Health Canada has approved the use of Panhematin to reduce the production and buildup of toxic compounds in the body induced by hepatic porphyria, and prevent associated symptoms such as severe abdominal pain.
“We are excited that Panhematin is now available for Canadian patients as a treatment for painful and devastating AIP attacks,” Renata Sklodowska, MD, director of medical affairs at Recordati Rare Diseases, said in a press release. “The fact that a disease is uncommon does not mean that it should go untreated. Recordati remains committed to supporting patients with rare disorders, including the needs of the Canadian AIP community.”
Panhematin is derived from processed human red blood cells. It is believed to inhibit the ALAS enzyme and to limit the rate of the porphyrin/heme biosynthesis pathway, preventing the accumulation of compounds known to induce acute hepatic porphyria attacks, such as aminolevulinic acid (ALA) and porphobilinogen (PBG), by about 58-100%.
The effectiveness of Panhematin in reducing the rates of recurrent AIP attacks was demonstrated in five open-label studies, with approximately 85.5% of treated patients showing a positive response.
In addition, an observational study that investigated patient-reported outcomes in 108 patients with acute porphyrias revealed that 74% of patients who received Panhematin during acute attacks had successful resolution of abdominal pain and other symptoms. This was a significant improvement over the 44% of patients treated with opiates who reported a similar positive response.
Clinical evidence of the beneficial effect of Panhematin led to its initial approval by the U.S. Food and Drug Administration in 1983.
Panhematin is administrated directly into a large vein in the arm by intravenous infusion or through a central line. Administration in small arm veins is not recommended since vein inflammation has been reported as a potential adverse side effect.
It should be used in doses between 0.8 to 3.1 mg/kg per day for three to 14 days according to the presenting clinical signs. The maximum daily dose should not exceed 4.6 mg/kg, even when using multiple dosing in more severe cases.
Panhematin should not be used while taking estrogen-based agents such as oral contraceptives, barbiturates to regulate sleep or to treat epilepsy, or steroids, as these class agents may trigger an AIP attack or make one worse.