Panhematin (hemin for injection), an approved medication to treat acute intermittent porphyria (AIP), has been deemed an essential medicine by the U.S. Food and Drug Administration (FDA), a designation intended to secure reliable long-term access to the therapy.
Following an executive order — Ensuring Essential Medicines, Medical Countermeasures, and Critical Inputs are Made in the United States — issued in August of this year, the FDA has worked with other federal partners to develop a list of medically necessary medicines to be available at all times in the amounts and dosages needed to serve patients.
Selected therapies were those that can be used in the broadest population with the greatest potential impact on public health.
The goal is to ensure the protection of Americans against future outbreaks of emerging infectious diseases, such as COVID-19, as well as biological, chemical, radiological, and nuclear threats.
The executive order, issued by the U.S. president, seeks to establish long-term domestic production of these essential medicines while minimizing potential shortages by scaling down the country’s dependence on foreign manufacturers.
Panhematin was one of 227 medicines and biological products plus 96 medical devices considered most necessary in acute care facilities to address life-threatening injuries or medical conditions.
The devices include diagnostic testing kits and materials to develop and process rapid tests, personal protective equipment, vital sign monitoring devices, vaccine delivery devices, and those needed for acute illnesses such as ventilators.
AIP is a rare genetic disorder caused by a deficiency of the enzyme hydroxymethylbilane synthase (HMBS), which is essential in the production of heme — part of the hemoglobin protein that carries oxygen in red blood cells.
A lack of HMBS leads to the toxic buildup of heme precursors known as porphyrins, causing symptoms that include abdominal pain and nausea, and that can come as discrete attacks.
Panhematin works by supplying heme, which lowers the activity of other enzymes upstream of HMBS, thereby reducing the amount of porphyrin that the body produces.
The executive order also directs the FDA to coordinate with the Department of Health and Human Services to find strategies to acquire the essential medications on the list, help accelerate domestic manufacturing, and identify and address vulnerabilities in the supply chain.
The FDA is encouraging pharmaceutical manufacturers in the U.S. to adopt advanced manufacturing technologies to strengthen their competitiveness with companies in foreign countries to ensure a stable supply of medicines to U.S. patients.
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