Key Takeaways

• Arrowhead Pharmaceuticals reported a Phase-3 study of its medicine for a rare genetic disorder met all its primary endpoints and key secondary endpoints.

• The drug, called Plozasiran, helped patients with familial chylomicronemia syndrome (FCS), which has no currently approved treatment.

• Arrowhead said it plans to file a new drug application with the Food and Drug Administration (FDA) this year.

Shares of Arrowhead Pharmaceuticals (ARWR) gained Tuesday after the biotech firm announced positive results in a late-stage study of its experimental treatment for a rare metabolic disorder, and said it plans to request regulatory approval to market it.

The company said a Phase 3 trial of its Plozasiran drug met the primary endpoint and all multiplicity-controlled key secondary endpoints for patients with familial chylomicronemia syndrome (FCS), a genetic disorder that has no approved medicine currently available.

Arrowhead said it plans to file a new drug application with the Food and Drug Administration (FDA) by the end of the year. It also will seek approval from medical authorities in other countries.

Arrowhead Chief Medical Scientist Dr. Bruce Given said the firm views Plozasiran as “potentially best-in-class and supportive of development across the spectrum of triglyceride disorders.”

Arrowhead Pharmaceuticals shares were up about 2% in early trading Tuesday, though despite Tuesday’s gains, they've lost over one-fifth of their value since the start of the year.

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