orphan drugs Tag

01 Dec

Posted at 12:42h in Allergies, AstraZeneca, Author Interviews
MedicalResearch.com Interview with: [caption id="attachment_46299" align="alignleft" width="125"]Mr. Tosh Butt Vice President, Respiratory AstraZeneca Mr. Butt[/caption] Mr. Tosh Butt Vice President, Respiratory AstraZeneca Mr. Butt discusses the recent announcement that the FDA has granted Orphan Drug Designation for Fasenra for the treatment of Eosinophilic Granulomatosis with Polyangiitis.  MedicalResearch.com: What is the background for this announcement? Can you tell us a little more about Eosinophilic Granulomatosis with Polyangiitis/Churg Strauss? How does it differ/resemble severe eosinophilic asthma?
  • The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for FASENRA™ (benralizumab) for the treatment of Eosinophilic Granulomatosis with Polyangiitis (EGPA). The ODD application was based on epidemiology demonstrating the rarity of the disease (<200k US patients) and a scientific rationale that FASENRA may benefit patients with this condition. The core role of eosinophilia in EGPA and FASENRA’s demonstrated eosinophil-depleting properties provided this rationale and suggest it may deliver benefit to patients with EGPA.
  • EGPA is a rare autoimmune disease that can cause damage to multiple organs and tissues. EGPA is characterized by inflammation of blood vessels and the presence of elevated levels of eosinophils, a type of white blood cell. All patients with EGPA have very high levels of eosinophils at some point in their disease. FASENRA induces rapid and near-complete depletion of eosinophils in the blood and has proven efficacy in severe eosinophilic asthma, which suggest it may deliver benefit to patients with EGPA. 

28 Oct

Posted at 14:46h in Author Interviews, Pediatrics
MedicalResearch.com Interview with: [caption id="attachment_45547" align="alignleft" width="200"] Michael Snape, Ph.D., chief executive officer and chief scientific officer of AMO Pharma Dr. Michael Snape[/caption] Michael Snape, Ph.D. Chief executive officer Chief scientific officer AMO Pharma  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: This is a further analysis of the Phase II Proof of Concept study that was described in March.  We have extended the findings from that study by performing further analyses of the data obtained and also commencing an analysis of the relationship between the levels of AMO-02 involved and the clinical response seen. The main finding is a confirmation that our previously reported conclusions are supported.  The concordant trend analysis revealed a clear dose-response relationship that favored the 1000 mg over 400 mg dose for four of the 10 response variables differed in favor of 1000 mg over 400 mg dose on key clinician and caregiver measures, autism scores and grip strength. 

04 Jul

Posted at 18:50h in Author Interviews, BMJ, Cost of Health Care
Igho Onakpoya MD MSc Clarendon Scholar University of Oxford Centre for Evidence-Based Medicine Nuffield Department of Primary Care Health Sciences Oxford UKMedicalResearch.com Interview with: Igho Onakpoya MD MSc Clarendon Scholar University of Oxford Centre for Evidence-Based Medicine Nuffield Department of Primary Care Health Sciences Oxford UK MedicalResearch: What is the background for this study? What are the main findings? Dr. Onakpoya: Several orphan drugs have been approved for use in Europe. However, the drugs are costly, and evidence for their clinical effectiveness are often sparse at the time of their approval. We found inconsistencies in the quality of the evidence for approved orphan drugs. We could not identify a clear mechanism through which their prices drugs are determined. In addition, the costs of the branded drugs are much higher than their generic or unlicensed versions. MedicalResearch: What should clinicians and patients take away from your report? Dr. Onakpoya: Because of inconsistencies in the evidence regarding the benefit-to-harm balance of orphan medicines, coupled with their high prices, clinicians and patients should assess whether the orphan drugs provide real value for money before making a decision about their use for a medical condition.