‘Cause For Major Celebration’: Impressive Results From Study Spark Optimism Over New Cystic Fibrosis Therapy
Two new studies find that, among other things, patients who received the therapy showed "striking" improvement in a key measure of lung capacity. The drug will cost about $311,000 per year, the same as Vertex’s previous cystic fibrosis drug.
The New York Times:
Studies Yield ‘Impressive’ Results In Fight Against Cystic Fibrosis
A pair of new studies report “impressive” benefits from a drug therapy for cystic fibrosis, a deadly and devastating disease that affects tens of thousands of people worldwide, the director of the National Institutes of Health wrote in an editorial published in The New England Journal of Medicine on Thursday. “These findings indicate that it may soon be possible to offer safe and effective molecularly targeted therapies to 90 percent of persons with cystic fibrosis,” wrote the director, Dr. Francis S. Collins, who led the team that in 1989 identified the gene that causes the genetic disease affecting the lungs and digestive system. (Chokshi, 10/31)
The Washington Post:
New Cystic Fibrosis Drug Trikafta Could Make Deadly Disease Manageable
A new cystic fibrosis therapy dramatically improved patients’ lung function and showed clear signs of targeting the genetic root of the disease, instead of just alleviating symptoms — a breakthrough so long-sought that many doctors and patients are moved to tears when talking about it. The data, being unveiled Thursday at a national conference in Tennessee and simultaneously published in two leading medical journals, was so persuasive that the Food and Drug Administration last week approved the three-drug combination, called Trikafta — five months ahead of the agency’s deadline. (Johnson, 10/31)
In other pharmaceutical news —
Stat:
Giving Vouchers To Pharma To Develop Antibiotics Could Be Costly
One approach federal lawmakers have used to jumpstart certain types of drug development has been to offer vouchers to companies that can later be redeemed when seeking approval for yet another medicine. But a new analysis suggests the notion may not be worth the cost if it were used to entice drug makers to develop much-needed antibiotics. Citing a bill proposed last year to offer vouchers for new antibiotics, the researchers calculated the idea would have cost an extra $4.5 billion in spending on medicines over a 10-year period, had the legislation taken effect in 2007. (Silverman, 10/31)
Stat:
Amgen Acquires Stake In BeiGene To Grow Cancer Drug Business To China
Amgen is acquiring a premium stake in BeiGene (BGNE), one of the largest cancer-focused drug companies in China, for $2.7 billion as part of a broad commercial and drug development partnership, both companies announced Thursday. The California-based biotech joins a long list of American firms seeking to tap the pharmaceutical market in China, which is said to have as many cancer patients as the U.S. and Europe combined. (Feuerstein, 10/31)
