VL, also known as kala azar ("black fever"), is a fatal disease transmitted by sand flies, which spread leishmania parasites that attack internal organs. An estimated 1.5 million people worldwide are currently infected; the number of new VL cases per year is estimated at 500,000; and as many as 200,000 people die annually. More than 90 percent of VL cases occur in five countries: India, which bears the greatest disease burden, Bangladesh, Nepal, Sudan, and Brazil.
The U.S. Orphan Drug Act provides for formal protocol assistance when requested by the sponsors of drugs for rare diseases or conditions. The EMEA regulations are similar, and have the added advantage of applying to all 25 members of the European Union. Orphan Drug Designation also provides incentives such as exemption from certain registration fees, and may help pave the way for approvals in other countries.
OneWorld Health will file for approval of paromomycin in India this year where the need is greatest. It will begin the drug approval process with the FDA and/or the EMEA next year.
"We are pleased with the swift, positive responses by the FDA and EMEA agencies," said Victoria Hale, Ph.D., CEO and Founder of OneWorld Health. "By meeting the rigorous standards of either agency, we will be prepared to accelerate approvals in countries that have a critical need to treat VL."
Paromomycin is an off-patent aminoglycoside antibiotic first used in the 1960s and is still marketed in the U.S. as an oral formulation to treat intestinal parasites. The largest-ever Phase III clinical trial for visceral leishmaniasis was completed in November 2004 by OneWorld Health in collaboration with the Special Programme for Research and Training in Tropical Diseases of the World Health Organization (WHO/TDR). The clinical trial used the injectable form of paromomycin in 667 patients in India.