rare disease
Sarepta gene therapy approved for most Duchenne patients
The FDA yesterday dramatically expanded its approval for Elevidys, a gene therapy for Duchenne muscular dystrophy, authorizing access for nearly all patients with the fatal muscle-wasting disease.
It's clear that the treatment, made by Sarepta Therapeutics, won't be a panacea — "No one pretends this is a cure," one patient's mother told STAT's Jason Mast — but the gene therapy offers at least incremental benefit to patients with Duchenne. It is now approved for all patients over the age 4 who can walk on their own. For patients who rely on wheelchairs, the agency granted accelerated approval, a form of conditional authorization that will have to be confirmed in a larger, ongoing study.
It's worth noting the approval decision was made almost exclusively by Peter Marks, the FDA's director of the Center for Biologics Evaluation and Research, who overruled three review teams and two top lieutenants.
immunotherapy
Can JAK inhibitors bolster checkpoint inhibitors?
Checkpoint inhibitors like Keytruda and Opdivo are fickle: At times they work beautifully in patients, but in others, not so much. But two small clinical trials published in Science found that a JAK inhibitor made by Incyte, when combined with checkpoint inhibitors, led to most patients responding to immunotherapy. In one trial, patients were able to overcome resistance to checkpoint inhibitors, STAT's Angus Chen writes.
In that study, 10 out of 19 participants with Hodgkin lymphoma whose disease had already progressed or relapsed responded to the drug — and six achieved a complete response. In the other study, researchers combined Keytruda with the JAK inhibitor and saw an improved response.
"It could really change the landscape in terms of treatment for some patients," an immunologist not involved with the work told STAT. "It opens the door for a whole series of studies for cancers which are treated with immune checkpoint inhibitors which work already, but they're not perfect and not enough. It may give another arrow in the armamentarium of clinicians."
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infectious disease
Gilead's experimental PrEP for HIV impresses
Gilead's new antiviral drug lenacapavir completely protected cisgender women from contracting HIV in a large Phase 3 trial. None of the 2,134 women taking the twice-a-year injection contracted the virus. By comparison, 16 of 1,068 women taking Truvada and 39 of the 2,136 women taking Descovy contracted HIV.
"To have no infections in over 2,0000 women… I mean that's a remarkable result," the study's principal investigator told STAT's Jason Mast. The trial was conducted in Uganda and South Africa; if a follow-up study in other countries confirms the results, lenacapivir could be approved as a potent pre-exposure prophylaxis, or PrEP, for HIV.
"This offers a really different option, because it is just twice a year, it's discrete and private, because it's not in a bottle that you have to carry home, that someone could find," Gilead's HIV franchise head told STAT. "For so many people out there in so many different places, this could provide a PrEP option that's workable for them."
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