevulinic acid (ALA) and porphobilinogen (PBG).

Current Porphyria Treatments include Haem infusions, in the form of haemin (Panhematin, Recordati Rare Diseases, Xellia Pharmaceuticals USA, LLC) or haem arginate (Normosang, Recordati Rare Diseases, Puteaux, France). Liver transplantation is a last-resort treatment alternative for severely affected AIP individuals. In November 2019, Givosiran, a ribonucleic acid interference (RNAi) therapy, was approved for the treatment of adults with acute hepatic porphyria in the U.S. It uses synthetic small interference RNA (siRNA) to reduce hepatic ALAS1 messenger RNA conjugated to N-acetyl-galactosamine for liver-specific delivery. The drug received a positive opinion in January 2020 in EU for treatment in adults and adolescents aged 12 years and older. Later in March 2020, the European Commission approved Givosiran (GIVLAARI) for the treatment of AHP in adults and adolescents 12 years and older.

Enzyme replacement therapy, gene replacement therapy, mRNA therapeutics, and hepatocyte transplantation are potential porphyria treatments in developments. In June 2020, Alnylam Pharmaceuticals, Inc., an RNAi therapeutics company, announced the presentation of positive data from ENVISION Phase 3 study that assessed the benefits of givosiran in patients with acute hepatic porphyria.

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