During its May 2012 meeting, the Committee for Orphan
Medicinal Products (COMP) adopted a total of 16
recommendations for orphan designation. Orphan designation can be granted early
in the development process to substances that are intended for diagnosis,
prevention or treatment of life-threatening or very serious conditions that
affect not more than 5 in 10,000 people in the European Union (EU) or that
would not be developed without incentives.
One of these diseases for which no adequate treatment currently exists is
infection-associated haemolytic uraemic syndrome (HUS). During its meeting, the
COMP recommended granting an orphan designation for eculizumab used for the
treatment of this severe, life-threatening complication of enteric infections
with Shiga toxin-producing Escherichia coli bacteria, such as
enterohaemorrhagic E. coli, also called EHEC.
Infection-associated HUS occurs predominantly in childhood. Although it is
a very rare disease, estimated to occur in less than 0.07 per 10.000 patients a
year, infection-associated HUS is recognised as the most common cause of acute
kidney failure in infants and young children. It can also affect adults
and the elderly, who often die as a result of the disease.
Eculizumab, a monoclonal antibody, is already authorised in the EU for the
treatment of paroxysmal nocturnal haemoglobinuria, a rare, life-threatening
genetic disease that causes the red blood cells to be broken down too quickly.
Its mechanism of action is thought to be also effective in patients with
infection-associated HUS.
The COMP also recommended granting of an orphan designation for recombinant
human interleukin-7 for the treatment of progressive multifocal
leukoencephalopathy (PML). PML is a severe demyelinating disease of the central
nervous system caused by the JC virus. It is seen in many conditions that
affect immune system function such as HIV-1/AIDS, certain cancers and organ
transplant subject to immunosuppressive therapies. Over the last five to 10
years, it has been increasingly reported as a serious adverse event of therapeutic
monoclonal antibodies that modulate the immune system.
PML is a very rare disease, affecting less than 4 out of a million
patients. There are currently no medicines available to treat PML or to prevent
JC virus infection. The granting of an orphan designation is intended to
encourage the development of a medicine for the treatment of this disease,
which often leads to death or serious disability.
For the full list of opinions adopted by the COMP during its May 2012
meeting, see the COMP
monthly report.
Next steps
The COMP recommendations for these substances are sent to the European
Commission for the adoption of a decision. Once an orphan designation has been
granted, sponsors have access to certain incentives during the development of
the medicine.
For each medicine that has been granted an orphan designation, the Agency
publishes a summary of the opinion on its website.