apitegromab(Scholar Rock Holding Corp) · Sep 30, 2026 BLA
Spinal muscular atrophy — a genetic motor-neuron disease where many patients still plateau with residual muscle weakness even on today's SMN-targeted therapies.
Apitegromab is an anti-myostatin antibody that works on muscle rather than the SMN gene defect that causes spinal muscular atrophy. Existing therapies — nusinersen, risdiplam and onasemnogene — restore SMN protein but do not directly rebuild muscle; apitegromab is designed to be added on top to improve motor function. Scholar Rock resubmitted the BLA on March 31, 2026 after a September 2025 Complete Response Letter, and the FDA granted Priority Review with a target action date at the end of September. The filing rests on the Phase 3 SAPPHIRE trial and also carries Fast Track, Orphan Drug and Rare Pediatric Disease designations. Watch whether the second-round review clears the issues that drove the CRL.
zidesamtinib(Nuvalent, Inc.) · Sep 18, 2026 NDA
Advanced ROS1-positive non-small cell lung cancer in patients whose tumors have already progressed on one or more ROS1-targeted therapies.
Zidesamtinib is Nuvalent's selective ROS1 inhibitor, engineered to stay active against the resistance mutations that emerge on earlier TKIs and to penetrate the brain, where ROS1-positive lung cancer frequently spreads. The NDA covers TKI-pretreated patients — a setting with few durable options once first-line ROS1 drugs fail. The application is based on the ARROS-1 trial and is under standard review. The FDA granted Breakthrough Therapy designation specifically for patients previously treated with two or more ROS1 TKIs, plus Orphan Drug status. With a September 18 target date, watch the depth and durability of responses in the pretreated and CNS-metastatic subgroups that define the unmet need.
Brepocitinib(Roivant Sciences Ltd.) · Sep 30, 2026 NDAPriority ReviewBreakthrough
Dermatomyositis — a rare, often debilitating autoimmune disease that inflames muscle and skin and is still largely managed with steroids and broad immunosuppression.
Brepocitinib is an oral dual TYK2/JAK1 inhibitor from Priovant, a Roivant company, aimed at the interferon-driven inflammation central to dermatomyositis. Most patients today rely on corticosteroids, conventional immunosuppressants and IVIG; a targeted oral agent that dials down the specific signaling pathways behind the disease would be a meaningful addition. The NDA is built on the Phase 3 VALOR study and carries an unusually strong regulatory package — Breakthrough Therapy, Orphan Drug and Priority Review. Priovant has guided a commercial launch by the end of September 2026. Watch how broad the eventual label is and how strongly the functional and skin endpoints read out.
