A recent clinical trial showed that a drug used for a small coterie of patients suffering from rare inflammatory disorders can reduce the risks of serious complications in people who have suffered a heart attack.
The FDA has approved the first new treatment for patients with sickle cell disease in almost 20 years.
Acting on a request from three influential senators, the Government Accountability Office (GAO) has confirmed that it will investigate potential abuses of the Orphan Drug Act (ODA), according to a report from Kaiser Health News (KHN).
Building on weeks of mounting pressure to address high prescription drug prices, three influential senators have asked the government’s accountability arm to investigate potential abuses of the Orphan Drug Act, according to a report from Kaiser Health News (KHN).
A few weeks ago, Jeffrey Aronin, CEO of Marathon Pharmaceuticals, announced his decision to charge $89,000 per year for deflazacort––a cheap, generic steroid––for patients with Duchenne muscular dystrophy. Now a group of irate senators wants some answers.
Orphan drugs have been in the spotlight recently for reaping hefty profits from tiny patient populations. Some major new prospects are coming down the late-stage pipeline. An article in Endpoint News highlights the top 10 orphan drugs headed for potential FDA approval.