Orphan drugs – market exclusivity outside patent protection

Medicinal products can be protected but not only by patents. In the case of diseases for a small number of patients (rare diseases), protection as so-called "orphan drugs" is also possible. These are medicines for the prevention, diagnosis and treatment of rare diseases.

In the EU, medicines for life-threatening or chronically and seriously debilitating diseases affecting no more than 5 in 10,000 people can be designated as orphan through a voluntary procedure with the Committee for Orphan Medicinal Products (COMP) (see here). In other countries, e.g. the USA, Japan and Australia, there are comparable regulations.

This enables protection or exclusivity even in cases with a small market and therefore low turnover, which would not be interesting for patent protection. Orphan drugs are also approved centrally by the European Medicines Agency (EMA), which enables fee reductions for scientific advice and the regulatory procedure, among other things.

An overview of medicinal products with this status in the EU can be found in the Union Register of orphan medicinal products for human use of the European Commission here: Union Register of medicinal products

Drugs for special purposes (indications) that are otherwise more widely applicable may also be registered here. Randomly selected examples would be the monoclonal antibody rituximab (used, among other things, in cancer immunotherapy and against autoimmune diseases) for the treatment of primary membranous nephropathy or the treatment of amyotrophic lateral sclerosis with the small-molecule tyrosine kinase inhibitor sotuletinib, which is still being tested in clinical trials.

Examples of use in rare diseases would also include treatment of inclusion body myositis with Ulviprubart, treatment of gliomas with CD34+ haematopoietic progenitor and stem cells transduced with a lentiviral vector, encoding the interferon α 2 gene, or as a diagnostic agent iodine (^124I) evuzamitide in the diagnosis of AL amyloidosis, chosen completely at random to illustrate the range from simple organic molecules to cellular therapeutics and diagnostics.

Orphan drug status in the EU implies that 10 years after obtaining marketing authorisation, following a simplified marketing authorisation procedure (which takes place after registration as an orphan drug), there is exclusivity for the applicant of the orphan drug - i.e. exclusivity analogous to that conferred by a patent - for the registered indication. In the case of paediatric indications, the exclusivity can be extended for a further two years.

The aim of "market exclusivity" is to encourage the development of drugs for rare diseases by protecting these drugs - for the protected indication - from competition with (also similar and "closely related") drugs with similar indications, which then cannot be marketed during the exclusivity period. One criticism here is that the definition of closely related medicines may mean that third parties could not develop their own medicines (see here).

A ruling by the Munich I Regional Court of 4 August 2023 (Case No. 21 O 6235/23) essentially confirmed a previously issued interim injunction for infringement of the right to market exclusivity. The pharmaceutical companies (defendants) sued by the holder of the orphan drug market exclusivity right (plaintiff; a pharmaceutical company) were ordered to cease and desist the distribution of their drug. The exclusivity concerned the use of "Eculizumab", a humanised monoclonal antibody, by a biosimilar of this antibody.

A ruling by the Munich I Regional Court of 4 August 2023 (Case No. 21 O 6235/23) essentially confirmed a previously issued interim injunction for infringement of the right to market exclusivity. The pharmaceutical companies (defendants) sued by the holder of the orphan drug market exclusivity right (plaintiff; a pharmaceutical company) were ordered to cease and desist the distribution of their drug. The exclusivity concerned the use of "Eculizumab", a humanised monoclonal antibody, by a biosimilar of this antibody.

The product was registered as an orphan drug for four indications (paroxysmal nocturnal haemoglobinuria (PNH), atypical haemolytic uraemic syndrome (aHUS), refractory generalised myasthenia gravis (gMG) and neuromyelits optica spectrum disorders (NMOSD). The market exclusivity right for PNH had already expired. The defendants (they had already withdrawn their product from the market after the injunction) were nevertheless obliged to refrain from marketing in the other three indications as well. The board ruled that the injunction defendants, as so-called indirect interferers, had made an adequate-causal contribution, for example through letters of recommendation, to the cross-indication use of their drug also in the three indications still protected for the plaintiff's group of companies. The risk of infringement made credible by the plaintiff in the injunction had not been eliminated by the defendants in the injunction by the relevant conclusion of the oral proceedings.

See Regional Court of Munich I, press release of 04.08.2023

The Board also confirmed that the applicant had a civil right of prohibition; the relevant Regulation 141/2000/EC was not only about authorisation, but this also included the possibility for the holder to take individual action against circumvention of his right.

We would be happy to advise you on this alternative to patent protection in the case of orphan drugs and diagnostics.

For further information and to contact the author