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 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.

Julie Kim

“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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