
5 First-in-Class, First-in-Disease FDA Approvals From August
Key Takeaways
- Dordaviprone is the first approved therapy for recurrent H3 K27M-mutant diffuse midline glioma, showing a 22% overall response rate in clinical trials.
- Brensocatib, a DPP1 inhibitor, is the first approved therapy for non-cystic fibrosis bronchiectasis, reducing pulmonary exacerbation rates significantly.
From respiratory conditions to rare diseases and beyond, here are 5 groundbreaking FDA approvals from last month.
August was another busy month for the
Here are 5 of the most notable August FDA approvals:
FDA Approves Dordaviprone as First and Only Treatment for Aggressive Form of Glioma
On August 6, the FDA granted
The approval was supported by an efficacy analysis of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma enrolled in 5 open-label clinical trials: ONC006 (
The median duration of response was 10.3 months (95% CI, 7.3-15.2), with 73% of patients maintaining their response for at least 6 months and 27% for at least 12 months. The pooled analysis also showed a median time of response of 8.3 months (range, 1.9-15.9) with dordaviprone.
"This approval represents a long-awaited treatment option for families affected by H3 K27M-mutant diffuse midline glioma," David F. Arons, president and chief executive officer of the National Brain Tumor Society,
Brensocatib Becomes First FDA-Approved Therapy for Bronchiectasis
The FDA’s
This approval was based on data from the ASPEN trial (
Annualized pulmonary exacerbation rates were 1.02 with 10 mg, 1.04 with 25 mg, and 1.29 with placebo, yielding rate ratios (RRs) of 0.79 (95% CI, 0.68-0.92; adjusted P = .004) for 10 mg and 0.81 (95% CI, 0.69-0.94; adjusted P = .005) for 25 mg vs placebo. At week 52, 48.5% of patients treated with brensocatib remained exacerbation-free vs 40.3% treated with placebo. The RRs for remaining exacerbation-free were 1.20 (95% CI, 1.06-1.37; adjusted P = .02) for 10 mg and 1.18 (95% CI, 1.04-1.34; adjusted P = .04) for 25 mg.
“[Brensocatib is] the first treatment that targets neutrophilic inflammation, which is the underlying driving force for a lot of exacerbations,” James D. Chalmers, MBChB, PhD, ASPEN trial lead investigator,
FDA Approves Zopapogene Imadenovec for Recurrent Respiratory Papillomatosis
On August 19, the FDA
Zopapogene imadenovec-drba is a nonreplicating adenoviral vector-based immunotherapy that expresses a fusion antigen made up of selected regions of HPV types 6 and 11. It is administered via 4 subcutaneous injections over a 12-week interval.
This approval was based on data from an open-label, single-arm, pivotal study (
"This long-awaited FDA approval represents a momentous milestone for the RRP community," Kim McClellan, president of the Recurrent Respiratory Papillomatosis Foundation,
FDA Approves First RNA-Targeted Treatment for Hereditary Angioedema
The FDA
Donidalorsen is self-administered in 80-mg doses through a subcutaneous autoinjector and can be taken every 4 or 8 weeks. Its approval was based on the results of the phase 3 OASIS-HAE trial (
The monthly HAE attack rate decreased by 81% (95% CI, 65%-89%) through 24 weeks among patients who took donidalorsen every 4 weeks. Similarly, the median reduction in attack rate from baseline was 90% in those who received the medication every 4 weeks.
In comparison, the mean attack rate for participants taking the dose every 8 weeks was 55% lower (95% CI, 22%-74%) compared with placebo. Also, the median reduction in attack rate was 83% compared with only 16% in the placebo group. The results among both donidalorsen groups prove that it was effective in reducing the number of HAE attacks.
