Shots:
- The US FDA approved seven novel therapies in August 2026, spanning rare diseases, oncology, neurology, hematology, and autoimmune disorders, including Orzeyful (oveporexton), Tauklarify (florquintau F 18), Zenbexus (iberdomide), Pasatru (garetosmab-grts), Rasonque (daraxonrasib), LISRAYA (brepocitinib), and Mimrylo (rusfertide).
- Key approvals introduced new treatment options across multiple high-unmet-need indications, including narcolepsy type 1, Alzheimer’s disease imaging, relapsed/refractory multiple myeloma, fibrodysplasia ossificans progressiva, metastatic pancreatic ductal adenocarcinoma, dermatomyositis, and polycythemia vera.
- Several approvals were supported by pivotal Phase III data demonstrating clinically meaningful benefits, including Rasonque’s 60% reduction in risk of death in metastatic PDAC, Pasatru’s substantial reduction in new heterotopic ossification lesions, Zenbexus’ improvement in MRD-negative complete response, and LISRAYA’s steroid-sparing effects in dermatomyositis.
- Takeda Reports US FDA Approval of Orzeyful (Oveporexton) for Narcolepsy Type 1
Company: Takeda
Product: Orzeyful
Active Ingredient: Oveporexton
Disease: Narcolepsy Type 1
Date: Aug 05, 2026
Shots:
- The US FDA has approved Orzeyful (oveporexton) for the treatment of narcolepsy type 1 (NT1, narcolepsy with cataplexy) in adults. DEA scheduling decision is expected within 90 days, after which it will be available through a specialty pharmacy
- Approval was based on extensive data, incl. P-III (FirstLight & RadiantLight) trial assessing Orzeyful (BID) in NT1 pts over 12wks, where FirstLight randomized 168 pts to 3 arms (2mg, 1mg & PBO) & RadiantLight randomized 105 pts to 2 arms (2mg & PBO)
- Both trials demonstrated improvements across the full spectrum of disease symptoms, incl. excessive daytime sleepiness, cataplexy, and health-related quality of life
2. Lantheus Reports the US FDA Approval of Tauklarify as a PET Imaging Agent for Alzheimer’s Disease
Company: Lantheus
Product: Tauklarify
Active Ingredient: florquinitau F 18
Disease: Alzheimer’s Disease
Date: Aug 13, 2026
Shots:
- The US FDA has approved Tauklarify (florquinitau F 18 injection/ MK-6240) for PET of the brain in adults with cognitive impairment who are being evaluated for Alzheimer’s disease to identify pts with tau neurofibrillary tangle (NFT) pathology
- Approval was supported by Study 1 & 2 assessing Tauklarify (IV, 185 MBq) PET images from >500 pts who participated in 3 trials, where, independent readers classified Tauklarify scans as positive or negative for tau NFT pathology against a standard
- Study 1 incl. 279 pts, with PPA ranging from 80 to 88%, NPA from 98 to 99%, & high inter-reader agreement (generalized Fleiss’ kappa 0.92). Study 2 incl. 338 pts, with PPA of 68 to 82%, NPA of 93 to 99%, & high inter-reader agreement (generalized Fleiss’ kappa 0.86)
3. BMS Reports the US FDA Accelerated Approval for Zenbexus (Iberdomide) Combination in R/R Multiple Myeloma (MM)
Company: BMS
Product: Zenbexus
Active Ingredient: iberdomide
Disease: Multiple Myeloma
Date: Aug 13, 2026
Shots:
- FDA has granted accelerated approval to Zenbexus (iberdomide) + daratumumab & hyaluronidase-fihj + dexamethasone (ZDd) for the treatment of pts with MM who received ≥1L of prior therapy incl. a proteasome inhibitor & an immunomodulatory agent
- Approval was supported by the P-III (EXCALIBER-RRMM) trial assessing ZDd (n=207) vs daratumumab + bortezomib + dexamethasone (DVd; n=213) pts with r/r MM, which showed improvement across one of the dual 1EP of MRD-negative CR in 41% pts vs 21% at an mFU of 16mos.
