Related
FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies
Rare Daily Staff The U.S. Food and Drug Administration announced a pilot program aimed at accelerating the […]
Read moreEuropean Trade Group Warns Proposed Overhaul to Orphan Regulations Decrease Innovation and Hurt Patients
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Rare Daily Staff The U.S. Federal Trade Commission said it has temporarily withdrawn its administrative complaint of […]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 more