SAPROPTERIN DIHYDROCHLORID PULVER UND TABLETTEN SIND JETZT IN UK ERHÄLTLICH
Chiasso, Switzerland and Reading, England, October 4th, 2023 – Dipharma SA (“Dipharma”) and LogixX
Pharma (“LogixX”) are delighted to announce the official launch of Sapropterin Dihydrochloride
(“Sapropterin”) in the United Kingdom.
Sapropterin Dihydrochloride, a generic equivalent to Kuvan®, is now available in all dosage forms,
including sugar free soluble tablets of 100mg strength. What makes this launch even more noteworthy is
the introduction of Sapropterin Dihydrochloride in powder form for oral solution, available in 100mg and
500mg strengths, marking the first-ever release of this formulation in the UK.
Marc-Olivier Geinoz, CEO of Dipharma, expressed his enthusiasm, stating, “After successfully introducing
Sapropterin Dipharma in several major European countries, we are excited to announce its launch also in
the UK. This achievement is the result of a strong collaboration with our esteemed UK partner, LogixX.
Starting today, patients in the UK will have access to Sapropterin Dihydrochloride in the convenient
powder for oral solution form. This innovative formulation, which dissolves 25 times faster than tablets,
represents a significant advancement in patient compliance and their overall quality of life. Dipharma and
LogixX remain steadfast in their commitment to supporting the PKU and BH4 deficiency community, with
a continued presence in the UK territory and active engagement with healthcare professionals.”
He further highlighted, “All our Sapropterin Dihydrochloride products boast an impressive 36-month shelf
life, underscoring our dedication to environmental sustainability by reducing the risk of product wastage,
while also affirming the quality and stability of our drug.”
This achievement marks a significant milestone in Dipharma’s history, solidifying its presence with
Sapropterin Dipharma in all the major European countries.
Michael Close, CEO of LogixX Pharma, added, “We are proud to be part of this milestone, bringing
innovative solutions to patients in the UK. This partnership signifies our dedication to improving the lives
of individuals with PKU and BH4 deficiency with Swiss made products in a range of formulation and dosage
types for these important patient groups. We look forward to a continued fruitful collaboration with
About BH4-responsive phenylketonuria and BH4 deficiency
PKU is a rare, genetic metabolic disorder that affects an estimated 1 in 7000+ newborn infants. In those
affected, there is a reduction in the activity of the enzyme phenylalanine hydroxylase (PAH). This defect
impairs the degradation of the amino acid phenylalanine (Phe) into tyrosine (Tyr) and promotes
hyperphenylalaninemia. Left untreated, hyperphenylalaninemia can lead to progressive and irreversible
central nervous system damage. This deficiency manifests as mental and physical impairments, such as
loss of emotionality and inappropriate changes in behavior. Nowadays, PKU can be diagnosed and treated
early through newborn screening. In addition, PKU is linked to another rare condition, tetrahydrobiopterin
(BH4) deficiency disorder (incidence of 1 to 2 in 1,000,000). Here, the synthesis or regeneration of BH4,
the essential cofactor of PAH, is impaired. This results in hyperphenylalaninemia and a deficiency of the
monoamine neurotransmitters, dopamine, serotonin, noradrenaline and epinephrine. The effects on
those affected by these conditions depend on a particular genetic mutation that underlies the disease.
For more information on these diseases, please visit www.dipharma.ch/metabolic-diseases/.
Sapropterin dihydrochloride is a synthetic version of the naturally occurring 6R-BH4, which is a cofactor
of the hydroxylases for phenylalanine, tyrosine and tryptophan. Sapropterin formulations are
administered to patients with BH4-responsive phenylketonuria and BH4 deficiency. In patients with
phenylketonuria, sapropterin enhances the activity of the defective phenylalanine hydroxylase and
thereby increases or restores the oxidative metabolism of phenylalanine (“Phe”) sufficiently to reduce or
maintain blood Phe levels, prevent or minimize further Phe accumulation, and increase tolerance to Phe
intake in the diet. In patients with BH4 deficiency, sapropterin formulations complements the deficient
levels of BH4, thereby restoring the activity of phenylalanine hydroxylase.
Dipharma SA is a Swiss specialty pharmaceutical company, developing high quality, improved, medicines
for rare diseases. Dipharma SA is part of a third-generation group of family-owned companies that have
grown to a global presence.
With a portfolio of generic orphan products for the treatment of Hyperphenylalaninemia, Gaucher
Disease, Hereditary Tyrosinemia Type 1, Urea Cycle Disorders and others, Dipharma SA provides improved
solutions for patients affected by inborn metabolic diseases at an affordable cost and with a global reach.
For more information, please visit www.dipharma.ch
About LogixX Pharma
LogixX Pharma is a specialty pharmaceutical company based in Berkshire, United Kingdom. LogixX Pharma
was founded in 2010 and is an independent, privately owned entrepreneurial and dynamic enterprise –
with core competencies in the fields of pharmaceuticals and medical devices.
The management and treatment of niche therapeutic conditions represents an exciting growth
opportunity for LogixX. Many of these conditions are poorly treated with a high level of unmet medical
need, so we are proud to be a part of the solution for these patients.
LogixX Pharma’s mission is to bring new therapies to patients, new treatment approaches to healthcare
professionals and better health outcomes for all.
For more information, please visit www.logixxpharma.co.uk
Kuvan® is a registered trademark of Biomarin. In the European Union Kuvan® was approved in December
2008 for the treatment of hyperphenylalaninemia (HPA) in adults and pediatric patients of all ages with
phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency who have been shown to be responsive to
This press release may contain certain forward-looking statements. Although the Company believes its expectations are based on
reasonable assumptions, these forward-looking statements are subject to numerous risks and uncertainties, including scientific,
business, economic and financial factors, which could cause actual results to differ materially from those anticipated in the
forward-looking statements. The company assumes no responsibility to update forward-looking statements or adapt them to
future events or developments.
Dipharma SA and LogixX Pharma operates respectfully of any third-party IP rights and/or regulatory exclusivities that may exists
in each specific country.
This press release may contain information on pharmaceuticals that are not currently approved or available in your country or