orphanix’ mission is to improve the lives of newborns with rare diseases by filling blind spots with safe, innovative and readily accessible medical solutions. We are dedicated to fundamentally understand the needs of affected babies and to facilitate innovative opportunities for treatments.
We identify, develop and commercialize innovative orphan drugs across all paediatric indications based on in-depth evaluation of academic and real-world data.
orphanix stands for fast-track solutions and effective treatments by focusing on indications where there is
- High unmet medical need
- High level clinical evidence
- Globally no adequate product available fulfilling the needs of a particular rare disease
- High probability to gain intellectual property rights and orphan designations from Food and Drug Administration (USA) and European Medicines Agency (EU) implying market exclusivity after authorization.
We are dedicated to develop innovative solutions for high unmet medical needs in the paediatric rare disease space and to complement portfolios of fully integrated pharmaceutical companies.
Founders & Network
By founding orphanix in 2015 we followed our vision of joint forces of
- profound R&D know-how,
- academic expertise in paediatrics and
- the world leading neonatal web community.
Our competence synergy is key to filling the blind spots in paediatric medicinal care by providing effective orphan medicines.
Managing Director & Founder
has extensive industry experience, incl. global roles in Research & Development. Successfully bringing life-science projects from the visionary idea to successful market introduction is his strength and passion.
Co-Founder & Chief Medical Officer
is Consultant Neonatologist and Associate Professor at Karolinska Insitutet, Sweden and Founder of 99nicu, the world leading neonatal web community. Preterm infants in Intensive Care are his daily business.
Together Philipp Novak and Stefan Johansson also founded Neobiomics AB, a Stockholm based start-up providing ProPrems®, a dietary supplement specifically developed for the needs of preterm infants.
About Orphan Drugs
Orphan drugs are medicinal products treating rare diseases, known as orphan diseases. A rare disease is any disease that affects a very small percentage of the population, and is fatal or severely debilitating. Given the small number of patients an orphan disease is often not “adopted” by the pharmaceutical industry – hence the expression “orphan” drug.
The EU defines a disease as rare if not more than 5 in 10,000 people are affected. In the USA a disease is considered rare if not more than 200,000 patients are affected in total. There are over 7,000 known paediatric rare diseases worldwide affecting several million people over the world – 30 million people in Europe alone. Until today, only up to 300 orphan diseases have adequate treatments.
Around 80% of rare diseases are genetic in origin or can be partially traced back to genetic causes. This means that many rare diseases have their onset in childhood. They are often chronic causing a long life of suffering for the patients.
orphanix provides innovative medicinal products to improve the lives of children with rare diseases.