Table 1.
Basic and preclinical animal research and first-in-class drugs approved by the United States Food and Drug Administration in 2016.
| Trade Name (Drug) | Disease | Animal Use |
|---|---|---|
| Defitelio (defibrotide sodium) | Hepatic veno-occlusive disease afterhaematopoietic stem cell transplantation * | Derived from the intestinal mucosa of pigs, defibrotide has been tested in several cell lines and in animals, such as mice [20,21,22]. |
| Exondys 51 (eteplirsen) | Duchenne muscular dystrophy * | Eteplirsen works by causing exon skipping to correct a genetic mutation. Animal studies on the approach used mice and dogs [23]. However, its approval using a surrogate marker of efficacy in humans is controversial [24]. |
| Ocaliva (obeticholic acid) | Primary biliary cirrhosiss * | Obeticholic acid is a farnesoid-X receptor agonist. The farnesoid-X receptor was recommended as a drug target based on studies in rats [25,26,27]. |
| Spinraza (nusinersen) | Spinal muscular atrophy * | Nusinersen (like eteplirsen) works by modulating gene splicing to increase levels of a protein affected by an inherited genetic mutation. It was advanced to clinical trials based on work in mice and non-human primates [28,29,30]. |
| Venclexta (venetoclax) | Chronic lymphocytic leukemia * | Targets the Bcl2 receptor, based on basic research into apoptosis and mouse cancer models [31,32,33]. |
| Xiidra (lifitegrast) | Dry eye disease | Dry eye disease can affect animals like dogs, cats, and horses and animal models have provided evidence of an inflammatory role. Lifitegrast reduces inflammation by preventing LFA-1/ICAM-1 interactions and has been tested indogs and mice [34,35,36]. |
| Zinbryta (daclizumab) | Multiple sclerosis | Daclizumab was originally developed as immune suppressant for transplant patients and based on studies in mice showing mechanisms to suppress autoimmune responses. Human clinical studies facilitated its translation for multiple sclerosis [37,38]. |
| Zinplava (bezlotoxumab) | Clostridium difficile infection | Bezlotoxumab neutralises C. difficile toxin B. Early work involved characterising the effect of toxin B in animals like hamsters and rabbits and using rabbits to generate antibodies against toxin B [39,40,41]. |
* “Orphan” or rare diseases affecting fewer than 200,000 Americans often have limited treatment options or no drug treatment available.