Business Wire

Rare Diseases: PKU GOLIKE® Debuts in Germany

Share

PKU GOLIKE®, the brand new generation of Phenylalanine (PHe)-free medical food for Phenylketonuria (PKU) dietary management, is now available in Germany and Austria, promoted and distributed directly by APR Applied Pharma Research s.a. (APR) through its local subsidiary, APR Deutschland.

The company has taken advantage of the 33th Annual Meeting of the APS (German Association for Pediatric Metabolic Medicine) in Fulda to showcase to healthcare professionals dealing with PKU the distinctive features of the patented amino acids (AAs) mixtures and the pharma technology enabling it – the Physiomimic™ Technology.

This conference, summoning every year more than 300 pediatricians from Germany, Austria and Switzerland, gives the participants the opportunity to be updated on the most significant advancements in metabolic diseases as well as new therapeutic approaches.

Germany is the biggest country in Europe in terms of prevalence of PKU and population of patients under treatment. According to IMS data, the outlook of the German market for PKU medical food is very positive, showing a growth rate of 9% on a yearly basis, driven mostly by products addressed to patients from 3 years of age onwards.

As confirmed by both German HCPs and patients interviewed during an international market research, palatability (taste and odor) of AA formulations is still one of the key criteria when deciding on a specific AA supplement, as it impacts significantly on the long-term compliance to the dietary treatment and consequently on the overall clinical outcomes.

PKU GOLIKE®, developed by APR and powered by the proprietary Physiomimic™ Technology, could offer a new dietary treatment option to the benefit of patients and HCPs.

During the event, physicians who visited the company booth learned about the key benefit of the innovative AAs mix – the prolonged absorption of AAs, how it makes PKU GOLIKE® a major step in PKU dietary management towards a more efficient AA utilization as well as the scientific data supporting its profile.

Physicians had also the chance to literally taste the unique formulation in special coating micro granules, experiencing how the Physiomimic™ Technology is able to mask the unpleasant taste and odor of free AAs. The audience agreed on the extent of this significant advantage for PKU patients’ compliance to life-long dietary regimen, while appreciating how PKU GOLIKE® could be easily integrated into the daily food routine.

“With PKU GOLIKE® our ambition is to set a new pace in PKU management by providing healthcare professionals, patients and caregivers with a treatment option which can make a real change in their lives.” said Paolo Galfetti, CEO of APR. “In Italy where we launched the product last October with our own sales & marketing organization, we are indeed receiving extremely positive feedbacks from physicians who have already recommended PKU GOLIKE® to a promising number of patients. We are confident this will soon happen also in Germany, a strategic market for us, where again we will be in direct control of the distribution and promotion of the product”.

To further support PKU GOLIKE® awareness among the healthcare professionals dealing with PKU, APR Deutschland is going to participate to the forthcoming congress of the VDD (German Dieticians Association) in Wolfsburg beginning of May, where scientific data will be divulged, while other initiatives are being prepared both on a local and international basis.

About Phenylketonuria or PKU

Phenylketonuria or PKU is a rare, genetic, recessive metabolic disorder affecting about 50.000 people worldwide. PKU is characterized by the deficiency or the malfunctioning of a liver enzyme needed to process phenylalanine (“Phe”). PKU has to be treated through a strict, life-long, low-protein (low-Phe) dietetic treatment combined with a daily assumption of low-protein modified foods and medical food or irreversible mental and physical disabilities will develop.

About PKU GOLIKE®

PKU GOLIKE is an innovative food for special medical purposes (FSMP) consisting of a Phe-free amino acid mixture. For the first time a pharmaceutical technology has been applied to a FSMP and the innovative PKU GOLIKE, engineered with Physiomimic technology™, provides a prolonged-released of amino acids and grants a remarkable taste and odor masking while preventing aftertaste. This prolonged release leads to a physiological absorption of amino acids, similar to dietary proteins, to favor a more efficient amino acid utilization and health benefits. The improvement of free-amino acids' organoleptic features aims to support compliance to the nutritional management of the diet for a better quality of life.

The product line is formed by: PKU GOLIKE PLUS 3-16 & PKU GOLIKE PLUS 16+ with amino acids, vitamins & minerals, and PKU GOLIKE PURE 3+ with only amino acids.

For more information about PKU GOLIKE® and the Physiomimic™ Technology, please visit: www.apr.ch/apr-pharma-products/medical-prescription/genetic-metabolic-disease/

About APR Applied Pharma Research s.a.

APR is a Swiss independent pharma company focused on development and global commercialization of innovative, research-driven products designed to address unmet needs in specialized therapeutic areas and rare diseases. APR combine pharmaceutical development expertise with proprietary drug delivery technologies to realize solutions that meaningfully improve the lives of people with rare diseases and empower families through novel approaches to disease management. A diverse and balanced portfolio of revenue-generating products in all major markets is complemented by a robust pipeline of innovative products at different stages of development for the treatment of recessive metabolic disorders, as well as rare dermatological and ocular diseases. Products are directly commercialized by APR through inhouse sales and marketing teams in strategically important countries across Europe and a growing worldwide network of commercial partners.

