Volume 9 Issue 12, December 2010

For more than 50 years, warfarin has been the standard of care for long-term anticoagulation therapy in the United States, but now the FDA has approved the first oral anticoagulant that could replace it.

As the US FDA prepares to implement an abbreviated pathway for follow-on biologics, it gathered stakeholders to discuss key concerns. Analytical assessment and clinical development programmes were high on the agenda.

The European Medicine Agency's outgoing Executive Director Thomas Lönngren discusses his 10 years on the job, the importance of regulatory science and the challenges that lie ahead for his successor.

Regulatory and economic incentives to develop drugs for rare diseases, known as orphan drugs, have resulted in substantial improvements in the treatment for patients with some such diseases. However, the advent of orphan drug development has also raised several questions, from the definition of rarity, to the pricing of orphan drugs and their impact on health-care systems. Tambuyzer considers such questions and related misconceptions with the aim of aiding future progress in the field.

Data on the fraction of protein-bound drug are frequently used to guide chemical structure design and to prioritize compounds forin vivostudies. Here, the authors highlight how these practices are misleading and could result in the wrong compounds being progressed through discovery programmes.

The vitamin D endocrine system is a well-established target for metabolic bone disorders. Furthermore, the identification of the vitamin D receptor in tissues not related to calcium and bone has stimulated interest in its therapeutic significance in autoimmune diseases, cardiovascular disorders, infectious diseases and cancer. Plum and DeLuca highlight the emerging opportunities for vitamin D-based therapies in those diseases for which the evidence of their potential is most compelling.

Here, the authors discuss the potential of tyrosine kinase inhibitors — currently used for the treatment of cancer — as anti-proliferative agents in non-malignant disorders such as cardiac hypertrophy, pulmonary hypertension, lung fibrosis and rheumatoid disorders.

The expression of TSPO, a mitochondrial translocator protein involved in steroid synthesis, in the nervous system is altered in numerous neurological and psychiatric conditions. Rupprecht and colleagues review the potential of TSPO ligands for the treatment and diagnosis of disorders including anxiety, neuropathic pain and amyotrophic lateral sclerosis.