FDA Green Lights Drugs for Two Rare Diseases  

Big news out of the FDA last week!

Alexion Pharmaceuticals got approval for ALXN1210 for paroxysmal nocturnal hemoglobinuria, or PNH, which will now be sold as Ultomiris, while Stemline got approval to market Elzonris (tagraxofusp-erzs) for rare cases of blastic plasmacytoid dendritic cell neoplasm, or BPDCN.

These very rare diseases now have a much greater chance to find effective, stable treatment!

What is Paroxysmal Nocturnal Hemoglobinuria?

PNH is a rare acquired, life-threatening disease of the blood. The disease is characterized by destruction of red blood cells (hemolytic anemia), blood clots (thrombosis), and impaired bone marrow function (not making enough of the three blood components). PNH affects 1-1.5 persons per million of the population and is primarily a disease of younger adults.

The median survival after diagnosis is 10 years; however, some patients can survive for decades with only minor symptoms.

What is Blastic Plasmacytoid Dendritic Cell Neoplasm?

BPDCN is a rare and aggressive hematologic malignancy of the bone marrow and blood that can affect other organs such as the lymph nodes, spleen, central nervous system, and skin. The cornerstone symptom of BPDCN are deep, purple-colored skin lesions.

What’s Next?

Alexion is now in the pricing phase for Ultomiris (to treat PNH) while Stemline’s Elzonris comes with a black box warning for potential side effects, but because BPDCN has no approved drugs, the FPA put it on its priority list.

With this move, the FDA has now approved 59 new drugs this year, making 2018 a record-setting year.

Here’s to hoping for record setting years in cures and treatments!

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