You can also find information about the treatment of your type of Porphyria under your specific type.
The American Porphyria Foundation (APF) promotes comprehensive care necessary for treating individuals with Porphyria. Although there is no cure for Porphyria, there is treatment available for each type of the disease.
In this section you'll find detailed information on treatment options, tips on finding a local doctor and building a good working relationship with your doctor. You will also find the Rare Disease ToolKit – an invaluable tool to use for each doctor visit. Please see the disease descriptions for additional information.
Panhematin® is a treatment for the acute Porphyrias manufactured by Recordati Rare Diseases in Lebanon New Jersey. It is a lyophilized form of alkaline heme that has to be reconstituted immediately prior to administration. Panhematin® should be infused into a large peripheral vein. A large central line or port may be used, if available.
Doctors administer Panhematin® to correct heme deficiency in the liver and repress production of porphyrin precursors. Panhematin® almost always normalizes porphyrin and porphyrin precursor values. Three to four mg/kg of Panhematin® given once daily for four days early in an attack produces a highly beneficial effect in most patients. Commonly noted are decreases in pulse rate, blood pressure, abdominal pain, as well as decreased levels of urinary porphobilinogen (PBG). These effects can occur within a day.
Panhematin® is the only commercially available heme therapy in the United States. (Heme arginate is another preparation, but is only available outside of the U.S.) While a high carbohydrate diet is recommended for patients with Porphyria, it is not regarded as highly effective by itself. Intravenous glucose therapy is a treatment option for mild attacks. When heme therapy was introduced as a treatment, it was recommended that it be initiated only after several days of glucose therapy was unsuccessful.
Today, physicians experienced in treating patients with attacks of Porphyria recommend early use of Panhematin® rather than waiting to see if glucose alone will be of decisive help.
GIVLAARI is a treatment used to prevent acute hepatic porphyria (AHP) in adults. There are 4 types of AHP: acute intermittent porphyria (AIP), variegate porphyria (VP), hereditary coproporphyria (HCP), and ALA-dehydratase deficient porphyria (ADP). GIVLAARI is given once a month as a subcutaneous injection (under the skin) by a healthcare professional.
GIVLAARI is a double-stranded small interfering RNA (siRNA) therapeutic specifically targeting ALAS1 mRNA, reducing ALAS1 mRNA levels and leading to reductions in urinary ALA and PBG.1
ALA, delta-aminolevulinic acid; ALAS1, delta-aminolevulinic acid synthase 1; mRNA, messenger RNA; PBG, porphobilinogen
SCENESSE® is a prescription medication that contains the active substance afamelanotide. Afamelanotide is used to increase tolerance to the sun and light in adults with a confirmed diagnosis of erythropoietic protoporphyria (EPP).
SCENESSE® (pronounced “sen-esse”) acts by increasing the levels of eumelanin in the skin, shielding against UV radiation (UVR) and visible light, including sunlight. Afamelanotide is a synthetic form of a hormone called alpha-melanocyte stimulating hormone (?-MSH). Afamelanotide works in a way similar to the natural hormone, by making skin cells produce eumelanin which is a brown-black type of melanin pigment in the skin. By increasing the amount of eumelanin and acting as an antioxidant, SCENESSE® can help to reduce the sensitivity of the skin to sunlight and artificial UV light sources.
Implant is given subcutaneously by a trained health care professional.