1. Optogenetic gene therapy partially restores light sensitivity in advanced retinitis pigmentosa
A phase 1/2 trial published in NEJM on October 7 treated 10 people with late-stage retinitis pigmentosa. Each received one eye injection of a light-sensitive protein gene, then wore light-projecting goggles [1][2][3]. The treatment was judged safe. Six participants had clinically meaningful gains in light sensitivity, and 4 of 8 tested improved on object tasks, though none regained normal sight [2][3]. The study was small and uncontrolled, and the visual findings are exploratory [3].
2. FDA says its mifepristone safety review will run into 2027
In a court-ordered status report filed late on October 7 in Louisiana's lawsuit, the FDA said it expects to finish its safety study by mid-December 2026. A report would follow no later than March 2027, after which the agency will decide on any new restrictions [4][5]. The review uses data-mining of an electronic database of medical-injury and side-effect reports from health networks and insurers [4]. Anti-abortion groups criticised the timeline [4]. Access to the drug, including by mail, is unchanged while the Supreme Court's order stands [4][5].
3. WHO issues its first global guideline on child and adolescent obesity
On October 7, WHO advised against obesity medicines, surgery or devices for children under 10. It recommended diet, physical activity and behavioral support instead [6][7]. For ages 10–19, medicines such as GLP-1 drugs may be considered only after supervised programmes fail. WHO made this a conditional recommendation and cited limited evidence [6][7]. About 170 million 5- to 19-year-olds were living with obesity in 2024, and prevalence in this age group has quadrupled since 1990 [6].
4. PhRMA sues to block Medicare's "most-favored-nation" GLOBE drug-pricing model
The brand-name drug industry's trade group filed suit on October 7 in federal court in Washington, D.C. It is challenging the GLOBE model, which CMS finalised on September 30 [8]. GLOBE uses international prices as benchmarks for rebates on certain Medicare Part B drugs. It is set to run from January 2027 to March 2032 in areas covering about 25% of Original Medicare enrollees [8]. PhRMA argues the model exceeds Medicare's statutory authority [8][9]. No government response to the suit was reported in the sources reviewed.
Digest references
Sahel J-A, et al. "Optogenetic Therapy for Restoring Aspects of Visual Function." New England Journal of Medicine, October 7, 2026. DOI: 10.1056/NEJMoa2602215. Link
University of Pittsburgh. "Optogenetic therapy safe, shows signs of vision restoration…" EurekAlert!, October 7, 2026. Link
GenSight Biologics. "NEJM Publication on GS030 Phase 1/2 Trial Results in Late-Stage Retinitis Pigmentosa." BioSpace, October 8, 2026. Link
Perrone M (Associated Press). "FDA says its safety review of abortion pill will continue into next year." STAT, October 8, 2026. Link
Medical Daily. "Mifepristone Access by Mail Continues as FDA Tells Louisiana Court Its Safety Review Should Finish by March 2027." October 2026. Link
Reuters. "WHO endorses lifestyle changes over GLP-1 drugs in child obesity fight." The Star, October 7, 2026. Link
Health Policy Watch. "GLP-1 drugs, surgery: WHO issues guidelines for obesity in children." October 7, 2026. Link
Pharmaceutical Commerce. "Why PhRMA sued to block Medicare's GLOBE pricing model." October 7, 2026. Link
Silverman E. "Pharmalittle: PhRMA suing Trump, biotech frustration over tariffs, and more." STAT, October 8, 2026. Link
