The Debiopharm Group (Debiopharm) has recently received European orphan drug designation from the Commission of the European Community (EC) for Debio-0513 (formerly known as PTR-262), a dual altered ligand peptide analog of the myasthenogenic epitopes of the acetylcholine receptor (AchR) for the treatment of myasthenia gravis (MG).
The following are the criteria for the designation of European Orphan Drug Status:
The Commission for the EC found that DEBIO-0513 met the above requirements for the treatment of MG, a rare disease affecting approximately 0.7 to 1.5 in 10,000 people in Europe.
According to Loic Maurel, President and CEO of the Debiopharm Group Canadian subsidiary:
"Due to its unique mechanism of action, we hope that DEBIO-0513 will become an important novel treatment opportunity for MG sufferers.
It has the potential to offer patients benefits such as modifying the progressive course of the disease, preventing relapses, and possibly working in combination with other available treatments.
The sparing effect of DEBIO-0513 should decrease the risks usually related to current medications like steroids and other immunosuppressant.”
Read the full press release.
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