FDA Approves Subcutaneous Keytruda as Pharmaceutical Giants Make Strides in Cancer and Rare Disease Treatments

The pharmaceutical industry has seen significant advancements in recent days, with major players securing regulatory approvals and reporting promising clinical trial results. From new drug delivery methods to potential breakthroughs in rare diseases, these developments underscore the sector's ongoing innovation and commitment to addressing unmet medical needs.

Merck's Keytruda QLEX Gains FDA Approval

Merck & Co. has achieved a notable milestone with the Food and Drug Administration's approval of Keytruda QLEX, a subcutaneous formulation of its blockbuster cancer drug. This new version, which can be administered in just one or two minutes, offers a more convenient alternative to the traditional intravenous infusion. The approval covers most solid tumor indications for which Keytruda is already approved in adults.

The introduction of Keytruda QLEX is part of Merck's strategy to maintain its market position as the drug approaches patent expiration later this decade. This approval aligns Merck with competitors Bristol Myers Squibb and Roche, who have already launched subcutaneous versions of their respective immunotherapies, Opdivo and Tecentriq, since late 2024.

Promising Results in Rare Diseases and Breast Cancer

MBX Biosciences has reported encouraging Phase 3 trial results for canvuparatide, its experimental treatment for chronic hypoparathyroidism. The study met its primary endpoint, with 63% of treated patients maintaining normal blood calcium levels over 12 weeks without requiring rescue therapy. This outcome positions canvuparatide as a potential first-in-class once-weekly treatment for this rare endocrine condition.

Meanwhile, Roche announced positive results from its Phase 3 trial of giredestrant in breast cancer. The hormone-degrading drug, when combined with everolimus, demonstrated improved progression-free survival in both a key patient subgroup and the overall trial population of pretreated patients with metastatic ER-positive, HER2-negative breast cancer. While survival data is still maturing, Roche reported a "clear positive trend."

In another development, Ionis Therapeutics revealed that its RNA-based therapy, zilganersen, met its objectives in a Phase 3 trial for Alexander disease, a rare and fatal neurological disorder. The treatment showed statistically significant improvement in a walking test and consistent benefits across multiple secondary measures. Ionis plans to file for regulatory approval next year, although the limited patient population may constrain the drug's commercial impact.

Regulatory Updates and Clinical Progress

Sanofi faced a setback as the FDA delayed its decision on tolebrutinib, a potential treatment for non-relapsing secondary progressive multiple sclerosis, by three months. The new decision date is set for December 28, following the submission of additional information deemed a "major amendment" to Sanofi's filing. If approved, tolebrutinib could become the first therapy specifically indicated for this less common form of multiple sclerosis.

These developments collectively highlight the pharmaceutical industry's continued focus on innovative drug delivery methods, targeted therapies for rare diseases, and advancements in treating complex conditions like cancer and neurological disorders. As companies navigate regulatory processes and clinical trials, the sector remains poised for further breakthroughs that could significantly impact patient care and treatment options.