PhRMA: More than 400 rare disease treatments in development, pending approval

WASHINGTON — More than 400 drugs are in late-stage clinical development or awaiting Food and Drug Administration approval as treatments for rare diseases, according to a report by the drug industry lobby.

The Pharmaceutical Research and Manufacturers of America said a record 460 drugs were in late stages of the pipeline in a report timed to coincide with Rare Disease Day on Feb. 28. The rare or “orphan” diseases — defined by the government as those that affect fewer than 200,000 Americans — range from autoimmune disorders to cancer to blood disorders to genetic diseases. In total, there are 7,000 known rare diseases, about half of which affect children, and 80% of rare diseases affect fewer than 6,000 patients.

“Once you’ve talked to a desperate parent whose child is suffering or dying, you will be forever convinced of the need for medical innovation, including for terrible diseases that afflict even just a few,” PhRMA president and CEO John Castellani said. “Researchers at biopharmaceutical companies are working every day to bring hope to patients with limited options.”