“[Donidalorsen] represents a significant advance for people living with HAE who need improved treatment options,” Brett P. Monia, PhD, CEO of Ionis,
Rilzabrutinib Becomes First FDA-Approved BTK Inhibitor for ITP
On August 30, the FDA
This decision was based on results from the phase 3 LUNA 3 study (
Of the adult patients analyzed, 133 received rilzabrutinib and 69 received a placebo. At week 25, a durable platelet response was achieved in 23% (n = 31) of patients receiving rilzabrutinib and 0% of those on placebo (P < .0001). Based on data from both study periods, a durable response was achieved in 29% (n = 38) of patients treated with rilzabrutinib.
"With its differentiated mechanism of action, Wayrilz has the potential to become a treatment of choice for immune thrombocytopenia patients who have not responded to a prior therapy," Brian Foard, executive vice president and head of specialty care at Sanofi,
References
- McNulty R. FDA approves dordaviprone as first and only treatment for aggressive form of glioma. AJMC. August 6, 2025. Accessed September 12, 2025.
https://www.ajmc.com/view/fda-approves-dordaviprone-as-first-and-only-treatment-for-aggressive-form-of-glioma - Jazz Pharmaceuticals announces U.S. FDA approval of Modeyso (dordaviprone) as the first and only treatment for recurrent H3K27M-mutant diffuse midline glioma. News release. Jazz Pharmaceuticals. August 6, 2025. Accessed September 12, 2025.
https://investor.jazzpharma.com/news-releases/news-release-details/jazz-pharmaceuticals-announces-us-fda-approval-modeysotm - McCormick B. Brensocatib becomes first FDA-approved therapy for bronchiectasis. AJMC. August 12, 2025. Accessed September 12, 2025.
https://www.ajmc.com/view/brensocatib-becomes-first-fda-approved-therapy-for-bronchiectasis - McCormick B, Chalmers JD. Brensocatib becomes first approved therapy for bronchiectasis, expanding treatment options: James D. Chalmers, MBChB, PhD. AJMC. August 20, 2025. Accessed September 12, 2025.
https://www.ajmc.com/view/brensocatib-becomes-first-approved-therapy-for-bronchiectasis-expanding-treatment-options-james-d-chalmers-mbchb-phd - McNulty R. FDA approves zopapogene imadenovec for recurrent respiratory papillomatosis. AJMC. August 19, 2025. Accessed September 12, 2025.
https://www.ajmc.com/view/fda-approves-zopapogene-imadenovec-for-recurrent-respiratory-papillomatosis - Precigen announces full FDA approval of Papzimeos (zopapogene imadenovec-drba), the first and only approved therapy for the treatment of adults with recurrent respiratory papillomatosis. News release. Precigen. August 15, 2025. Accessed September 12, 2025.
https://investors.precigen.com/news-releases/news-release-details/precigen-announces-full-fda-approval-papzimeos-zopapogene - Bonavitacola J. FDA approves first RNA-targeted treatment for hereditary angioedema. August 21, 2025. Accessed September 12, 2025.
https://www.ajmc.com/view/fda-approves-first-rna-targeted-treatment-for-hereditary-angioedema - Dawnzera (donidalorsen) approved in the US as first and only RNA-targeted prophylactic treatment for hereditary angioedema. News release. Business Wire. August 21, 2025. Accessed August 21, 2025.
https://www.businesswire.com/news/home/20250818615141/en/DAWNZERA-donidalorsen-approved-in-the-U.S.-as-first-and-only-RNA-targeted-prophylactic-treatment-for-hereditary-angioedema - McCormick B. Rilzabrutinib becomes first FDA-approved BTK inhibitor for ITP. AJMC. August 30, 2025. Accessed September 12, 2025.
https://www.ajmc.com/view/rilzabrutinib-becomes-first-fda-approved-btk-inhibitor-for-itp - ASH: rilzabrutinib demonstrated significant patient benefit in the first positive phase 3 study of a BTK inhibitor in ITP. News release. Sanofi. December 7, 2024. Accessed August 30, 2025.
https://www.sanofi.com/en/media-room/press-releases/2024/2024-12-07-16-30-00-2993342
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