Orphan Drugs

News Orphan Drugs

• Hybrigenics starts phase II clinical trial of inecalcitol in chronic lymphocytic leukemia

  Hybrigenics announced the start of a phase II clinical trial of oral inecalcitol in patients with chronic lymphocytic leukemia (CLL). World-wide, about 130,000 new patients are diagnosed each year with CLL, for which there is currently no definitive cure. CLL is designated as an orphan disease in th more

• to-BBB awarded EUR 1.25 million European FP7 funding to develop treatments for inherited retinal diseases

  to-BBB has been awarded funding of EUR 1.25 million by the European Commission’s 7th Framework Programme as part of a EUR 5 million consortium project for the preclinical development of novel treatments for inherited retinal degenerative diseases. The consortium additionally consists of Coordinator more

• Big Pharma and Biotech Increasingly Tackling Rare and Niche Diseases

  Rare and niche diseases are being increasingly spotlighted by pharmaceutical and biotechnology corporations in the hope of developing initial treatments or improving upon existing therapies for these conditions, according to a new report released by GlobalData.The new report, "Rare and Niche Disease more

White papers Orphan Drugs

• Orphan Drugs – An Alternative to the declining Blockbuster Model?
  

  Up until the new millennium, the pharmaceutical industry grew stronger by 11%. However, the blockbuster model is increasingly at risk and the declining growth rates in the industry require a search for new strategies. In this context, orphan drugs are becoming increasingly important more

Publications Orphan Drugs

• Molecular characterization of carbamoyl‐phosphate synthetase (CPS1) deficiency using human recombinant CPS1 as a key tool

  ABSTRACT The urea cycle disease carbamoyl‐phosphate synthetase deficiency (CPS1D) has been associated with many mutations in the CPS1 gene [Häberle et al. Hum Mutat 2011; 32:579–589]. The disease‐causing potential of most of these mutations is unclear. To test the mutations effects, we have deve more

• Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU

  Purpose To assess the methodological quality of Orphan Medicinal Product (OMP) dossiers and discuss possible reasons for the small number of products licensed. Methods Information about orphan drug designation, approval, refusal or withdrawal was obtained from the website of the European Medicines A more

• Do we need authorized orphan drugs when compounded medications are available?

  Summary What is known and Objective:  Orphan drugs are used to diagnose, prevent or treat a rare disease. This Commentary aims to present a number of case studies questioning the need for designating compounded medications with a long history of effective use, which is well‐supported by publishe more

Market studies Orphan Drugs

• Rare and Niche Diseases Catalyst Monitor - Key Event Analysis H1 2012

  Introduction GlobalData’s new report, "Rare and Niche Diseases Catalyst Monitor - Key Event Analysis H1 2012" takes a look at rare and niche diseases, which are an area of intense interest in the global biopharmaceutical industry today. The passage of the Orphan Drug Act in the United States in 19 more

• Re-evaluating Pharmaceutical Pipelines - Emerging Markets, Biologics and Orphan Drugs to Shape Future Decision Making

  GBI Research, a leading business intelligence provider, has released its latest research, “Re-evaluating Pharmaceutical Pipelines - Emerging Markets, Biologics and Orphan Drugs to Shape Future Decision Making”, which examines the key issues influencing the re-evaluation of pharmaceutical pipelines d more

• Pharmaceutical Pricing and Reimbursement - Adoption of Health Technology Assessments (HTA) Optimizes Drug Pricing and Accelerates Reimbursement Decision-Making

  GBI Research, leading business intelligence provider has released its latest research report, entitled “Pharmaceutical Pricing and Reimbursement - Adoption of Health Technology Assessments (HTA) Optimizes Drug Pricing and Accelerates Reimbursement Decision-Making”. The report provides a comprehensiv more

Websites Orphan Drugs