Stolmár & Partner successfully secures EMA Orphan Drug Designation for the Treatment of amyotrophic lateral sclerosis (ALS) for Federal Institute of Technology (Ecole Polytechnique Fédérale ) Lausanne (EPFL), Switzerland.
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Stolmár & Partner is happy to announce that the European Medicines Agency (EMA) has granted orphan drug designation (EU/3/18/2008) on 16 April 2018 to Adeno-associated viral vector serotype 9 encoding miRNA against human superoxide dismutase 1 for the treatment of amyotrophic lateral sclerosis for its client Ecole Polytechnique Fédérale Lausanne (EPFL), Switzerland, where S & P acted as EU sponsor.
Amyotrophic lateral sclerosis (ALS) is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. The exact causes are unknown but are believed to include genetic and environmental factors. The symptoms of ALS depend on which muscles weaken first, and include loss of balance, loss of control of hand and arm movement, and difficulty speaking, swallowing and breathing. ALS usually starts in mid-life and men are more likely to develop the disease than women.
ALS is a debilitating and life-threatening disease because of the gradual loss of function and its paralyzing effect on muscles used for breathing, which usually leads to death from respiratory failure.
This novel medicine might be of significant benefit for patients with ALS. Laboratory studies suggested that the medicine could lead to improvements in the ability to move.
The medicine is made of a virus that contains small fragments of genetic material (RNA) which are expected to interfere with the production of the defective SOD1 protein in nerve cells. This is expected to reduce the production of defective SOD1 and help relieve the symptoms of the disease. We are encouraged that the EMA has recognized the potential of this important contribution to the treatment of ALS.
The type of virus used in this medicine (adeno-associated virus) does not cause disease in humans. For more information please see the attached PDF.
EMA orphan designation is intended to encourage development of medicines for the diagnosis, treatment, or prevention of life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in the EU. Medicines that receive orphan designation are eligible for a number of incentives, including assistance with development of the medicine; reduced fees for marketing-authorization applications; and extended market exclusivity once the medicine is authorized. Orphan designation is conferred following a positive opinion by the EMA's Committee for Orphan Medicinal Products (COMP).
S & P is happy to have secured to its client EPFL the first orphan drug designation ever for a University in the EU. S & P thanks our regulatory affairs lead attorney, Dr. Leopold Gruner for his success for our client.
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