The drug, for hereditary angioedema, is Ionis’ second wholly owned asset.
The FDA on Thursday approved Ionis Pharmaceuticals’ donidalorsen for the prevention of disease attacks in patients with hereditary angioedema. The product will carry the brand name Dawnzera.
Dawnzera is the first and so far only RNA-targeting therapy approved as a prophylactic treatment for hereditary angioedema (HAE), Ionis said in its Thursday news release. The product, which can be used in patients 12 years and above, will launch in the U.S. in the coming days.
“We see Dawnzera’s profile as superior to current prophylactic options, with less frequent dosing and at-home auto-injector dosing being further differentiators,” William Blair analysts told investors in a Thursday note, adding that Dawnzera marks Ionis’ second wholly owned asset. “We view Dawnzera as a highly competitive HAE prophylactic.”
Data from the OASIS-HAE study backed Thursday’s approval. Results published in the New England Journal of Medicine in May 2024 showed that over 24 weeks of follow-up, the RNA-targeted therapy lowered the monthly HAE attack rate by 81% versus placebo. Dawnzera also demonstrated an approximately 90% reduction in moderate-to-severe HAE attacks over 24 weeks when measured from the second dose, and significantly improved patients’ quality of life.
Ionis filed data from the Phase III OASISplus open-label extension study to bolster its application. According to Thursday’s release, Dawnzera treatment resulted in a 94% drop in the total mean attack rate from baseline at one year of follow-up. Meanwhile, a readout last month demonstrated that patients who switched to the RNA-targeting therapy from other prophylactics did so without experiencing spikes in attacks.
In a survey, 84% of patients indicated that they preferred Dawnzera over their previous prophylactic.
Administered subcutaneously every four weeks, Dawnzera is a conjugated antisense oligonucleotide that targets the prekallikrein mRNA, causing its destruction. This reduces overall levels of PKK, a protein that plays a role in swelling and pain attacks in HAE. Dawnzera does not come with a boxed warning, though its label has precautions for hypersensitivity reactions such as anaphylaxis.
Dawnzera’s approval on Thursday continues a series of history-making approvals for the rare disease space this month. Leading the pack is Jazz Pharmaceuticals, which on Aug. 6 won the FDA’s go-ahead for dordaviprone, now marketed as Modeyso, for the treatment of diffuse midline glioma with an H3 K27M mutation in patients aged one year and up who have progressed after previous interventions. Modeyso is the first drug approved for this ultrarare brain tumor.
A week later, Insmed’s brensocatib won approval for non-cystic fibrosis bronchiectasis for patients 12 and over. The drug, branded as Brinsupri, is the first therapy approved for this indication and is also the first DPP1 inhibitor to win the FDA’s blessing. Finally, Precigen on Aug. 14 bagged the industry’s first immunotherapy approval for recurrent respiratory papillomatosis with zopapogene imadenovec-drba, which will carry the brand name Papzimeos.
