Marketdash
Back to News

Arrowhead Pharma Wins FDA Nod for First Self-Injected Treatment for Ultra-Rare Fat Disorder

MarketDash Editorial Team
19 days ago
Arrowhead Pharmaceuticals scored FDA approval for Redemplo, a groundbreaking siRNA therapy that slashes dangerously high triglyceride levels in adults with familial chylomicronemia syndrome and can be self-administered at home every three months.

The FDA just handed Arrowhead Pharmaceuticals (ARWR) approval for Redemplo (plozasiran), and this one's kind of a big deal. It's the first siRNA medicine cleared to treat familial chylomicronemia syndrome, which is about as rare and nasty as metabolic disorders get.

Here's what makes FCS so problematic: it's an inherited condition where your body either lacks or can't properly use an enzyme called lipoprotein lipase. Without it, triglycerides—basically fat particles in your blood—accumulate to wildly dangerous levels. We're talking 10 to 100 times higher than what's considered normal. Around 6,500 people in the U.S. are living with genetic or clinical FCS, walking around with ticking time bombs of fat in their bloodstreams.

Redemplo works as an adjunct to diet, targeting those sky-high triglyceride levels with small interfering RNA technology. What makes it particularly noteworthy is the delivery method: patients can self-administer it at home with a simple subcutaneous injection once every three months. No hospital visits, no frequent appointments—just quarterly shots you can handle yourself.

The approval rides on results from the Phase 3 PALISADE study, which hit all the marks the FDA wanted to see. Patients taking 25 mg of Redemplo saw a median triglyceride reduction of 80% from baseline, compared to just 17% in the placebo group. The study also showed significant reductions in APOC3 (another lipid marker) and a lower rate of acute pancreatitis compared to placebo—which matters enormously, since pancreatitis is one of the scariest complications FCS patients face.

For Arrowhead, this represents a transformative moment. Redemplo is the company's first FDA-approved medicine, built on their proprietary Targeted RNAi Molecule (TRiM) platform. It's the validation every biotech dreams about—going from clinical-stage hopeful to commercial-stage reality.

The drug will hit the U.S. market before the year wraps up, giving FCS patients their first targeted treatment option that doesn't require constant medical supervision.

Price Action: ARWR stock climbed 6.00% to $42.56 on Tuesday following the announcement.

Back to News

Arrowhead Pharma Wins FDA Nod for First Self-Injected Treatment for Ultra-Rare Fat Disorder

MarketDash Editorial Team
19 days ago
Arrowhead Pharmaceuticals scored FDA approval for Redemplo, a groundbreaking siRNA therapy that slashes dangerously high triglyceride levels in adults with familial chylomicronemia syndrome and can be self-administered at home every three months.

The FDA just handed Arrowhead Pharmaceuticals (ARWR) approval for Redemplo (plozasiran), and this one's kind of a big deal. It's the first siRNA medicine cleared to treat familial chylomicronemia syndrome, which is about as rare and nasty as metabolic disorders get.

Here's what makes FCS so problematic: it's an inherited condition where your body either lacks or can't properly use an enzyme called lipoprotein lipase. Without it, triglycerides—basically fat particles in your blood—accumulate to wildly dangerous levels. We're talking 10 to 100 times higher than what's considered normal. Around 6,500 people in the U.S. are living with genetic or clinical FCS, walking around with ticking time bombs of fat in their bloodstreams.

Redemplo works as an adjunct to diet, targeting those sky-high triglyceride levels with small interfering RNA technology. What makes it particularly noteworthy is the delivery method: patients can self-administer it at home with a simple subcutaneous injection once every three months. No hospital visits, no frequent appointments—just quarterly shots you can handle yourself.

The approval rides on results from the Phase 3 PALISADE study, which hit all the marks the FDA wanted to see. Patients taking 25 mg of Redemplo saw a median triglyceride reduction of 80% from baseline, compared to just 17% in the placebo group. The study also showed significant reductions in APOC3 (another lipid marker) and a lower rate of acute pancreatitis compared to placebo—which matters enormously, since pancreatitis is one of the scariest complications FCS patients face.

For Arrowhead, this represents a transformative moment. Redemplo is the company's first FDA-approved medicine, built on their proprietary Targeted RNAi Molecule (TRiM) platform. It's the validation every biotech dreams about—going from clinical-stage hopeful to commercial-stage reality.

The drug will hit the U.S. market before the year wraps up, giving FCS patients their first targeted treatment option that doesn't require constant medical supervision.

Price Action: ARWR stock climbed 6.00% to $42.56 on Tuesday following the announcement.

    Arrowhead Pharma Wins FDA Nod for First Self-Injected Treatment for Ultra-Rare Fat Disorder - MarketDash News