Basilea reports isavuconazole orphan drug designation by U.S. FDA

Basilea reports isavuconazole orphan drug designation by U.S. FDA 
BASEL, SWITZERLAND -- (Marketwired) -- 05/28/13 --  Basilea
Pharmaceutica AG / Basilea reports isavuconazole orphan drug
designation by U.S. FDA 
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Basilea Pharmaceutica Ltd. (SIX: BSLN) reported today that the U.S.
Food and Drug Administration (FDA) has granted orphan drug
designation to isavuconazole for the treatment of invasive
An FDA orphan drug designation provides several benefits to the
sponsor including a seven-year market exclusivity from product
approval in the U.S. Isavuconazole was previously granted FDA fast
track status that is designed to
facilitate development and expedite
the review of drugs to treat serious diseases and that fill an unmet
medical need in order to get important new drugs
to patients earlier. 
Prof. Achim Kaufhold, Basilea's Chief Medical Officer, commented:
"Invasive aspergillosis is a life-threatening infection caused by
Aspergillus molds. It
typically affects patients with an impaired or
weakened immune system such as
transplant and cancer patients and is
associated with high mortality. The granting of orphan designation
for isavuconazole in the U.S. reflects the high
medical need and is
an important regulatory milestone for Basilea and our partner
Topline data from two isavuconazole phase 3 studies are expected in
the second
half of 2013. These include the SECURE phase 3 registration
study, evaluating
safety and efficacy of once-daily isavuconazole
versus twice-daily voriconazole
for the primary treatment of invasive
fungal disease caused by Aspergillus species and from the open-label
VITAL study investigating isavuconazole for the
treatment of patients
with invasive life-threatening fungal disease caused by
fungi and the treatment of aspergillosis patients with pre-existing
renal impairment. 
The isavuconazole ACTIVE phase 3 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, will
continue to recruit into 2014. 
Invasive aspergillosis is estimated to occur in 5-13 % of recipients
of bone
marrow transplants, 5-25 % of patients who have received heart
or lung transplants, and 10-20 % of
patients who are receiving
intensive chemotherapy for leukemia.(1)Mortality rates for transplant
patients with invasive aspergillosis have been reported to be between
34 and 58 %.(2) 
About isavuconazole 
Isavuconazole (drug substance: isavuconazonium sulfate) is an
intravenous and oral broad-spectrum antifungal. In
collaboration with Astellas
Pharma Inc., isavuconazole is being
investigated in phase 3 clinical studies for the treatment of severe
invasive fungal infections. The drug demonstrated in-vitro and
in-vivo coverage of a broad range of yeasts (such as Candida
and molds (such as Aspergillus species) as well as in-vitro
activity against
less prevalent but often fatal molds including those
that cause mucormycosis. In clinical studies to date, isavuconazole
achieved predictable drug levels supporting reliable dosing and a
switch from intravenous administration to a
once-daily oral dose. The
intravenous formulation of isavuconazole, which is
does not contain potentially kidney damaging solubilizers and has the
potential to be given also to patients with pre-existing renal
About Basilea 
Basilea Pharmaceutica Ltd. is headquartered in Basel, Switzerland,
and listed on the SIX Swiss Exchange (SIX: BSLN). Through the fully
integrated research and
development operations of its Swiss
subsidiary Basilea Pharmaceutica International Ltd., the Company
focuses on innovative pharmaceutical products in the therapeutic
areas of bacterial infections, fungal infections and
targeting the medical challenge of rising resistance and
non-response to current
treatment options. 
This communication expressly or implicitly contains certain
forward-looking statements concerning Basilea Pharmaceutica Ltd. and
its business. Such statements involve certain known and unknown
risks, uncertainties and other factors, which could cause the actual
results, financial condition, performance
or achievements of Basilea
Pharmaceutica Ltd. to be materially different from
any future
results, performance or achievements expressed or implied by
forward-looking statements. Basilea Pharmaceutica Ltd. is
providing this communication as of this date and does not undertake
to update any forward-looking statements contained herein as a result
of new information, future
events or otherwise. 
This press release can be downloaded from 
1 Harman EM, Medscape Reference, Drugs, Diseases & Procedures,
Clinical Presentation, 
2 Baddley JW et al. Factors Associated with Mortality in Transplant
with Invasive Aspergillosis. Clinical Infectious Disease
2010 (50), 1559-1567 
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+41 61 606 1102 
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Barbara Zink, PhD, MBA         
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