The FDA has approved Madrigal Pharmaceuticals’ Rezdiffra™ (resmetirom) for nonalcoholic steatohepatitis (NASH). The approval is a watershed event, marking a historic turning point in the long-standing quest to find an effective treatment for the millions of Americans afflicted by this devastating condition. A targeted treatment for NASH has eluded pharmaceutical scientists for nearly a half century……
This year, Rare Disease Day is on February 29, 2024. Rare Disease Day is observed on the last day of February, which is the “rarest of calendar dates1 to underscore the nature of these diseases” and serves to raise awareness of rare diseases and those individuals affected by them. FDA is holding a virtual meeting2…
Changes Are Coming to Advisory Committee Meetings Advisory Committee meetings (AdComs) are a long-standing feature of the FDA drug approval process. How and where those meetings are conducted has been evolving over the past few years, since the introduction of virtual meetings, and even more considerable reforms may be coming sooner rather than later. According…
“Silent killer” is an apt description for a potentially fatal disease that progresses slowly, without any obvious symptoms in the early stages. When you couple this insidious type of pathology with a rapidly increasing prevalence, the disease is often described in terms akin to an epidemic. Nonalcoholic steatohepatitis (NASH)—a disease characterized by fat accumulation in the…
Today, the US Food and Drug Administration (FDA) approved exagamglogene autotemcel (exa‑cel), the first CRISPR-based gene therapy for the treatment of sickle cell disease (SCD). Exa‑cel was co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics. This is a landmark approval that paves the way not only for better outcomes for patients with SCD, but for many…
Preparing for an FDA advisory committee meeting (AdCom) is a daunting task that can take anywhere from 3 to 9 months of intense preparation. At ProEd Regulatory, we partner with our clients to ensure that they’re well prepared to present their data and answer questions from the committee with confidence and poise. Our clients have…
On Thursday, September 21, 2023, the FDA held a meeting of the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) to discuss Intarcia’s new drug application (NDA) for ITCA-650, an implantable device intended to provide continuous dosing of a GLP-1 agonist (exenatide) to treat type 2 diabetes. In a highly unusual move, Intarcia opted to request…
Introduction Throughout history, humans have had a complex relationship with psychedelics. For millennia, ancient indigenous cultures used them for spiritual and healing purposes. For example, in 2007, archaeologists in Spain discovered mushrooms found at a burial site dated more than 7,000 years old. The mushrooms found at the site were identified as psilocybin, a type…
Historic Regulatory Decision for First FDA-Approved Gene Therapy for DMD On June 22, 2023, in a-much anticipated decision, the United States Food and Drug Administration (FDA) granted accelerated approval for Elevidys (also known as SRP-9001), Sarepta’s one-time gene therapy for ambulatory children with Duchenne’s muscular dystrophy (DMD). However, the accelerated approval was limited to the…
Imagine your newborn baby has been hospitalized with a serious respiratory infection and is struggling to breathe. That’s a reality for thousands of US newborns and their parents every year during the respiratory syncytial virus (RSV) season. This common endemic virus doesn’t cause clinically significant disease in most children and adults, but it can cause…
