PBGD-conjugate: improving hemin efficacy in AIP treatment

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Acute Intermittent Porphyria (AIP) is a rare metabolic liver disorder resulting from mutations in the porphobilinogen deaminase (PBGD), which encodes for the third enzyme involved in the production of heme.
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Current Standard of Care (SoC) in AIP is intravenous hemin administration, which provides exogenous heme for the negative feedback inhibition of ALAS, decreasing further ALA and PBG production.
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Hemin treatment effect is slow and requires 3 or 4 daily infusions. Moreover, side effects as headache, thrombophlebitis, hepatosiderosis and decreasing sensitivity should be considered.
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A new molecule rhPBGD‐conjugate has been developed with the following properties:
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metabolizes serum PBG.
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subcutaneous administration.
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increased half‐life in circulation than previous failed approaches rhPBGD (Zymenex A/S).
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Indication: combined administration with hemin in AIP acute attacks.
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