Contact information

APR Applied Pharma Research s.a.:
Elisabetta Bianchi, Corporate Communication Manager
elisabetta.bianchi@apr.ch
+41 91 6957020

About Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Celebrity Stylist Tara Swennen and Glamhive Founder Stephanie Sprangers Once Again Bring Together Some of the Biggest Names from Around the World in Style, Fashion, Beauty and Women’s Empowerment for Groundbreaking Digital Event8.7.2020 22:20:00 CESTPress release

Celebrity stylist Tara Swennen and Glamhive founder Stephanie Sprangers will once again bring together some of the biggest names from around the world in style, fashion, beauty and women’s empowerment for the Glamhive Digital Summer Bazaar—a groundbreaking digital event. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200708005857/en/ Adir Abergel, Celebrity Hair Stylist (Photo: Glamhive) During Glamhive’s first digital event, Glamhive LIVE Style Summit, audience members were asked to engage with panels through live, real-time question and answer sessions. The conversations ranged from intimate questions about how to break down gender barriers when starting a business to how to find ways to break in into business through marketing. Due to that event’s overwhelming success, Swennen and Sprangers decided to raise the bar for their next global event. “The style, beauty, and designer communities are a close-knit one; many of us h

International WELL Building Institute Opens Enrollment for WELL Health-Safety Rating In Response to COVID-19 With Significant Early Adoption8.7.2020 19:18:00 CESTPress release

The International WELL Building Institute (IWBI) announced that enrollment has opened today for the WELL Health-Safety Rating for Facility Operations and Management, with more than 100 organizations and real estate portfolios across the globe already enrolled. Organizations across a wide range of industries and geographic locations have responded swiftly to implement the program’s science-backed guidance as they seek to instill confidence and trust among their staff, stakeholders and the broader community. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200708005768/en/ The WELL Health-Safety Rating is an evidence-based, third-party verified rating for all new and existing building and space types focused on operational policies, maintenance protocols, emergency plans and stakeholder engagement strategies to help organizations prepare their spaces for re-entry in a post COVID-19 environment. Adapted from features in the WELL

Croma-Pharma announces submission for their botulinum toxin to treat glabellar (frown) lines to the German authority BfArM8.7.2020 15:00:00 CESTPress release

On July the 8th, Croma-Pharma GmbH (Croma) has stated that the company submitted the drug file of its botulinum toxin product to the German authority BfArM. The filing of the dossier stands for the next essential step towards Croma’s complete aesthetic portfolio in Europe, which already comprises of a comprehensive HA filler range, threads, PRP and skincare products. Croma has licensed the product from the Korean toxin producer Hugel Inc. for Europe and established a joint venture company in 2018 with Hugel, Inc., in order to develop and commercialize Croma’s HA filler products together with Hugel’s botulinum toxin product in the US, Canada, Australia and New Zealand. The filing to BfArM marks the first milestone in Croma’s approach to a European wide regulatory approval. “We are pleased to announce that our toxin has finally been submitted – since quite some time hence, a European company will launch a botulinum toxin, which makes us both, happy and proud. Combining our present produc

Esri President Jack Dangermond Addresses UN High Level Political Forum8.7.2020 15:00:00 CESTPress release

Esri, the world leader in GIS, location intelligence, and mapping technology, today announced that its founder and president, Jack Dangermond, addressed the United Nations’ High Level Political Forum on Sustainable Development. His remarks to the assembly were delivered July 7th at the key annual meeting for UN and Member State review of the Sustainable Development Goals (SDGs) globally. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200708005264/en/ The United Nations Sustainable Development Goals (SDGs) are 17 goals with 169 targets that all 191 UN Member States have agreed to try to achieve by the year 2030. Even as the global community has come together to battle a raging pandemic, it continues to face multiple complex issues all related to the SDGs. The response to COVID-19 has demonstrated the power of data and technology to increase organizations’ effectiveness and impact. Esri users, such as Johns Hopkins University

Fluence Leads Global Research Initiative to Study Impact of Light Quality on Plant Development, Yield and Crop Quality8.7.2020 14:00:00 CESTPress release

Fluence by OSRAM (Fluence), a leading global provider of energy-efficient LED lighting solutions for commercial cannabis and agriculture production, has expanded its global photobiology research program, which encompasses studies on multiple vine crops, leafy greens and medical cannabis in the United States, Canada, Germany, Belgium and the Netherlands. Tapping into a global network of trusted research institutions Fluence leverages a network of leading research institutions and partners for its program, including Wageningen University & Research (WUR) for tomatoes; Proefstation voor de Groenteteelt (Proefstation) to study cucumbers; Harrow Research and Development Centre for peppers; The Technical University of Munich's Greenhouse Lab Centre for lettuce; Wageningen Plant Research’s Greenhouse Horticulture business unit and Compassionate Cultivation for medical cannabis. The latest studies utilized Fluence’s VYPR Series top light and expanded PhysioSpec™ spectra offering—which features

Takeda and the New York Academy of Sciences Announce 2020 Innovators in Science Award Winners8.7.2020 14:00:00 CESTPress release

Takeda Pharmaceutical Company Limited (“Takeda”) (TSE:4502/NYSE:TAK) and the New York Academy of Sciences announced today the Winners of the third annual Innovators in Science Award for their excellence in and commitment to innovative science that has significantly advanced the field of rare disease research. Each Winner receives a prize of US $200,000. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200708005039/en/ The 2020 Winner of the Senior Scientist Award is Adrian R. Krainer, Ph.D., St. Giles Foundation Professor at Cold Spring Harbor Laboratory. Prof. Krainer is recognized for his outstanding research on the mechanisms and control of RNA splicing, a step in the normal process by which genetic information in DNA is converted into proteins. Prof. Krainer studies splicing defects in patients with spinal muscular atrophy (SMA), a devastating, inherited pediatric neuromuscular disorder caused by loss of motor neurons, res