Ionis Pharma Wins FDA Nod For First RNA-Targeted Therapy For Rare Genetic Swelling Disorder

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On Thursday, the U.S. Food and Drug Administration (FDA) approved Ionis Pharmaceuticals, Inc.’s (NASDAQ:IONS) Dawnzera (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older.

Dawnzera is the first and only RNA-targeted medicine approved for HAE, designed to target plasma prekallikrein (PKK), a key protein that activates inflammatory mediators associated with acute attacks of HAE.

HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect approximately 7,000 people in the U.S.

Also Read: Ionis Posts Surprise Q2 Profit, Double Revenues On Stronger Sales From Newly Approved Genetic Disease Drug

Dawnzera 80mg is self-administered via subcutaneous autoinjector once every four (Q4W) or eight weeks (Q8W).

The approval was based on positive results from the Phase 3 OASIS-HAE study.

The study met its primary endpoint, with Dawnzera Q4W significantly reducing the monthly HAE attack rate by 81% compared to placebo over 24 weeks.

Mean attack rate reduction increased to 87% when measured from the second dose, a key secondary endpoint.

Additionally, Dawnzera Q4W reduced moderate-to-severe HAE attacks by around 90% over 24 weeks when measured from the second dose.

The OASISplus study also includes a switch cohort evaluating Dawnzera Q4W in patients previously treated with Takeda Pharmaceutical Co Ltd’s (NYSE:TAK) Takhzyro (lanadelumab) or BioCryst Pharmaceuticals Inc.’s (NASDAQ:BCRX) Orladeyo (berotralstat) for at least 12 weeks.

Switching to Dawnzera reduced the mean HAE attack rate by 62% from prior prophylactic treatment over 16 weeks, with no mean increase in breakthrough attacks observed during the switch.

In June, BioCryst agreed to sell its European Orladeyo (berotralstat) business to Neopharmed Gentili for up to $264 million.

In July, the FDA approved KalVista Pharmaceuticals Inc.’s (NASDAQ:KALV) Ekterly (sebetralstat) for acute attacks of HAE in adult and pediatric patients aged 12 years and older.

Ekterly is the first and only oral on-demand treatment for HAE. HAE is characterized by recurrent episodes of severe swelling (angioedema).

In June, the FDA approved CSL Limited’s (OTC:CSLLY) Andembry (garadacimab-gxii), the only treatment targeting factor XIIa for prophylactic use to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older.

Andembry, the only treatment to offer once-monthly dosing from the start for all patients, is a subcutaneous self-injection delivered in 15 seconds or less via an autoinjector with a citrate-free formula.

Price Action: IONS stock is trading higher by 0.91% to $43.20 at last check Thursday.

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