Around 800 Drugs in Development for Rare Diseases, Says PhRMA

There are almost 800 drugs for rare diseases either in clinical trials or awaiting review by the FDA, according to a new report by the Pharmaceutical Research and Manufacturers of America (PhRMA).

Of the 791 rare disease drug candidates in trials or awaiting approval, 35 percent are for rare cancers (168) and rare blood cancers (120), the report said.

There are 192 candidates for genetic disorders, including cystic fibrosis and spinal muscular atrophy, and 56 for neurological disorders, including amyotrophic lateral sclerosis and seizures, according to PhRMA.

And drug developers are working on 54 candidates for blood disorders, including sickle cell disease and hemophilia, 51 for autoimmune diseases, including systemic sclerosis and juvenile arthritis, and 36 for infectious diseases, including rare bacterial infections and hepatitis.