FDA accepts new drug application for donidalorsen

Key takeaways:

• Recent posters presented at the ACAAI Annual Scientific Meeting showed the safety and efficacy of donidalorsen.
• The FDA set an action date of Aug. 21, 2025.

The FDA accepted a new drug application for review of donidalorsen, a prophylactic RNA-targeting drug used to prevent hereditary angioedema attacks in patients aged 12 years and older, according to a press release from Ionis.

“Hereditary angioedema (HAE) is a rare genetic disease where patients experience recurring and unpredictable attacks of severe swelling, which can be life-threatening,” Kyle Jenne, executive vice president and chief global product strategy officer at Ionis, told Healio.

Kyle Jenne

“The unpredictability of attacks can greatly impact daily life. Despite currently available prophylactic treatments, many patients continue to experience painful and potentially life-threatening breakthrough attacks,” Jenne continued.

The new drug application was based on results from the phase 3 OASIS-HAE and OASISplus studies and the ongoing phase 2 open-label extension (OLE) study.

“The phase 3 clinical data demonstrate durable efficacy, unique switch data and a favorable tolerability and safety profile, with the simplicity of monthly or every 2 months self-administration via an autoinjector — attributes HCPs are looking for in a prophylactic treatment,” Jenne said.

In October, positive results from all three studies were presented at the American College of Asthma, Allergy & Immunology Annual Scientific Meeting.

In the ongoing phase 2 OLE and phase 3 studies, donidalorsen was found to reduce HAE attacks with an overall sustained mean reduction rate of 96% from baseline. This result was maintained up to 3 years. The studies also showed that donidalorsen was well tolerated with no serious treatment-emergent adverse events. The most common treatment-emergent adverse events were injection-site reactions.

“There is still an urgent need for a medicine that can effectively reduce HAE attacks, is well tolerated and is also simple to administer,” Jenne said. “With the convenience of monthly or every 2-month self-administration via autoinjector, we believe that donidalorsen has a unique profile that may address the needs of people living with HAE.”

The FDA set an action date of Aug. 21, 2025. Jenne explained that donidalorsen has the potential to be Ionis’ second independent commercial launch in addition to olezarsen, which is an antisense oligonucleotide targeting mRNA for apolipoprotein C-III for patients with familial chylomicronemia syndrome.

“We’re on the cusp of independently delivering a steady stream of new transformational medicines to patients,” Jenne said.

Reference:

• New positive donidalorsen data to be presented at 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting. https://ir.ionis.com/news-releases/news-release-details/new-positive-donidalorsen-data-be-presented-2024-american. Published Oct. 3, 2024. Accessed Nov. 6, 2024.