This Blog Post discusses recent global non-US regulatory events for orphan drugs.
I – Europe
At a January meeting, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) gives the following opinions for three orphan drugs :
• Positive recommendation for Bayer’s Adempas (Riociguat) for the treatment of Chronic Thromboembolic Pulmonary Hypertension (CTEPH) and Pulmonary Arterial Hypertension (PAH)
• Negative opinion for AB Science’s Masiviera (Masitinib) which is intended for the treatment of non resectable locally advanced or metastatic pancreatic cancer
• Negative opinion for PTC Therapeutics’ Translarna (Ataluren) which is intended for the treatment of Duchenne Muscular Dystrophy.
Also, EMA’s Committee for Orphan Medicinal Products (COMP) issues 15 positive opinions for ODD at their January meeting (Reference Blog Post). These ODDs are to be presented to the European Commission (EC) for final approval. If the EC approves these ODDs, the drugs receive ODD in the…
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