A spinal cord therapy being developed by a Universitat Autònoma de Barcelona (UAB) team has gained EMA orphan status, which could help speed its development.
The designation means the compound, based on the bioactive lipid molecule Maresin-1 (MaR1), could offer relevant clinical benefit in an area of high unmet medical need.
Spinal cord injuries have an estimated incidence of between 20 and 45 cases per million inhabitants per year.
There is currently no pharmacological treatment that improves neurological recovery after injury.
The only drug approved in the acute phase is methylprednisolone, the use of which is discouraged in many countries because of its adverse effects and limited efficacy.
MaR1 is a lipid produced by macrophages, which are immune system cells, during the resolution phase of inflammation.
It acts to promote the end of the inflammatory process and the protection of tissue.
The treatment is being investigated by the Neuroplasticity and Regeneration Group of the Institut de Neurociències and the department of cell biology, physiology and immunology at the UAB, led by Rubèn López-Vales.
In animal models, the compound has shown a strong ability to significantly reduce post-injury inflammation and promote processes that limit neurodegeneration, or the loss of nerve cells. These effects translate into a substantial improvement in locomotion recovery.
Rubèn López-Vales said: “This designation is the recognition of many years of research in the biology of inflammation resolution and in spinal cord injury models and allows us to move forward towards the goal of one day being able to offer therapy to people affected by this serious condition.”
The EMA’s orphan drug designation brings regulatory advantages that can speed up the development of the compound, including priority scientific advice and accelerated evaluation pathways. It also protects marketing exclusivity if the treatment reaches the market.
To move towards a clinical trial, the team must complete regulatory preclinical studies to support product safety and establish a fully validated manufacturing process.
It must also secure approval from the regulatory agency and ethics committee for the documentation submitted by the research team.
These steps are expected to take about three to five years before a patient trial can begin.
