Rare diseases currently afflict 300 million people worldwide and 30 million people in the U.S. alone. However, 95% of these diseases lacked an FDA-approved treatment as of January 2023. One reason, industry leaders say, is cost. A 2019 study estimated that orphan drug...
Rocket Pharma is getting ready to light up national landmarks on Thursday as part of its annual rare disease awareness effort, but this year the event is even more meaningful as Rocket awaits an FDA decision on its first drug candidate. Rocket’s gene therapy...
With another quarter exceeding analysts’ expectations, Blueprint Medicines has raised its peak sales estimate for rare disease drug Ayvakit to $2 billion. The Massachusetts biotech unveiled the new target Thursday along with its latest earnings results, which showed...
In a recent announcement, Rocket Pharmaceuticals, Inc. has encountered a three-month postponement by the U.S. Food and Drug Administration (FDA) for its groundbreaking autologous gene therapy aimed at treating a rare white blood cell disorder. This decision shifts the...
Rare diseases, by their very nature, don’t fit the mold—so neither should the trials for therapies designed to treat them nor the regulatory process to approve them. This was the tone set during a recent panel discussion where Peter Marks, director of the FDA’s Center...
