September 15, 2023

The need-to-know this morning

The Food and Drug Administration extended by three months its review of a cell therapy for advanced melanoma from Iovance Biotherapeutics. The agency blamed "resource constraints" for the additional time needed to review the application, Iovance said. The new decision deadline is Feb. 24.
Two biotechs — Neumora Therapeutics and RayzeBio— begin trading today after pricing their respective initial public offerings on Thursday evening. Neumora, which raised $250 million, is developing treatments for neuropsychiatric and neurodegenerative diseases. RayzeBio, developing radiation-emitting medicines for cancer, raised $311 million.
Novartis shareholders approved the spin-off of Sandoz, its generics and biosimilars business.

drug pricing

The FTC will sanction drugmakers who don't play by 'Orange Book' rules

The patents that underpin branded drugs are all collated in an FDA registry called the "Orange Book." One way some drugmakers try to circumvent generic competition, however, is to not list their patents correctly. The FTC is now cracking down: In a new policy statement, it tells biopharma companies that it will "scrutinize improper Orange Book listings" and those that fail to pass muster could face legal action.

"Improper patent listings in the Orange Book illegitimately delay or lock out generic manufacturers from entering the market," FTC Chair Lina Khan said in a statement. "We won't hesitate to use all our tools to combat illegal practices that are inflating the price of health care, including medicines."

gene therapy

Insurer reverses course, covers Duchenne therapy

Cash Huber, a 6-year-old Duchenne muscular dystrophy patient, received a gene therapy from Sarepta Therapeutics this week. His treatment is notable because, as STAT wrote last month, insurance group Highmark Delaware initially denied him access to the $3.2 million drug, deeming it "experimental." It was an early test case in how insurers would approach a medicine that might alter the lives of patients with the fatal muscle-wasting disease but has only limited data and a sky-high price tag.

But after a public campaign from his family and advocates, Highmark reversed its decision, putting it in line with other payers, which have largely chosen to cover the treatment. By that point, Cash had technically aged out — it's approved only for 4- and 5- year olds — but doctors were still willing to administer it. The effects shouldn't kick in for a few weeks, but his dad, Phil Huber, reports Cash is doing well so far. Highmark said it could not comment, citing patient privacy.

podcast

What does Covid-19 have in store for winter?

When are good data not good enough? And how many would-be Wegovys are there? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast.

Our colleague Helen Branswell joins us to discuss the state of the Covid-19 pandemic heading into the fall and the rollout of booster shots aimed at the latest viral variants. We also discuss the latest news in the life sciences, including an IPO implosion, a debate at the FDA, and the ups and downs of a career in biotech.

Listen here.

organ donation

Engineered pig kidney works for two months

For two months, a genetically modified pig's kidney functioned properly in brain-dead man. This is the longest time a non-human kidney has ever worked in a human body. The experiment was done at NYU Langone Health, in the body of Maurice "Mo" Miller — a man whose body remained alive even after an aggressive tumor in his brain rendered him otherwise deceased.

Efforts at xenotransplantation have been ongoing for decades, but this is one of the most promising efforts yet to bring it to fruition. There's a pressing global need for donated kidneys, and thousands of people die before they are ever able to receive one.

"It's a combination of excitement and relief," the transplant surgeon who led the experiment said. "Two months is a lot to have a pig kidney in this good a condition. That gives you a lot of confidence."