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FDA Grants Orphan Drug Designation to Astellas for Isavuconazole for the Treatment of Invasive Candidiasis

NORTHBROOK, Ill., Nov. 3, 2014 /PRNewswire/ -- Astellas announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to isavuconazole for the treatment of invasive candidiasis.

Astellas is a pharmaceutical company dedicated to improving the health of people around the world.

An FDA orphan drug designation provides several benefits to the sponsor including a seven-year period of market exclusivity in the United States, should the FDA grant orphan drug exclusivity at approval. The FDA has previously granted orphan drug designation for isavuconazole for the treatment of invasive aspergillosis and invasive mucormycosis.

"Invasive candidiasis is a serious hospital-acquired fungal infection that is associated with increased morbidity and mortality in certain immunocompromised patients. Increasing reports of Candida resistance to available therapies necessitates the development of new antifungals to treat these serious infections," said Bernie Zeiher, M.D., executive vice president, Global Development and therapeutic area head of Infectious Disease at Astellas. "The FDA's decision to grant isavuconazole orphan drug status underscores the growing need for additional tools to treat invasive candidiasis."

Previously, the FDA also designated isavuconazole as a Qualified Infectious Disease Product (QIDP) for invasive candidiasis, invasive aspergillosis and invasive mucormycosis. QIDP status provides priority review and a five-year extension of market exclusivity in the United States. QIDP incentives were established under the 2012 U.S. Generating Antibiotic Incentives Now (GAIN) Act as a part of the FDA Safety and Innovation Act.


About invasive candidiasis
Infections by Candida yeasts are the most frequent invasive fungal infections in critically ill patients, accounting for 70% to 90% of all invasive mycoses. They are the fourth and seventh most common cause of bloodstream infections in the U.S. and Europe, respectively, and are associated with increased morbidity and mortality. Estimates of the attributable mortality of Candida bloodstream infections (candidemia) range from 15% to 49%.


About Isavuconazole
Isavuconazole (drug substance: isavuconazonium) is an investigational once-daily intravenous and oral broad-spectrum antifungal being developed for the treatment of severe invasive and life-threatening fungal infections. Isavuconazole is the active moiety of the prodrug isavuconazonium. Information regarding isavuconazole ongoing clinical trials is available at  

Isavuconazole demonstrated in-vitro and in-vivo coverage of a broad range of yeasts (such as Candida species) and molds (such as Aspergillus species) as well as activity against emerging and often fatal molds including those that cause mucormycosis.

In the Phase 3 invasive aspergillosis study, isavuconazole demonstrated non-inferiority to voriconazole on the primary endpoint of all-cause mortality at day 42.  Also, in an open-label phase 3 study of isavuconazole, isavuconazole demonstrated efficacy in the treatment of invasive Mucormycosis (n = 37). The adverse reactions most frequently reported with isavuconazole in therapeutic clinical trials were nausea, vomiting, diarrhea, headache, elevated liver chemistry tests, hypokalemia, abdominal pain, dyspnea, edema peripheral, and constipation.  During IV administration, infusion related reactions included hypotension, dyspnea, chills, dizziness, paresthesia, hypoesthesia, nausea, and headache. Isavuconazole is being co-developed with Basilea Pharmaceutica International Ltd.


About Astellas Infectious Disease
Astellas' commitment to the field of infectious diseases is an active one. We're expanding the knowledge base of this therapeutic area and empowering physicians to make evidence-based clinical decisions.

Our proud history of collaborating with investigators around the world provides ideal environments to study compounds that have the potential for significant breakthroughs and allows us to generate and publish key information on our advances. In fact, Astellas has performed some of the world's largest clinical trials in fungal infections.


About Astellas
Astellas is a pharmaceutical company dedicated to improving the health of people around the world through provision of innovative and reliable pharmaceuticals. For more information on Astellas, please visit our website at and follow us on Twitter at

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SOURCE Astellas

For further information: Marjorie Moeling,, 224-205-5205 (office), 847- 682-7471 (mobile)
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