STAT+: Pharmalittle: FDA approves BioMarin gene therapy for hemophilia A; former Pfizer worker charged with insider trading
The FDA approved a BioMarin Pharmaceutical gene therapy for people with hemophilia A, an inherited and rare bleeding disorder.
And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is still shaping up, but we expect to mingle with familiar faces, promenade with the official mascots, catch up on our reading, and hold a listening party — the rotation will likely include this, this, this, this and this. And what about you? This is a fine time to enjoy the great outdoors — beaches, lakes, and woods are beckoning. You could take a drive in the country or stroll city streets. Perhaps this is an opportunity to connect with someone special. Or simply plan the rest of your life. Well, whatever you do, have a grand time. But be safe. And by the way, a holiday is coming up on this side of the pond, so we will take an extended break. Enjoy, and see you on Wednesday. …
The U.S. Food and Drug Administration approved a gene therapy from BioMarin Pharmaceutical to treat people with hemophilia A, an inherited and rare bleeding disorder, STAT writes. The treatment, called Roctavian, is a one-time therapy that, in clinical trials, dramatically reduced bleeding episodes and helped patients live without the blood transfusions used to treat the disease. The U.S. list price is $2.9 million, or $1.9 million after customary insurer discounts. In Europe, Roctavian is priced at roughly $1.6 million. There are approximately 6,500 adults living with severe hemophilia A in the U.S., of which approximately 2,500 will be eligible to receive the drug.
One of the pharmaceutical industry’s dealmakers is striking again to revive Bausch + Lomb, the eye-care company, The Wall Street Journal reports. The company will pay $1.75 billion to acquire a dry-eye drug from Novartis, marking the first big move by chief executive officer Brent Saunders since he returned to Bausch + Lomb in March. But Saunders must find new growth at a company whose $3.8 billion in revenue last year was relatively flat from the prior year. The acquisition expands Bausch’s presence in the lucrative and growing market for dry eye, which affects some 16 million people in the U.S. Diagnoses have been increasing partly because the population is aging.
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