FDA
21st October 2013

FDA Supports use of Miltefosine Capsules for Leishmaniasis

On Friday, a panel of the US Food and Drug Administration (FDA) endorsed miltefosine capsules for the treatment of visceral, cutaneous, and mucosal leishmaniasis.

FDA Supports use of Miltefosine Capsules for Leishmaniasis

On Friday, a panel of the US Food and Drug Administration (FDA) endorsed miltefosine capsules for the treatment of visceral, cutaneous, and mucosal leishmaniasis.

The FDA’s Anti-Infective Drugs Advisory Committee took 3 separate votes on the efficacy and safety profile of Paladin Labs Inc’s alkyllysophospholipid analogue miltefosine (Impavido),voting 15 to 1 in favor of treatment of visceral leishmaniasis (VL) caused by Leishmania donovani, 14 to 2 for treatment of cutaneous leishmaniasis (CL) caused by members of the Leishmania viannia subgenus (Lv braziliensisLv guyanensis, and Lv panamensis), and 13 to 3 for treatment of mucosal leishmaniasis (ML) caused by the 3 aforementioned Leishmania viannia subtypes.

Leishmaniasis is rare in developed countries, typically only imported by travelers and members of the military, but Paladin is seeking US market clearance to make the medication more globally available. 

Paladin’s proposed indication for the oral miltefosine capsules is for children and adults aged 12 years and older who weigh 30 kg or more. The drug has been marketed for the last decade in 14 countries for VL and CL, but not ML. Most of those countries, including several in Latin America and Asia, are where the parasitic infection is most endemic.

VL is an infection of the liver, spleen, and bone marrow. It affects about 0.5 million people, primarily in the Indian subcontinent and East Africa and is fatal if left untreated. Current treatments include intravenous or intramuscular antimonials or intravenous amphotericin B for antimonial-resistant cases; both of which, have high rates of adverse effects.

There are about 1.5 million cases worldwide of CL, an infection of the macrophages of the skin. Although routine cases self-heal in 3 to 15 months, CL can metastasize to ML, a progressive destruction of the mucosa and the cartilage and bones of the nose, pharynx, and larynx that does not usually self-heal.

On the vote regarding VL, panel member Barbara Herwaldt, MD, a leishmaniasis expert from the Centers for Disease Control and Prevention, Atlanta, Georgia, voted yes and stated that, “I think miltefosine would be advantageous to have available, but I do have issues in terms of what I think should be in the package insert.” Dr. Herwaldt recommended that the label stress the importance of contraception and explain that there are differences between leishmaniasis species, and even among the same species in different areas, so the drug may not work everywhere uniformly.

For CL, Alan J. Magill, MD, malaria director for the Bill and Melinda Gates Foundation, Seattle, Washington, said “I voted an enthusiastic yes…. I think it really fulfills an unmet need in the United States. The numbers of patients are not huge, but it’s important to have options available,” he said, adding that there are data to suggest that miltefosine could work for CL because of species other than those listed in the indication.

Panel members cited the drug’s demonstrated efficacy and the unmet need for an oral leishmaniasis medication. However, many also said the package label should include cautions regarding some of the drug’s potential toxicities, including vomiting, diarrhea, and adverse male and female reproductive effects.

Several committee members also requested that the FDA recommend direct observation of therapy for miltefosine treatment for leishmaniasis, as is currently done with tuberculosis treatment. Some panel members also requested more data on use of the drug in children younger than 12 years and on dosing for patients with weights above the cutoffs in the studies performed in endemic countries.

Currently, there are no drugs approved for either CL or ML in the United States.

The FDA is not obligated to follow the advice given by their panels but normally does.

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