While there has still yet to be a single gene therapy approved in the United States, Europe is now closer to marketing another: bluebird bio was just granted accelerated assessment status for LentiGlobin, a gene therapy that targets sickle cell disease.
While there has still yet to be a single gene therapy approved in the United States, Europe is now closer to marketing another: bluebird bio (NASDAQ:BLUE) was just granted accelerated assessment status for LentiGlobin, a gene therapy that targets sickle cell disease.
After reviewing bluebird bio’s application, the European Medicines Agency (EMA) accepted LentiGlobin into the PRIME—Priority Medications—program they developed last year. Similar to the FDA’s Breakthrough Therapy designation, acceptance into PRIME is limited to treatments that meet an as yet neglected medical need.
What does that mean for investors?
Acceptance into PRIME, or programs like it, are always of significant interest to investors. It means that a company’s product could get to market—and start making money—earlier than expected.
But it also signals great market potential. After all, these initiatives are intended to get life-saving medications to market for those who have few—or zero—other treatment options.
LentiGlobin compared to existing treatments
So how does LentiGlobin fit that criteria? The therapy is intended for patients with sickle cell disease—about 25 million people worldwide. It’s also in clinical trials for transfusion-dependent beta-thalassemia, or TDT.
Many patients with these two diseases are currently treated with hydroxyurea, however, toxicities associated with that drug can make it impossible for them to take the dose they require.
LentiGlobin attempts to correct the genetic mutation that causes sickle cell disease or TDT. It adds a functional beta-globin gene to patient stem cells and these stem cells are then transplanted into the blood stream.
If approved, therefore, it could have a strong impact on the market. Here is a therapy that stands to greatly improve the lives of millions—people who may find their current therapy wanting.
And indeed, LentiGlobin has had promising clinical trial results to date, significantly reducing the number of blood transfusions that patients required. More research is necessary, of course, but thanks to its newfound PRIME status, LentiGlobin could be on the European market sooner than anticipated.
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Securities Disclosure: I, Chelsea Pratt, hold no direct investment interest in any company mentioned in this article.