Drug Targeting Rare Form Of ALS Gets Some Support From FDA Panel
The drug, tofersen from maker Biogen, got "partial" support from a panel of Food and Drug Administration advisers Wednesday, which could lead to a possible accelerated approval. A hearing over future Medicare coverage of Alzheimer's drugs is also in the news.
Bloomberg:
Biogen’s ALS Drug Gets Partial Backing From FDA Panel
A Biogen drug that targets a rare form of ALS got partial support from a panel of US Food and Drug Administration advisers Wednesday, paving the way for a possible accelerated approval. If it’s approved, the medication, tofersen, would be the first drug targeting a specific genetic cause of the incurable paralysis disease to get the regulatory green light. (Langreth, 3/22)
Stat:
Senators Interrogate Health Secretary On Alzheimer's Drug Coverage
The future of Alzheimer’s treatments and coverage hung heavily over lawmakers’ Wednesday hearing with Health and Human Services Secretary Xavier Becerra. Dotted throughout the hearing room for Becerra’s testimony on the president’s proposed health care budget for 2024 were purple-clad advocates for Alzheimer’s disease treatments, who Democrats and Republicans alike acknowledged repeatedly throughout the hearing. But while senators from both parties pushed for speedy approvals and Medicare coverage of new drugs for the disease, they unsurprisingly diverged on how to manage the costs. (Owermohle, 3/22)
WMFE:
Alzheimer's Study Explores New Drug's Potential To Delay Or Prevent Symptoms
In the past two years, the FDA has approved the first two drugs that treat the actual disease of Alzheimer’s, the most common form of dementia affecting more than 6 million people in the U.S. An ongoing study -- with participants in Central Florida -- examines whether one of those drugs can prevent or at least delay the appearance of symptoms. (Byrnes, 3/22)
On drug shortages —
NPR:
Why Drugs Like Adderall, Antibiotics And Flu Medications Are In Short Supply
It's not just your imagination: Drugs such as children's flu medication, common antibiotics and ADHD treatments are getting harder to buy, according to a Senate report published Wednesday. Democrats on the Homeland Security and Governmental Affairs Committee say the number of new drug shortages rose by 30% between 2021 and 2022, an increase that has had "devastating consequences" for patients and doctors. (Olson, 3/23)
Roll Call:
Lawmakers Say Reliance On China Exacerbates Drug Shortages
Lawmakers say the worsening drug shortage in the United States is hurting the American health care system and called for shifting more manufacturing from China to the United States to help alleviate the problem during a Senate hearing Wednesday. (Cohen, 3/22)
In other pharmaceutical news —
Stat:
NIH Rejects Bid To Cut A Cancer Drug's Price By Sidestepping Patents
In a long-awaited decision, the National Institutes of Health rejected a petition urging the agency to use a controversial provision of federal law to widen access to a cancer drug by forcing the manufacturers to license their patents. (Silverman, 3/22)
Stat:
Drugmakers Push Back On A Clever Tactic Employers Use To Avoid Paying For Specialty Medicines
In the face of rising drug prices, health plan sponsors have quietly used a clever, but questionable tactic over the past few years to deflect costs. And now, some pharmaceutical companies are pushing back. (Silverman, 3/22)
Stat:
89bio Treatment Reduces Liver Scarring In Patients With NASH
Biotech company 89bio said Wednesday that its experimental treatment improved liver scarring at more than three times the rate of placebo without worsening other symptoms of patients with the fatty liver disease known as NASH. The results met the main goal of a mid-stage clinical trial, with the potential for less frequent injections than a similar experimental treatment being developed by a competing drugmaker. (Feuerstein, 3/22)
Stat:
Gene Researchers Try To Find Ways To Get Around Key CRISPR Flaw
It took just three years, after the tool’s invention, for researchers to devise ways of using CRISPR-Cas9 gene editing to treat mice with Duchenne muscular dystrophy. Since those first 2015 papers, dozens more have been published and multiple startups have launched, spurred by the hope that CRISPR could outperform the gene therapy approaches now nearing approval. One company was even bought out for over $200 million. Yet, with one exception, no gene-editing treatments for the rare muscle-wasting disease have entered the clinic and none appear particularly close. Why? (Mast, 3/22)
Quartz:
Is There A Bird Flu Vaccine Available For Humans?
The good news is, there are vaccines. More accurately: There can be, quickly, if we need them. The US has a stockpile of avian flu vaccines, and vaccine makers say they would be able to turn around hundreds of millions of doses of vaccines for the bird flu latest subtype in months. But there’s a downside to that: Most of the potential millions of doses of bird flu vaccines that would be needed in a potential (though, yes, unlikely) human epidemic are already reserved. For rich countries, of course. (Merelli, 3/22)
Fox News:
Is Parkinson’s Disease, 'World’s Fastest Growing Brain Disease,’ Mostly Preventable? Study Offers Clues
Researchers warn that a colorless chemical known as trichloroethylene (TCE) — which has been used to dry-clean clothes, degrease metals and decaffeinate coffee — may be linked to the dramatic increase in Parkinson’s disease (PD) cases. They recently published a series of seven cases in the Journal of Parkinson’s disease that illustrate TCE’s harmful health effects and the potential PD association. (Sudhakar, 3/22)
