Ionis wins first approval for Alexander disease

The FDA has approved Zanastro, an antisense drug from Ionis Pharmaceuticals for Alexander disease, an ultra-rare and often fatal neurological disorder.

In a pivotal study of roughly 50 patients, walking speed remained stable among those receiving the antisense drug; it declined 33% in controls. The data in children ages 2 to 4 also suggested that early treatment could actually improve motor function. When the trial’s results were first unveiled to the companies, STAT’s Jonathan Wosen writes, Ionis employees were bowled over:

“There was people crying. There was people gasping. There was people clapping,” the company's chief development officer, Holly Kordasiewicz, told Jonathan. “Everybody had to take a minute.”

The approval is also a commercial milestone for Ionis: Zanvastro will be the biotech’s first neurology drug launched on its own, as it continues its shift away from relying on pharma partners to sell its medicines.

xenotransplantation

Pig kidneys could bridge the wait for transplant

Two of the first patients to receive genetically engineered pig kidneys from eGenesis have now successfully received human kidney transplants, STAT’s Eric Boodman writes. The cases offer strong evidence that such xenotransplants could help patients survive the long wait for a donor organ without jeopardizing their chances of getting one later.

The pig kidneys ultimately had to be removed, and both patients returned to dialysis for months, but they provided months of dialysis-free time — potentially leaving patients healthier and better able to survive until a human kidney becomes available.

The results also ease concern that exposure to a pig organ might generate antibodies that complicate a later human transplant. eGenesis ultimately hopes its pig kidneys can become permanent replacements, but the cases suggest that even if they don’t, xenografts could still become a valuable bridge amid the chronic shortage of human organs.

regulation

FDA takes another look at botanical drugs

From STAT's Margaret Manto: The FDA has issued a request for information on botanical drug products, which the agency defines as “products that include or may be derived from plant materials, algae, macroscopic fungi, or combinations of these materials and are intended to diagnose, cure, mitigate, treat or prevent disease.” Acting Commissioner Kyle Diamantas said in a statement that the agency is hoping to evaluate “natural health options” to offer new therapies “while maintaining the highest safety standards.”

While many modern drugs include plant-derived compounds, botanical drugs are generally complex mixtures. To date, the FDA has only approved four botanical products as prescription drugs, including a plant bark extract for HIV-associated diarrhea and proteolytic enzymes from pineapple for thermal burns.

Relatedly, Rep. Lauren Boebert (R-Colo.) introduced a bill last week, the Advancing Botanical Drug Development Act of 2026, that would give companies a 12-year exclusivity period on new botanical drug products approved by the FDA.

mental health

The biggest barrier for clozapine, a top childhood schizophrenia drug

The FDA has finally scrapped the burdensome monitoring program that governed the psychiatric drug clozapine for decades. But psychiatrist Nitin Gogtay opines for STAT that bureaucracy was never the main reason one of psychiatry’s most effective drugs remains woefully underused.

Clozapine can be transformative for people with treatment-resistant schizophrenia — including children with the particularly severe childhood-onset form. But clinicians often cycle patients through less-effective drugs because they fear clozapine’s risks, which Gogtay says are serious but manageable.

Gogtay, who spent nearly two decades studying the drug at the NIMH, argues that removing mandatory monitoring should be an opportunity not merely to make clozapine easier to prescribe, but to reconsider why doctors wait so long to use it.

“To my colleagues who care for the youngest and sickest patients, I would put it as plainly as I can: Do not let fear make this decision for you,” he writes.

Thanks for reading! Until next week,

rare disease