Hello, everyone, and how are you this morning? We are doing just fine, thank you, courtesy of a warm and shiny sun enveloping the leafy Pharmalot campus. Our short people have left for their destinations, our official mascot has tired himself out and is now snoozing in a corner, and we are left with a beckoning coffee pot that is waiting to be fired up. And so, on with the show. Here are a few tidbits to help on your journey. We hope your day is productive and also meaningful. Why not add a little zen now and then? Good luck and keep in touch. …

The Food and Drug Administration approved a medicine from Jacobus Pharmaceuticals, a small, family-run company, for treating a neuromuscular disorder called Lambert-Eaton myasthenic syndrome, or LEMS, for children ages 6 to 17, STAT reports. The move potentially adds unforeseen competition for Catalyst Pharmaceuticals (CPRX), which last December won an FDA endorsement to market a treatment for adults. The approval adds an unexpected twist to a simmering controversy over a rare disease drug that briefly became a poster child for high-priced medicines.

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!


What is it?

STAT Plus is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

What's included?

  • Daily reporting and analysis
  • The most comprehensive industry coverage from a powerhouse team of reporters
  • Subscriber-only newsletters
  • Daily newsletters to brief you on the most important industry news of the day
  • Online intelligence briefings
  • Frequent opportunities to engage with veteran beat reporters and industry experts
  • Exclusive industry events
  • Premium access to subscriber-only networking events around the country
  • The best reporters in the industry
  • The most trusted and well-connected newsroom in the health care industry
  • And much more
  • Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy