Leigh syndrome and orphan drugs

31.1.2018

Question for written answer E-000571-18
to the Commission
Rule 130
Nicola Caputo (S&D)

According to European Medicines Agency data there are more than 6 000 rare diseases in Europe, and estimates put the total number of sufferers in the EU countries at roughly 30 million.

This category of diseases includes Leigh syndrome, or subacute necrotising encephalomyelopathy, a rare progressive neurological disease. Life expectancy is no more than a few years in most cases, and the condition affects about 1 in 36 000 newborns; 10%‐30% of patients have mitochondrial mutations. Prenatal diagnosis is possible only in some cases, and at present no specific treatment is available.

Pharmaceutical companies tend to be reluctant to develop orphan drugs for rare diseases.

Within its own sphere of responsibility:

What measures will the Commission take to provide greater incentives for research into orphan drugs and the treatment of rare diseases?

What strategies has it employed, or will it employ, to encourage European scientific research into Leigh syndrome?