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FDA grants orphan drug designation to Impavido for brain-eating infection

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The FDA has granted orphan drug designation to Impavido for the treatment of granulomatous amebic encephalitis, or GAE, a deadly infection caused by a brain-eating ameba, according to a press release.

Among the causes of the infection is the Balamuthia mandrillaris ameba, which is believed to enter the body by way of soil or dust through a wound, the mouth or inhalation before traveling to the brain through the bloodstream.

Impavido (miltefosine, Profounda) previously received orphan drug designation for treating primary amebic meningoencephalitis (PAM), a brain infection caused by the water-borne amebas Naegleria fowleri and Acanthamoeba keratitis. It is also the only FDA–approved oral treatment for visceral, mucosal and cutaneous leishmaniasis, a rare tropical parasitic disease, according to the release.

“While the number of patients dealing with these deadly amoebic infections is small, the devastating impact to families with loved ones is undeniable,” Profounda CEO Todd MacLaughlan said in the release. “Increased awareness can lead to faster diagnosis of GAE and PAM, and access to a potentially successful treatment is key.”

Often, GAE is diagnosed only after the patient has died, although blood, cerebrospinal fluid and tissue samples from a living patient can also lead to a diagnosis.

Because diagnosing and treating GAE quickly is critical, Profounda announced that it is offering a miltefosine consignment program for health care providers. The company charges only a $100 fee for shipping the drug and will replace expired product, MacLaughlan told Infectious Disease News.

There is also no charge for product that is never used for patients, he added.

“We expect that most hospitals won’t use it, but for the one that needs it, we know time matters,” MacLaughlan said.

As of June 29, 15 hospitals throughout the United States have participated in the program, he said. Instructions for ordering miltefosine through the program can be found at www.impavido.com. – by Joe Green

Disclosure: MacLaughlan is CEO of Profounda.