Shots:
- PharmaShots has compiled a list of US FDA-approved drugs in the month of August 2025
- The US FDA has approved a total of 5 new drugs, including 5 new molecular entities, leading to the treatment of patients and advances in the pharmaceutical industry
- The major highlighted drug was Jazz Pharmaceuticals’ Modeyso, securing FDA approval for Recurrent H3 K27M-mutant Diffuse Midline Glioma
Company: Jazz Pharmaceuticals
Product: Modeyso
Active Ingredient: Dordaviprone
Disease: Recurrent H3 K27M-mutant Diffuse Midline Glioma
Date: Aug 06, 2025
Shots:
- The FDA has granted accelerated approval for Jazz Pharmaceuticals’ Modeyso (dordaviprone) to treat diffuse midline glioma with an H3 K27M mutation in pts (aged≥ 1 yrs.) with progressive disease after prior therapy. Continued approval depends on results from the P-III (ACTION) trial
- Approval was based on data from 50 pts across 5 studies, showing a 22% ORR per blinded central review using RANO 2.0 criteria. MDoR (10.3mos.), with 73% responding for at least 6 mos. and 27% for at least 12 mos.
- Modeyso’s safety was assessed in 376 glioma pts across 4 open-label studies, with SAR reported in 33% of pts.
Company: Boehringer Ingelheim
Product: Hernexeos
Active Ingredient: Zongertinib
Disease: Inoperable or Metastatic NSCLC Harboring HER2 Mutations
Date: Aug 08, 2025
Shots:
- The US FDA has granted accelerated approval to Hernexeos (zongertinib) for the treatment of pts with inoperable or metastatic NSCLC harboring HER2 (ERBB2) mutations who have received prior systemic therapy
- Approval was based on P-Ib (Beamion LUNG-1) trial assessing Hernexeos as monotx., which showed improved ORR of 75% (N=71: 6% CR & 69% PR) & a ≥6mos. DoR in 58% pts (n=53); results were shared at AACR 2025 & published in The NEJM
- Hernexeos, a tyrosine kinase inhibitor (TKI) that selectively inhibits HER2, is being evaluated in multiple trials, across advanced solid tumors with HER2 alterations
Company: Insmed
Product: Brinsupri
Active Ingredient: Brensocatib
Disease: Non-Cystic Fibrosis Bronchiectasis
Date: Aug 12, 2025
Shots:
- The US FDA has approved Brinsupri (10 & 25mg) for treating NCFB pts (≥12yrs.); it is now available in the US. MAA is under EMA & MHRA review with a Japan filing planned in 2025 & potential launches in 2026 pending approvals
- Approval was based on P-III (ASPEN) trial (n=1721) & P-II (WILLOW) trial (n=256) assessing Brinsupri (10 or 25mg, PO) vs PBO in NCFB pts for 52 & 24wks., respectively, with WILLOW enrolling pts who had ≥2 documented pulmonary exacerbations in 12mos. prior to screening
- In ASPEN, Brinsupri (10 & 25mg) reduced annual exacerbations by 21.1% & 19.4%, showing benefits across exacerbation-related 2EPs with delayed first exacerbation, increased exacerbation-free pts; pts dosed with 25mg showed lower lung function decline per FEV₁ at wk. 52
Company: Ionis
Product: Dawnzera
Active Ingredient: Donidalorsen
Disease: Hereditary Angioedema
Date: Aug 21, 2025
Shots:
- The US FDA has approved Dawnzera (donidalorsen; 80mg SC, Q4W/Q8W) as a prophylactic treatment against attacks in pts (≥12yrs.) with hereditary angioedema
- Approval was based on P-III (OASIS-HAE) trial & OASISplus OLE study, where P-III showed 81% fewer monthly HAE attacks over 24wks. (87% from 2nd dose) & ~90% fewer mod. to sev. attacks, while OLE showed Q8W matched Q4W, with 94% reduction after 1yr.
- OASISplus also incl. a switch cohort of pts previously treated with lanadelumab, C1-esterase inhibitor, or berotralstat for ≥12wks. who switched to Dawnzera (Q4W) & observes a 62% attack rate reduction over 16wks. with no increase in breakthrough attacks; 84% preferred Dawnzera for better control, convenience, & less pain
Company: Sanofi
Product: Wayrilz
Active Ingredient: Rilzabrutinib
Disease: Immune Thrombocytopenia
Date: Aug 29, 2025
Shots:
- FDA has approved Wayrilz for adults with persistent or chronic ITP who did not respond adequately to prior therapy following approval in UAE in Jun 2025; Application under the EMA & NMPA’s review
- Approval was based on P-III (LUNA 3) trial assessing Wayrilz (400mg, BID, PO) vs PBO in adults (n=202) with ITP over 12wks., after which 64% vs 32% achieving durable platelet count continued for 24wks., followed by 28wk. OLE & 4wk. safety follow-up; study is ongoing in adolescents
- Trial met its 1 & 2EPs, showing sustained platelet response at 25wks. (23% vs 0%), faster onset (36 days vs not reached), & longer duration (7 vs 0.7wks.). Pts also achieved a 10.6-point QoL improvement vs 2.3 per ITP Patient Assessment Questionnaire; data was presented at ASH’24
Related Post: Insights+: The US FDA New Drug Approvals in July 2025
