Pafuramidine (formulated as the
maleic acid
Maleic acid or ''cis''-butenedioic acid is an organic compound that is a dicarboxylic acid, a molecule with two carboxyl groups. Its chemical formula is HO2CCH=CHCO2H. Maleic acid is the ''cis''-isomer of butenedioic acid, whereas fumaric ac ...
salt
Salt is a mineral composed primarily of sodium chloride (NaCl), a chemical compound belonging to the larger class of salts; salt in the form of a natural crystalline mineral is known as rock salt or halite. Salt is present in vast quant ...
pafuramidine maleate) is an experimental drug for the treatment of
pneumocystis pneumonia (PCP). In 2006, pafuramidine was given
orphan drug status by the
US Food and Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food ...
for PCP in patients with
HIV/AIDS
Human immunodeficiency virus infection and acquired immunodeficiency syndrome (HIV/AIDS) is a spectrum of conditions caused by infection with the human immunodeficiency virus (HIV), a retrovirus. Following initial infection an individual ...
. Preliminary clinical trials indicated that pafuramide was effective against pneumocystis pneumonia and had the potential for fewer side effects than the standard treatment with
trimethoprim/sulfamethoxazole
Trimethoprim/sulfamethoxazole, sold under the brand name Bactrim among others, is a fixed-dose combination antibiotic medication used to treat a variety of bacterial infections. It consists of one part trimethoprim to five parts sulfamethoxa ...
(TMP-SMX).
Pafuramidine also reached Phase III clinical trials for the treatment of first stage
African sleeping sickness, but development was halted in 2008 over concerns about kidney toxicity.
References
{{reflist
Furans
Antiparasitic agents
Amidines