FDA Approves CSL's Hereditary Angioedema Drug as Gene Editing Treatment Shows Promise

The pharmaceutical industry saw significant developments this week, with new drug approvals, promising clinical trial results, and corporate maneuvers shaping the landscape of rare disease treatments and obesity medications.

CSL's Andembry Gains FDA Approval for Hereditary Angioedema

The U.S. Food and Drug Administration (FDA) has approved CSL's Andembry (garadacimab) for the treatment of hereditary angioedema (HAE). This first-of-its-kind, once-monthly injection prevents swelling attacks associated with the condition. The approval comes just days after the FDA delayed a decision on a rival HAE drug from Kalvista Therapeutics, citing "resource constraints."

Andembry's approval marks a significant milestone in HAE treatment, offering patients a new option with a convenient dosing schedule. The timing of this approval is noteworthy, as the FDA is also expected to decide on Ionis Pharmaceuticals' donidalorsen, another HAE therapy, by August 21.

Intellia's Gene Editing Treatment Shows Lasting Effects in HAE Patients

Intellia Therapeutics has reported promising results from its Phase 1 trial of lonvo-z, a gene editing treatment for hereditary angioedema. The company revealed that a single dose of lonvo-z led to a 98% average reduction in monthly swelling attacks across all 10 treated patients, with effects persisting for up to three years in follow-up studies.

Notably, all participants have remained off other medications for a median of 23 months. These findings support lonvo-z's potential as a best-in-class treatment for HAE. However, Leerink Partners analyst Mani Foroohar notes that Intellia's "valuation disconnect" may persist until late-stage results are released or investor concerns about the company's financial footing are addressed. Late-stage data could be available as early as next year, according to a federal database.

Shifting Landscape in Obesity Treatment and Genetic Testing

In other industry news, Eli Lilly has expanded its online service for its obesity medicine Zepbound. The company will now provide all doses of Zepbound through LillyDirect, an online platform that sells the drug to individuals without health insurance or those who choose to pay out of pocket. The 12.5 and 15 milligram weekly vials will be available for $499 per month, in line with other doses of at least 5 milligrams.

Meanwhile, in the genetic testing sector, 23andMe's bankruptcy proceedings took an unexpected turn. A group led by the company's cofounder and former CEO Anne Wojcicki, operating under the name TTAM Research Institute, won an auction to buy the DNA testing firm out of bankruptcy with a $305 million offer. This development comes after Regeneron Pharmaceuticals had initially won the auction last month. Regeneron will now serve as the "backup bidder" pending court approval and the deal's closing, which is expected in the coming weeks.