Biopharmconsortium Blog

Expert commentary from Haberman Associates biotechnology and pharmaceutical consulting.

Monthly Archives: February 2013

FDA publishes Draft Guidance on developing drugs for early stages of Alzheimer’s disease

Tweet  Once again, approaches to improving clinical trials for candidate disease-modifying drugs for Alzheimer’s disease (AD) are in the news. On February 7, 2013, the FDA issued a Draft Guidance for Industry entitled “Alzheimer’s Disease: Developing Drugs for the Treatment of Early Stage Disease”. This document has been distributed for comment purposes only, and the…

A new wave of small-molecule disease-modifying drugs for cystic fibrosis

Tweet  In our January 24, 2013 article on this blog, we discussed the cases of two genetic diseases, sickle cell disease (SCD) and cystic fibrosis (CF). In both cases, the genetic cause of the disease was identified decades ago. However, no disease-modifying drugs for SCD have yet been developed. In the case of CF, small-molecule…

FDA approves mipomersen (Isis/Genzyme’s Kynamro)–the first systemically-delivered oligonucleotide drug to reach the market

Tweet  In our November 20, 2012 Biopharmconsortium Blog article, entitled “Novel hypercholesterolemia drugs move toward FDA decisions”, we discussed two drugs–Aegerion Pharmaceuticals’ lomitapide, and Isis/Sanofi/Genzyme’s mipomersen. These drugs were nearing approval decisions by the FDA, following the recommendations of the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee that both drugs be approved for treatment of…