FDA validates Orfadin® oral suspension filing
(Thomson Reuters ONE) -
Sobi (Swedish Orphan Biovitrum AB (publ)) today announced that the Food and Drug
Administration (FDA) has initiated review of the oral suspension formulation of
Orfadin (nitisinone) for the treatment of Hereditary Tyrosinaemia type-1 (HT-
1). HT-1 is a rare genetic disease characterised by signs and symptoms that
begin in the first few months of life. In affected infants the disease may
result in liver and kidney failure, and can be fatal if not diagnosed and
treated early in life.
The dossier was submitted to FDA in June and a formal decision is expected
during the second quarter 2016. The oral suspension of Orfadin, if approved,
will facilitate dosing accuracy and will simplify ease of use by reducing the
number of steps required to administer the medicine.
"The oral suspension formulation is a demonstration of Sobi's commitment to the
needs of the infants and children diagnosed with HT-1 early in life. As new-born
screening for HT-1 is becoming more widespread the need for such formulations is
evident," says Michael Yeh, Vice President Head of Medical for Sobi in North
America. "We are pleased about the FDA validation of the filing of the Orfadin
oral suspension, and believe that the oral suspension will contribute to address
an unmet need and improve the treatment opportunities of paediatric patients
with HT-1."
Orfadin is the only FDA approved therapy for use as an adjunct to dietary
restriction of tyrosine and phenylalanine for the treatment of hereditary
tyrosinaemia type 1 (HT-1). It is also approved in the EU and several other
countries and the European Commission recently approved the oral suspension
formulation of Orfadin.
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About Orfadin
Orfadin (nitisinone) blocks the breakdown of tyrosine, thereby reducing the
amount of toxic by-products in the body. People with Hereditary Tyrosinaemia
type-1 (HT-1) have problems breaking down an amino acid called tyrosine. Toxic
by-products are formed and accumulate in the body, which can cause liver
failure, renal dysfunction and neurological complications. In the most common
form of the disease, symptoms arise within the first six months of the child's
life. Patients must maintain a special diet in combination with Orfadin
treatment as tyrosine remains in the body. Approximately 1,000 persons are
identified as living with HT-1 today. Orfadin is a proprietary product and is
developed by and marketed globally by Sobi.
About Sobi
Sobi is an international specialty healthcare company dedicated to rare
diseases. Our mission is to develop and deliver innovative therapies and
services to improve the lives of patients. The product portfolio is primarily
focused on Haemophilia, Inflammation and Genetic diseases. We also market a
portfolio of specialty and rare disease products for partner companies across
Europe, the Middle East, North Africa and Russia. Sobi is a pioneer in
biotechnology with world-class capabilities in protein biochemistry and
biologics manufacturing. In 2014, Sobi had total revenues of SEK 2.6 billion
(USD 380 M) and about 600 employees. The share (STO: SOBI) is listed on NASDAQ
OMX Stockholm. More information is available at www.sobi.com.
For more information please contact
Media relations Investor relations
Oskar Bosson, Head of Jörgen Winroth, Vice President, Head of Investor
Communications Relations
T: +46 70 410 71 80 T: +1 347-224-0819, +1 212-579-0506, +46 8 697 2135
oskar.bosson(at)sobi.com jorgen.winroth(at)sobi.com
033e_FDA validation_Orfadin Oral Suspension:
http://hugin.info/134557/R/1950916/709389.pdf
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Source: Swedish Orphan Biovitrum AB (publ) via GlobeNewswire
[HUG#1950916]
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Datum: 10.09.2015 - 08:00 Uhr
Sprache: Deutsch
News-ID 419115
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