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Rare Daily Staff Proposed changes to the European Union’s Orphan Medicinal Product Regulation will reduce investment in […]
Read moreFTC Temporarily Withdraws Challenge to Amgen-Horizon Acquisition in Effort to Settle
Rare Daily Staff The U.S. Federal Trade Commission said it has temporarily withdrawn its administrative complaint of […]
Read moreEMA Approves First Digital Outcome Measure as Primary Endpoint in Pivotal Trials
Rare Daily Staff The European Medicines Agency said that it will accept a digital endpoint for ambulant […]
Read moreFTC Sues to Block Amgen’s $27.8 Billion Acquisition of Horizon
Rare Daily Staff The Federal Trade Commission filed a lawsuit to block Amgen’s acquisition of Horizon Therapeutics, […]
Read moreEMA Issues Draft Paper on Single-Arm Trials as Pivotal Evidence for Approval
Rare Daily Staff The European Medicines Agency has issued a draft paper on the use of single-arm […]
Read moreEuropean Commission Approves Amicus’ Pombiliti in Patients with Late-Onset Pompe Disease
Rare Daily Staff The European Commission has granted approval for Amicus Therapeutics’ Pombiliti, a long-term enzyme replacement […]
Read moreEMA Amends Policy of Orphan Designations of Therapies for Inherited Retinal Dystrophies
Rare Daily Staff The European Medicines Agency’s Committee on Orphan Medicinal Products said it has amended its […]
Read moreCAMP4 Raises $100 Million to Accelerate Expansion of Regulatory RNA-Targeting Platform and Advance Rare Disease Pipeline
Rare Daily Staff CAMP4 Therapeutics said it closed a $100 million series B round to advance the […]
Read moreFDA Releases Action Plan for Rare Neurodegenerative Diseases, Including ALS
Rare Daily Staff The U.S. Food and Drug Administration unveiled its Action Plan for Rare Neurodegenerative Diseases, […]
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