- Additionally, an NDA for mezigdomide, a CELMoD, combined with carfilzomib & dexamethasone, is under FDA review, with a PDUFA target date of May 13, 2027
4. Regeneron Reports US FDA Approval of Pasatru for Fibrodysplasia Ossificans Progressiva
Company: Regeneron
Product: Pasatru
Active Ingredient: garetosmab-grts
Disease: Fibrodysplasia Ossificans Progressiva
Date: Aug 19, 2026
Shots:
- The US FDA has approved Pasatru (garetosmab-grts) to reduce the formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva (FOP), based on the P-III OPTIMA trial assessing 10 mg/kg (n=23) & 3 mg/kg (n=19) Pasatru vs PBO (n=21)
- At Wk. 56, Pasatru 10 mg/kg reduced the total number of new HO lesions by 90% vs PBO (2 vs 19 lesions), while the 3 mg/kg dose reduced lesions by 94% (1 vs 19 lesions), as assessed by CT scan
- At Wk. 56, clinician-assessed flare-ups were reduced by 88% with Pasatru 10 mg/kg (9 vs 66 events) and by 15% with 3 mg/kg (53 vs 66 events) vs PBO. The proportion of patients reporting flare-ups through Wk 56 did not differ significantly between the PBO and Pasatru treatment groups.
5. Revolution Medicines Reports the US FDA Approval of Rasonque (Daraxonrasib) for Previously Treated Metastatic PDAC
Company: Revolution Medicines
Product: Rasonque
Active Ingredient: Daraxonrasib
Disease: Pancreatic Ductal Adenocarcinoma
Date: Aug 26, 2026
Shots:
- FDA has approved Revolution’s Rasonque to treat pts with metastatic pancreatic ductal adenocarcinoma (PDAC) who have received ≥1 prior systemic therapy or who are not candidates for multiagent systemic therapy
- Approval was backed by P-III (RASolute 302) trial assessing Rasonque (300mg, QD, PO) vs CT, in above-mentioned pts, regardless of RAS mutation, which met all 1 & key 2EPs; data were published in The NEJM
- In the ITT population, Rasonque reduced death risk by 60% vs CT, improving mOS to 13.2 vs 6.7mos & mPFS to 7.2 vs 3.6mos, with consistent RAS G12 results. It also delayed deterioration in global health status (median TTD: 5.7 vs 2.6mos) & pain (median TTD: 9.2 vs 3.8mos)
6. The US FDA approves Priovant Therapeutics’ LISRAYA (brepocitinib) for Dermatomyositis
Company: Priovant Therapeutics
Product: LISRAYA
Active Ingredient: brepocitinib
Disease: Dermatomyositis
Date: Aug 27, 2026
- Priovant Therapeutics announced that the FDA has approved LISRAYA (brepocitinib) 30 mg, a once-daily oral treatment for adults with dermatomyositis (DM)
- The Phase 3 VALOR trial showed that LISRAYA improved dermatomyositis symptoms as early as Week 4, with benefits increasing and sustained through 52 weeks. It also helped reduce steroid dependence, with 55% of patients achieving moderate or better improvement with minimal/no steroid use, vs 30%
- LISRAYA is a first-in-class TYK2/JAK1 inhibitor and the first targeted therapy approved for dermatomyositis, offering improvements in skin disease, muscle strength, physical function, overall disease burden, and steroid reduction
7. Takeda Reports the US FDA Approval of Mimrylo (Rusfertide) for Polycythemia Vera
Company: Takeda
Product: Mimrylo
Active Ingredient: Rusfertide
Disease: Polycythemia Vera
Date: Aug 28, 2026
Shots:
- The US FDA has approved Mimrylo (rusfertide) for the treatment of erythrocytosis in adults with polycythemia vera (PV)
- Approval was backed by the P-III (VERIFY) trial (n=293), which met all efficacy endpoints, showing higher response rates, reduced need for phlebotomy & improvement in fatigue as measured by PROMIS Fatigue Short Form 8a
- The VERIFY OLE is ongoing, with Takeda planning to share further findings at upcoming medical conferences & working with regulators outside the US to expand Mimrylo access globally
