FDA Grants Astellas Qualified Infectious Disease Product Designation for Isavuconazole for the Treatment of Invasive Aspergillosis

NORTHBROOK, Ill., Dec. 3, 2013 /PRNewswire/ -- Astellas Pharma US, Inc., a subsidiary of Astellas Pharma Inc. based in Tokyo, Japan, announced today that the U.S. Food and Drug Administration (FDA) designated isavuconazole as a Qualified Infectious Disease Product (QIDP) for the treatment of invasive aspergillosis. QIDP status provides priority review and a five-year extension of market exclusivity if a product receiving such a designation is approved in the United States. These incentives were granted under the 2012 U.S. Generating Antibiotic Incentives Now (GAIN) Act as part of the FDA Safety and Innovation Act.

The possibility of a five-year extension is in addition to the potential seven-year exclusivity based on isavuconazole's FDA orphan drug designation for the treatment of invasive aspergillosis. In the U.S., isavuconazole also received orphan drug designation for the treatment of zygomycosis, a life-threatening invasive fungal infection (IFI) caused by emerging molds.

"Invasive aspergillosis and zygomycosis are life threatening infections that typically occur in severely immunocompromised patients. Acceptance of Aspergillus as a qualified pathogen is a recognition of the need for new treatment options for patients infected with these fungi," said Bernie Zeiher, Senior Vice President and Global Therapeutic Area Head of Immunology and Infectious Diseases at Astellas Pharma Global Development, Inc. "We applaud the bipartisan Congressional supporters of the GAIN Act for their commitment to encouraging investment in treatments for these serious infectious diseases through the passage of this legislation."

IFIs are debilitating or life-threatening infections that attack internal tissues or organs and can spread through the bloodstream. Fungi commonly involved in IFIs include Aspergillus (molds) and Candida (yeasts).[1], [2] Invasive aspergillosis is caused by Aspergillus molds and typically affects patients with an impaired or weakened immune system. It is estimated to occur in five to 13 percent of recipients of bone marrow transplants often associated with leukemia, five to 25 percent of patients who have received heart or lung transplants, and 10 to 20 percent of patients who are receiving intensive chemotherapy for leukemia.[3] Mortality rates for transplant patients with invasive aspergillosis have been reported to be between 34 and 58 percent.[4] Zygomycosis is an important emerging fungal infection, associated with high morbidity and mortality. Mortality rates have been reported to be as high as 80 percent in infected transplant patients.[5]

About isavuconazole
Isavuconazole (drug substance: isavuconazonium sulfate) is an investigational once daily intravenous and oral broad-spectrum antifungal for the potential treatment of severe invasive and life-threatening fungal infections. It is currently in phase 3 of clinical development. 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 in in-vitro studies and animal models against emerging and often fatal molds including those that cause zygomycosis. Isavuconazole received U.S. FDA fast-track status and U.S. orphan drug designation for invasive aspergillosis and zygomycosis. Isavuconazole is being co-developed with Basilea Pharmaceutica Ltd.

About the isavuconazole phase 3 program
The phase 3 program with isavuconazole includes three studies, SECURE, VITAL and ACTIVE. Recently, positive topline results were reported from the SECURE study, a global randomized, double-blind phase 3 study, designed to evaluate the safety and efficacy of once-daily isavuconazole versus twice-daily voriconazole in the primary treatment of invasive fungal disease caused by Aspergillus species or certain other filamentous fungi. Isavuconazole demonstrated non-inferiority versus voriconazole. Isavuconazole was effective as determined by the primary endpoint of all-cause mortality through day 42 in the intent-to-treat population (N=516). Study drug-related adverse events were reported in 42.4% of the isavuconazole and 59.8% of the voriconazole treatment group. Overall drug- and non-drug-related adverse events were reported in 96.1% and 98.5% of patients in the isavuconazole and voriconazole treatment groups, respectively.

The VITAL study is an open-label phase 3 study including patients with invasive fungal disease caused by emerging fungal pathogens such as Zygomycetes and patients with aspergillosis and pre-existing renal impairment. Enrollment into VITAL has been completed (N=150). Based on the investigator reported data, approximately 45 patients were enrolled with zygomycosis and a similar number of patients were enrolled with pre-existing renal impairment. Review of diagnosis and outcomes by an Independent Data Review Committee is ongoing.

The phase 3 ACTIVE study is a randomized, double-blind study evaluating the use of isavuconazole i.v. and oral versus caspofungin i.v. followed by oral voriconazole for the treatment of invasive Candida infections. The ACTIVE study continues to recruit with anticipated completion of enrollment in the first part of 2015.

About Astellas
Astellas Pharma US, Inc., located in Northbrook, Illinois, is a US affiliate of Tokyo-based Astellas Pharma Inc. Astellas is a pharmaceutical company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceutical products. The organization is committed to becoming a global category leader in focused areas by combining outstanding R&D and marketing capabilities. For more information about Astellas Pharma US, Inc., please visit our website at www.astellas.us of follow us on Twitter at AstellasUS.

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[1] Alangaden GJ. Nosocomial fungal infections: epidemiology, infection control, and prevention. Infect Dis Clin N Am. 2011;25:201-225.

[2] Lass-Flörl C. The changing face of epidemiology of invasive fungal disease in Europe. Mycoses. 2009;52:197-205.

[3] Harman EM. Medscape Reference, Drugs, Diseases & Procedures, Aspergillosis Clinical Presentation. http://emedicine.medscape.com/article/296052-overview

[4] Baddley JW et al. Factors Associated with Mortality in Transplant Patients with Invasive Aspergillosis. Clinical Infectious Diseases 2010 (50), 1559-1567

[5] Greenberg RN et al. Zygomycosis (Mucormycosis): Emerging Clinical Importance and New Treatments. Current Opinion in Infectious Diseases 2004 (17), 517-525

SOURCE Astellas Pharma US, Inc.