Top of the morning to you. Soggy skies are hovering over the Pharmalot campus right now, but our spirits remain sunny, nonetheless. Why? We will trot out a bit of insight from the Morning Mayor, who would say “Every day should be unwrapped like a precious gift.” To celebrate the notion, we are brewing still more cups of stimulation and invite you to join us. Our choice today is strawberry creme. Meanwhile, here are a few items of interest. Hope you have a smashing day and, of course, do stay in touch. …
The U.S. Food and Drug Administration concluded that an experimental treatment for Huntington’s disease developed by UniQure was not providing benefit for patients based on existing clinical data, STAT reports. The agency blocked the company from submitting a marketing application and the dispute occurs as an increasing number of rare disease companies have come under heightened — and often contradictory — regulatory scrutiny. Last fall, UniQure’s gene therapy, called AMT-130, seemed to be on a path toward approval as the first genetic treatment for Huntington’s, a rare neurodegenerative condition. Under FDA Commissioner Marty Makary, meanwhile, the agency has touted efforts to accelerate rare disease drug approvals, while at the same time rejecting such treatments and insisting that they be tested in clinical trials that may not be feasible.
Major biotech and pharmaceutical companies are asking the Trump administration to rescind two proposals seeking to align U.S. drug prices with cheaper ones overseas, warning the sweeping policies harm innovation and exceed the government’s authority, Bloomberg Law explains. The plans would test whether alternative methods for calculating rebates under Medicare Part B and Part D, based on international pricing metrics, will reduce drug costs. The pricing demos, which are mandatory for manufacturers to participate in, would help implement the most-favored-nation plan that attempts to match U.S. prices with lower ones in other wealthy nations. But big drugmakers are rejecting the proposals, questioning the legal ground and methodologies. If finalized, the companies say the demonstrations could inject a new wave of instability into how they bring new therapies to the market and price them in the U.S.Continue to STAT+ to read the full story…