Market Overview

Sobi Receives Approval from Health Canada for Once-Daily Dosing of Orfadin® (nitisinone) for the Treatment of HT-1


- First and only nitisinone product in Canada approved for
once-daily use, designed to meet the needs of HT-1 patients entering
older childhood, adolescence and adulthood -

a pioneering international biopharmaceutical company dedicated to rare
diseases, has received approval from Health Canada for a reduced dosing
frequency for Orfadin® (nitisinone).

Orfadin is approved in combination with dietary restriction of tyrosine
and phenylalanine in the treatment of hereditary tyrosinemia type 1
(HT-1), a rare genetic disorder that may result in liver, renal and
neurological complications and in most cases is fatal if untreated. The
once-daily reduced dosing frequency is approved for use in patients who
weigh 20 kg or more and who have undetectable serum and urine
succinylacetone concentrations after a minimum of 4 weeks on a stable
twice-daily dosage of nitisinone.

"Orfadin is the first nitisinone product approved for once-daily use in
Canada. This is an important step towards reducing the treatment burden
of people with HT-1 and it follows Sobi's introduction of Orfadin oral
suspension and 20 mg capsules," said Bob McLay, Vice President and
General Manager of Sobi Canada, Inc. "In the U.S., where all Orfadin
dosing and formulation options have been available for some time, we see
patients increasingly choosing to take advantage of the flexibility that
these options offer. I am glad to be able to offer these choices to
patients in Canada as well."

Sobi currently provides a range of Orfadin dosing options in Canada
including 2mg, 5mg, 10mg, and 20mg capsules, as well as a 4mg/mL oral
suspension and the new once-daily dosing frequency. The original
Canadian approval of Orfadin was based on a multinational, uncontrolled,
open-label study of 291 HT-1 patients. Patients treated with Orfadin and
dietary restrictions experienced 2-, 4-, and 6-year survival
probabilities of 93% to 96%; historical controls treated with dietary
restriction alone experienced survival probabilities of 38% and 29% (1-
and 2-year survival, respectively, for patients younger than 2 months of
age at the onset of symptoms), 74% (for patients 2-6 months of age at
the onset of symptoms), and 96% (for patients older than 6 months of age
at the onset of symptoms). The most common reported adverse reactions in
the trial were thrombocytopenia, leucopenia, granulocytopenia and
conjunctivitis, corneal opacity, keratitis, and photophobia. No patients
discontinued treatment due to adverse drug reactions. More information
and the Product Monograph can be found at the Sobi
North America website

"HT-1 caregivers often feel their life is far from normal, revolving
around the demanding treatment schedule and dietary restrictions, so I
am grateful to Sobi for bringing new dosing options like once-daily
Orfadin to Canada," said Durhane Wong-Rieger, President of the Canadian
Organization for Rare Disorders. "It is particularly important to have
dosing options like this for patients approaching adolescence, as
studies have shown that adherence to medicines is more difficult at this

About hereditary tyrosinemia (HT-1)
People with hereditary
tyrosinemia 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, renal and neurological complications. In
most cases HT-1 is fatal if untreated. Approximately 1,000 persons
worldwide are identified as living with HT-1 today. Roughly 10 percent
of those patients reside in Canada.

About Orfadin®
(nitisinone) blocks the breakdown of tyrosine, thereby reducing the
amount of toxic tyrosine by-products in the body. Patients must maintain
a special diet in combination with Orfadin treatment as tyrosine is not
adequately broken down. Orfadin is a proprietary product and is
developed and made available globally by Sobi. For full European
prescribing information, please visit the EMA website. For full US
prescribing information please see
Full Canadian prescribing information can be found on the Sobi
North America website

About Sobi™
Sobi™ is an international specialty healthcare
company dedicated to rare diseases. Our vision is to be recognized as a
global leader in providing access to innovative treatments that make a
significant difference for individuals with rare diseases. The product
portfolio is primarily focused on treatments in Hemophilia and Specialty
Care. Partnering in the development and commercialization of products in
specialty care is a key element of our strategy. Sobi has pioneered in
biotechnology with world-class capabilities in protein biochemistry and
biologics manufacturing. In 2017, Sobi had total revenues of SEK 6.5
billion and approximately 850 employees. The share (STO:SOBI) is listed
on Nasdaq Stockholm. More information is available at

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