Government
Takeda snags expanded pediatric approval for rare genetic disorder drug
The FDA on Friday approved Takeda’s supplement to expand the use of Takhzyro, a hereditary angioedema (HAE) drug acquired as part of its $62 billion…
The FDA on Friday approved Takeda’s supplement to expand the use of Takhzyro, a hereditary angioedema (HAE) drug acquired as part of its $62 billion Shire deal, to children two to 12 years old.
The prophylactic monoclonal antibody is meant to prevent attacks of HAE, which can be painful, with severe swelling.
The single-dose prefilled syringe is recommended every four weeks in patients two to <6 years of age and every two weeks in patients 6 to <12 years of age, according to the company. Takhzyro first won FDA approval in 2018 for patients 12 years and older.
“Today’s approval of the expanded indication of TAKHZYRO represents a significant step forward for the HAE community,” Julie Kim, president of the US business unit and US country head at Takeda, said in a statement.
But competition may be coming soon for Takhzyro.
Late last year, Intellia unveiled its one-time, gene-editing treatment for HAE, called NTLA-2002. The treatment edits the gene underlying the protein responsible for the attacks. At the time, CEO John Leonard said it “may be a functional cure” after patients had no attacks or had them ranging from 10 weeks to 10 months after treatment.
But the only data available are from a small cohort of patients. According to Intellia’s study data, there were positive signs: in the three patients who got the low dose of kallikrein, levels were reduced by 64%, while levels were reduced in the three patients who got the high dose by 92%. At a middle dose of 50 mg, average kallikrein levels fell by 81%.
Swiss biotech Pharvaris was working on an oral drug, PHA121, for HAE, but the FDA halted the studies last August.
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