FDA Approves Hemophilia Treatment, But It’s One Of The Priciest Drugs Ever
The gene therapy, called Roctavian, is made by BioMarin Pharmaceutical. It costs $2.9 million and is infused just once. After the treatment, patients "should be free from having to really worry about this disease,” said Dr. Christopher Walsh, who directs Mount Sinai School of Medicine’s hemophilia program.
The Wall Street Journal:
New $2.9 Million Gene Therapy Promises To Remake Hemophilia Treatment
The FDA approved the new gene therapy, called Roctavian and made by BioMarin Pharmaceutical, for adults with a severe form of the disease. Roctavian is infused just once. Priced at $2.9 million, the drug now ranks among the most expensive in the world. But the price is in line with the cost of other new gene therapies, a groundbreaking type of treatment that replaces a missing or faulty gene. (Hopkins, 6/29)
In other pharmaceutical news —
AP:
Cheaper Competition For Humira Is Hitting The Market, But Savings Will Depend On Your Insurance
Patients who take the autoimmune disease treatment Humira may see some price relief when several lower-cost, biosimilar versions of the AbbVie drug reach the U.S. market in July. But lower pharmacy bills may not happen right away — or at all — for some patients. That’ll depend largely on your insurance coverage. Here’s a closer look. (Murphy, 6/29)
NPR:
Hepatitis C Treatment Underused Because Of High Cost And Insurance Restrictions
Ten years ago, safe and effective treatments for hepatitis C became available. These pills are easy-to-take oral antivirals with few side effects. They cure 95% of patients who take them. The treatments are also expensive, coming in at $20 to 25,000 dollars a course. (Huang, 6/29)
Axios:
Pharma Stares Down Another Term Without A Friend In The White House
Regardless of how the next year and a half shakes out, it seems increasingly likely that the pharmaceutical industry is going to have an adversary in the White House come 2025. Why it matters: Drugmakers are still reeling from Democrats' new law allowing Medicare to negotiate the price of drugs, but both President Biden and former President Trump are vowing even harsher crackdowns if either wins another term. (Owens and Goldman, 6/30)
Stat:
Duchenne Breakthrough Therapy Leaves Behind Pioneering Families
Pat Furlong was sitting in her home office in Middletown, Ohio, last Thursday, refreshing a Food and Drug Administration web page ad nauseam, when the phone rang. She answered and burst into tears. The FDA had just approved the first gene therapy for Duchenne muscular dystrophy, her friend and the therapy’s architect, Jerry Mendell, told her. It was a culmination of advocacy work Furlong began 39 years prior, after her own sons were diagnosed with the fatal muscle-wasting disease. (Mast, 6/30)
Also —
Stat:
Anesthesiologist Group: Stop Taking Ozempic Before Surgery
Patients fast before surgery to prevent food from getting into their lungs while they’re under — a serious concern that can lead to lung infection. But for those taking a class of treatments that include the widely popular Ozempic and Wegovy, fasting may not be enough to ensure an empty stomach. The American Society of Anesthesiologists issued guidance Thursday recommending that patients stop taking this class of treatments, called GLP-1 drugs, before undergoing surgery. (Chen, 6/29)
Las Vegas Review-Journal:
Man Sues CSL Plasma After Falsely Testing Positive For HIV
Jose Rivera is banned from donating plasma in the U.S. and Canada because of his HIV-positive status on the National Donor Deferral Registry. If he had a medical emergency, it would be difficult to receive a blood transfusion or organ transplant; he in turn cannot donate to a family member in need. The only problem: Jose Rivera does not have HIV. (Casale, 6/29)
KFF Health News and The Markup:
Need To Get Plan B Or An HIV Test Online? Facebook May Know About It
Looking for an at-home HIV test on CVS’ website is not as private an experience as one might think. An investigation by The Markup and KFF Health News found trackers on CVS.com telling some of the biggest social media and advertising platforms the products customers viewed. And CVS is not the only pharmacy sharing this kind of sensitive data. We found trackers collecting browsing- and purchase-related data on websites of 12 of the U.S.’ biggest drugstores, including grocery store chains with pharmacies, and sharing the sensitive information with companies like Meta (formerly Facebook); Google, through its advertising and analytics products; and Microsoft, through its search engine, Bing. (Tahir and Fondrie-Teitler, 6/30)
