Orphan Medicinal Product designation
by the European Commission provides certain regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union and has the potential to be of significant benefit.
The European Commission has granted orphan medicinal product designation
to avapritinib for the treatment of GIST.
Y-mAbs Therapeutics announced that the Committee for Orphan Medicinal Products, or COMP, of the European Medicines Agency, or EMA, has recommended the granting of orphan medicinal product designation
, or OMPD, in the European Union for naxitamab, one of the company's lead product candidates, for the treatment of relapsed or refractory high-risk neuroblastoma.
Received Orphan Medicinal Product Designation
from the European Medicines Agency for EDIT-101.
The subsidiary will allow AveXis to move forward with its plans to open clinical trials in the European Union and leverage European Medicines Agency's programs, such as Orphan Medicinal Product Designation
and the Micro-, Small-, and Medium-Sized-Enterprise (SME) program, to advance development.
The European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) officially granted the company's hESC-derived RPE cells orphan medicinal product designation
for the treatment of SMD.
(Nasdaq:XOMA), Berkeley, Calif,, a leader in the discovery and development of therapeutic antibodies, announced that the Committee for Orphan Medical Products of the European Medicines Agency (EMA) has adopted a positive opinion and recommends the granting of orphan medicinal product designation
for XOMA 052, an antibody to interleukin-1 beta, for the treatment of Behcet's disease.
Quark Pharmaceuticals, Inc., Fremont, CA, announced the European Commission has graphed Orphan Medicinal Product Designation
for QPI-1002 (also referred to as I5NP) for the prophylaxis of delayed graft function (DGF) in kidney transplant recipients.
Myozyme has received orphan medicinal product designation
, which applies to treatments for diseases that affect fewer than 5 in 10,000 people in the European Union.
The Orphan Medicinal Product Designation
from the European Commission has the potential to provide for up to ten years of market exclusivity in the European Union.
The product has already received orphan drug, fast track, and regenerative medicine advanced therapy designations from the US Food and Drug Administration (FDA), and orphan medicinal product designation
from the European Medicines Agency (EMA).
With the ability to communicate closely and often with the FDA, we are hopeful that we can expedite our commitment to bring a safe and effective therapy to patients in need." AAV-CNGB3 has been granted orphan drug designation by the FDA and orphan medicinal product designation
by the European Medicines Agency, as well as rare pediatric disease designation by the FDA and PRIME designation by the EMA, for the treatment of ACHM caused by mutations in the CNGB3 gene.