Pharmalittle: Novo boosts consumer focus, FDA okays gene therapy

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, although we do plan to attack our to-do list of chores and errands, promenade with the official mascots, and escort Mrs. Pharmalot to a soiree. We also hope to hold yet another listening party, where the rotation will likely include this, this, this, this and this. And what about you? Now that spring has sprung, there is opportunity to explore the great outdoors. Depending on your mood, you could hike a trail, stroll a city street, or, our favorite activity, take a long drive to nowhere. For those of you who prefer to stay home, there is always a good book or a flick on the telly. The possibilities are endless. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon. …

Novo Nordisk appointed Poul Weihrauch, who is chief executive officer of the Mars candy company, as board observer amid a move by the drug maker to strengthen its position in the highly competitive U.S. obesity market, Reuters points out. Novo Nordisk and its majority shareholder, the Novo Nordisk Foundation, carried out a leadership shake-up last year, replacing the chief executive officer ​and restructuring the board. This included the consolidation of Lars Rebien Sorensen’s leadership role through his ⁠appointment as board chair in addition to his role as chair of the foundation. Sorensen has promised ​to strengthen the board’s pharmaceutical and commercial expertise after criticizing the previous board for being slow to address U.S. market challenges. Novo is ​trying to boost consumer credentials in the U.S. market in a number of ways under its new management. In January, it launched its Wegovy pill across multiple cash-pay channels, rather than solely through traditional insurance routes.

The U.S. Food and Drug Administration approved a gene therapy for severe leukocyte adhesion deficiency type 1 (LAD-1), an ultra-rare disease that leaves children vulnerable to life-threatening infections, STAT notes. The therapy, marketed as Kresladi, was developed by Rocket Pharma and was initially rejected by the agency in 2024 over manufacturing concerns. Recently, though, the FDA has indicated its willingness to relax some of its manufacturing requirements to ease such therapies along. Rocket is likely to charge millions of dollars for the one-time treatment, but Kresladi is not expected to be a major money maker. LAD-1 is thought to affect only around 1 in a million people, with Rocket estimating around 25 new cases per year. The approval, however, will earn the company an FDA priority review voucher that can be used to have another drug approved in just six months, rather than the typical 10.