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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