August 2025 was a landmark month for rare disease innovation, as the U.S. Food and Drug Administration (FDA) granted four historic approvals, each representing a first for their respective conditions, while also expanding a blockbuster drug’s use and updating COVID-19 vaccine recommendations.
Four Breakthroughs for Rare Diseases
BioSpace.com reported that the month began with Jazz Pharmaceuticals’ Modeyso (dordaviprone), which became the first systemic therapy approved for diffuse midline glioma with the H3 K27M mutation. Designed for patients as young as one year who have relapsed after prior therapies, Modeyso’s accelerated approval was based on early-phase trial data showing a 22% overall response rate and a median response duration of 10.3 months. Continued approval depends on confirmatory Phase III results expected in 2026.
Next, Insmed’s Brinsupri (brensocatib) made history as the first approved treatment for non-cystic fibrosis bronchiectasis, a rare respiratory disease, and the first to target the DPP1 enzyme. Brinsupri, taken orally, reduced pulmonary exacerbations by 20% in pivotal trials and is now available for patients 12 and older.
Precigen soon followed with Papzimeos (zopapogene imadenovec-drba), the first non-replicating immunotherapy for recurrent respiratory papillomatosis (RRP)—a rare, HPV-driven airway tumor disorder. In trials, Papzimeos achieved complete response in over half of patients and drastically reduced the need for surgical interventions.
Ionis Pharmaceuticals capped the month’s achievements with Dawnzera (donidalorsen), the first RNA-targeting prophylactic for hereditary angioedema (HAE). Given by monthly injection, Dawnzera reduced HAE attack rates by 81% in clinical studies, offering new hope for those living with this debilitating swelling disorder.
Wegovy’s Expanded Role in Liver Disease
Novo Nordisk’s GLP-1 drug Wegovy also scored a key FDA approval, now indicated for adults with metabolic dysfunction-associated steatohepatitis (MASH) and moderate to advanced liver scarring. Supported by Phase III trial data, Wegovy demonstrated significant improvements in liver fibrosis and steatohepatitis resolution. Analysts anticipate this expansion could add nearly $2 billion in peak sales for Novo Nordisk.
COVID-19 Vaccine Updates and Setback for Friedreich’s Ataxia
On the vaccine front, the FDA authorized updated COVID-19 shots from Pfizer, Moderna, and Novavax, but limited their use to adults 65+ or at-risk younger individuals. Notably, routine vaccination for healthy children and pregnant women was removed from CDC guidelines earlier this year.
However, not all news was positive: PTC Therapeutics’ vatiquinone was rejected for Friedreich’s ataxia after failing to show sufficient efficacy in trials. The FDA has requested additional, robust studies before reconsidering approval.
Conclusion
August 2025 underscored the FDA’s pivotal role in advancing rare disease care, delivering transformative new therapies and expanding existing treatments to broader populations, while maintaining rigorous standards for approval. These regulatory milestones offer hope for patients with limited options and signal continued momentum in the rare disease landscape.
