New HAE treatment Dawnzera now available to US patients
Developer Ionis partnering with Orsini specialty pharmacy
New hereditary angioedema (HAE) preventive treatment Dawnzera (donidalorsen) will now be available in the U.S. to adults and adolescents, ages 12 and older, through the specialty pharmacy Orsini.
Following the therapy’s approval late last month as a prophylaxis for HAE swelling attacks, developer Ionis Pharmaceuticals partnered with the nationwide pharmacy to provide Dawnzera to U.S. patients.
Dawnzera is the first and only RNA-targeted therapy approved for HAE. It’s provided in a single-dose autoinjector, which allows patients or caregivers to administer it themselves after proper training.
“We are honored to be trusted by Ionis as the exclusive specialty pharmacy partner for Dawnzera and eager to demonstrate the meaningful impact that Orsini’s exceptional care has on HAE patients,” Darin DeCarlo, Orsini’s chief commercial officer, said in a company press release. “For more than 14 years, Orsini has been a leader in HAE care. This collaboration underscores our mutual commitment to the HAE community and putting patients first.”
HAE is marked by recurrent swelling episodes that can occur in various areas of the body. It occurs due to genetic mutations that result in the excessive production of a signaling molecule called bradykinin. When produced in excess, bradykinin causes blood vessels to become more permeable, allowing fluid to leak out into surrounding tissues.
Dawnzera is an antisense oligonucleotide, which is a short, lab-made strand of genetic material. The therapy is designed to lower the production of prekallikrein, a precursor to kallikrein, which is an enzyme essential for bradykinin generation. By doing so, Dawnzera aims to prevent bradykinin levels from rising too high, thus reducing the frequency of swelling attacks.
Treatment with Dawnzera led to 81% drop in HAE attack rates in trial
The therapy’s approval was supported by data from the Phase 3 OASIS-HAE clinical trial (NCT05139810), which evaluated the safety and efficacy of two Dawnzera dosing regimens against a placebo in 90 adults and adolescents, ages 12 and older. All participants had HAE types 1 or 2, and were experiencing recurrent swelling attacks.
The trial’s results showed that once-per-month treatment with Dawnzera led to an 81% reduction in monthly HAE attack rates compared with the placebo after about six months. Additionally, the frequency of moderate to severe attacks and those necessitating on-demand treatment fell by about 90% with monthly dosing from the second dose to the study’s end.
These positive findings were further confirmed in OASISplus (NCT05392114), an open-label extension study that enrolled both patients who had completed OASIS-HAE and some who had never received Dawnzera and were on other preventive therapies. Reductions in attack rates observed in OASIS-HAE were maintained for as long as a year in OASISplus, with mean HAE attack rates dropping by more than 90% among patients receiving Dawnzera every month or every other month.
In the extension study, patients who transitioned from other HAE preventive treatments to Dawnzera saw their attack rates drop by a mean of 62% after about four months of monthly treatment. Most of these patients expressed a preference for Dawnzera, citing better control of their disease, a quicker administration process, and fewer issues with injection site pain or reactions compared with their previous treatments.
Dawnzera has demonstrated a favorable safety profile across various studies. The most frequently reported side effects include reactions at the injection site, upper respiratory tract infections, urinary tract infections, and abdominal discomfort.
An Orsini webpage says the pharmacy provides “customized, high-touch care” to patients with HAE and other rare diseases.
“We take a holistic team approach to care management, providing customized clinical support to patients and coordinating care with provider offices. This includes side effect monitoring, disease management, medication adherence support, and proactively suggesting solutions as needed to prevent discontinuations and enhance patient outcomes,” the webpage states.
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