our expertise

Orphan Drug Research & Development

Standard clinical trials are not feasible for orphan drug development. Patients exhibit a wide range of heterogeneous symptoms often needing individually tailored regimens. The use of placebo controlled trials is usually not ethically possible and standardized treatments are not available for comparison. Since the very few patients are scattered all over the world, it is virtually impossible to bring them together in one trial.

Working closely with physicians, Orphan Europe designs tailor-made clinical programmes that define the key evaluation criteria for each drug. To this end we have developed a unique external network of experts who support and cooperate closely with us.

Regulatory Affairs

In 2009, Orphan Europe received its 7th market authorisation in Europe. You will find our current product portfolio here.

Building a marketing authorization file with very low patient numbers requires specialist knowledge and a solid working relationship with regulatory authorities. Orphan drugs undergo the same review processes as other pharmaceuticals; they therefore need to meet the same high standards of quality, safety and efficacy as all other medicines.

Orphan Europe has 19 years worldwide regulatory experience, including:
- orphan drug designation
- marketing authorization application
- post approval regulatory maintenance.

At Orphan Europe we are always conscious that patients are waiting for treatment.

Production and Distribution

Producing, packaging and distributing orphan drugs demands flexibility and cost-effectiveness while keeping pharmaceutical industry quality standards. At Orphan Europe every production stage follows Good Manufacturing Practice (GMP) standards. All products are produced in country-specific packaging covering 27 languages.

Orphan Europe believes patients should have access to treatment as soon as a diagnosis is made, therefore we distribute directly to the patient’s hospital or pharmacy. The company works closely with the quickest worldwide transport services to ensure prompt and safe delivery. Orphan Europe makes over 14,000 shipments per year to Europe, the Middle East, Africa, Asia and the Americas, and all product boxes are traceable. We also provide emergency deliveries when necessary.

An example of what we can do

In 1995, Orphan Europe became aware of an extremely rare metabolic disease called N-Acetylglutamate Synthase Deficiency. Only a few patients in Europe were correctly diagnosed and able to benefit from treatment with a structural analogue of the defective molecule. The molecule was produced in a powder form that needed to be prepared by hospital pharmacists.

Orphan Europe decided to take on the challenge of transforming the chemical into a medicine in accordance with pharmaceutical standards. In 1997 appropriate methods to synthesise the molecule were identified. Pharmaceutical and toxicological studies were performed and a registration dossier compiled for market authorisation. All the complex “orphan” parameters were involved in the development of this new drug (few patients, small production batches, doses suitable for infants and adults, different country requirements…) During the drug’s development, patient data was (and still is) continually recorded to confirm the product’s efficacy and safety.

On October 18, 2000 the product was granted an EU Orphan Designation by the European Commission and EU registration was granted in January 2003.