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EMA grants marketing authorisation for Prevymis against cytomegalovirus
10 November 2017 | By
The EMA has recommended granting a marketing authorisation Prevymis, an antiviral medicine that prevents CMV reactivation and disease...
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Orphan Drugs
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FDA approves Zelboraf for certain patients with Erdheim-Chester disease
The FDA has expanded the approval of Zelboraf to include the treatment of certain adult patients with Erdheim-Chester Disease, a rare cancer of the blood...
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FDA grants orphan drug status to Cellect’s ApoGraft
The FDA has granted orphan drug designation for ApoGraft for the prevention of acute and chronic graft versus host disease...
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FDA approves Mylotarg for treatment of acute myeloid leukemia
The FDA has approved Mylotarg for the treatment of adults with newly diagnosed acute myeloid leukaemia whose tumours express the CD33 antigen...
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FDA approves new targeted treatment for acute myeloid leukemia
The U.S. Food and Drug Administration has granted the approval of Idhifa to Celgene Corporation and RealTime IDH2 Assay to Abbott Laboratories...
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UK bioscience companies form Rare Disease Industry Group
The BIA is committed ensuring patient access to treatments for rare and very rare conditions, sometimes referred to as orphan and ultra-orphan medicines...
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Regulation of orphan drugs within the EU
Simply put, an orphan drug is indicated for the treatment of a very rare disease. Such diseases are often due to genetic defects and therefore children and newborns are affected to a great extent1.
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Issue #3 2017 – Digital version
In Issue #3 2017: In-Depth Focuses covering Raman Spectroscopy, Separations & Purifications, Formulation Development & Delivery; Guide To Testing Services; GMP and mutual recognition; Regulatory Insight; RMMs; Microbiology Series; and much more...
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The Good, the Bad, and the Donald: what Pharma can expect from 2017
11 January 2017 | By Niamh Marriott, Digital Editor
From an orphan drug sales boom to uncharted political landscapes, the CPhI Worldwide expert panel predict pharma’s biggest opportunities and threats in 2017.
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Cancer treatment receives EU drug designations
20 July 2016 | By Niamh Louise Marriott, Digital Content Producer
With the EU orhpan drug designations, Cell Medica can now obtain regulatory and financial incentives for developing and marketing CMD-003 as a cancer...
