Upcoming FDA Decisions to Watch
Summary:
This month, the FDA is expected to announce key rulings, including the August 31 decision on Biogen and Eisai’s subcutaneous version of Leqembi for Alzheimer’s, designed to ease treatment burden. Other notable reviews include Insmed’s brensocatib for bronchiectasis, Tonix Pharmaceuticals’ TNX-102 SL for fibromyalgia, and PTC Therapeutics’ vatiquinone for Friedreich’s ataxia.
FDA Adds Cancer Risk Warning to Skysona
Summary:
The FDA has updated Skysona’s label with a Boxed Warning for increased risk of blood cancers, including MDS and AML, after treatment for early cerebral adrenoleukodystrophy. As of July 2025, 10 of 67 patients developed these conditions, prompting restrictions to those without a suitable stem cell donor and requiring lifelong monitoring.
Perioperative Padcev & Keytruda Yield Breakthrough Results in Bladder Cancer
Summary:
A recent Phase 3 trial has shown that combining Padcev with Keytruda before and after surgery significantly improves survival rates in patients with muscle-invasive bladder cancer who are ineligible for cisplatin-based chemotherapy. This marks the first time a systemic treatment has demonstrated such benefits in this patient group. Pfizer and Merck plan to discuss these findings with global health authorities for potential regulatory approval.
PureTech Spins Out Celea to Advance IPF Therapy
Summary:
PureTech Health has launched Celea Therapeutics, led by former Teva North America CEO Sven Dethlefs, to focus on deupirfenidone (LYT-100) – a chemically optimized version of pirfenidone – for the treatment of idiopathic pulmonary fibrosis (IPF). The Phase 2b trial showed slowed lung-function decline and “improved efficacy and similar to slightly better tolerability” versus pirfenidone, and Celea plans to meet with the FDA by the end of Q3 to discuss its Phase 3 trial design.
Bayer Taps KRAS G12D Inhibitor in Major Oncology Move
Summary:
Bayer has signed a licensing deal worth up to $1.3 billion with Kumquat Biosciences to develop a KRAS G12D inhibitor for hard-to-treat cancers such as pancreatic, colorectal, and lung tumors; Kumquat will complete the Phase 1a study, now cleared by the FDA, while Bayer handles later-stage development and commercialization. Meanwhile, Novartis’ experimental ianalumab achieved its third successful Phase 3 outcome this week.
