PBGD-conjugate: improving hemin efficacy in AIP treatment

  • 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.

  • 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.  

  • 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.

  • A new molecule rhPBGD‐conjugate has been developed with the following properties:  

    • metabolizes serum PBG.

    • subcutaneous administration.

    • increased half‐life in circulation than previous failed approaches rhPBGD (Zymenex A/S).

  • Indication: combined administration with hemin in AIP acute attacks.

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